FUTURE TARGETED HEALTHCARE MANUFACTURING HUB
Lead Research Organisation:
University College London
Department Name: Biochemical Engineering
Abstract
By 2025 targeted biological medicines, personalised and stratified, will transform the precision of healthcare prescription, improve patient care and quality of life. Novel manufacturing solutions have to be created if this is to happen. This is the unique challenge we shall tackle. The current "one-size-fits-all" approach to drug development is being challenged by the growing ability to target therapies to only those patients most likely to respond well (stratified medicines), and to even create therapies for each individual (personalised medicines). Over the last ten years our understanding of the nature of disease has been transformed by revolutionary advances in genetics and molecular biology.
Increasingly, treatment with drugs that are targeted to specific biomarkers, will be given only to patient populations identified as having those biomarkers, using companion diagnostic or genetic screening tests; thus enabling stratified medicine. For some indications, engineered cell and gene therapies are offering the promise of truly personalised medicine, where the therapy itself is derived at least partly from the individual patient. In the future the need will be to supply many more drug products, each targeted to relatively small patient populations. Presently there is a lack of existing technology and infrastructure to do this, and current methods will be unsustainable. These and other emerging advanced therapies will have a critical role in a new era of precision targeted-medicines. All will have to be made economically for healthcare systems under extreme financial pressure. The implications for health and UK society well-being are profound
There are already a small number of targeted therapies on the market including Herceptin for breast cancer patients with the HER2 receptor and engineered T-cell therapies for acute lymphoblastic leukaemia. A much greater number of targeted therapies will be developed in the next decade, with some addressing diseases for which there is not currently a cure. To cope, the industry will need to create smarter systems for production and supply to increasingly fragmented markets, and to learn from other sectors. Concepts will need to address specific challenges presented by complex products, of processes and facilities capable of manufacture at smaller scales, and supply chains with the agility to cope with fluctuating demands and high levels of uncertainty.
Innovative bioprocessing modes, not currently feasible for large-scale manufacturing, could potentially replace traditional manufacturing routes for stratified medicines, while simultaneously reducing process development time. Pressure to reduce development costs and time, to improve manufacturing efficiency, and to control the costs of supply, will be significant and will likely become the differentiating factor for commercialisation.
We will create the technologies, skill-sets and trained personnel needed to enable UK manufacturers to deliver the promise of advanced medical precision and patient screening. The Future Targeted Healthcare Manufacturing Hub and its research and translational spokes will network with industrial users to create and apply the necessary novel methods of process development and manufacture. Hub tools will transform supply chain economics for targeted healthcare, and novel manufacturing, formulation and control technologies for stratified and personalised medicines. The Hub will herald a shift in manufacturing practice, provide the engineering infrastructure needed for sustainable healthcare. The UK economy and Society Wellbeing will gain from enhanced international competitiveness.
Increasingly, treatment with drugs that are targeted to specific biomarkers, will be given only to patient populations identified as having those biomarkers, using companion diagnostic or genetic screening tests; thus enabling stratified medicine. For some indications, engineered cell and gene therapies are offering the promise of truly personalised medicine, where the therapy itself is derived at least partly from the individual patient. In the future the need will be to supply many more drug products, each targeted to relatively small patient populations. Presently there is a lack of existing technology and infrastructure to do this, and current methods will be unsustainable. These and other emerging advanced therapies will have a critical role in a new era of precision targeted-medicines. All will have to be made economically for healthcare systems under extreme financial pressure. The implications for health and UK society well-being are profound
There are already a small number of targeted therapies on the market including Herceptin for breast cancer patients with the HER2 receptor and engineered T-cell therapies for acute lymphoblastic leukaemia. A much greater number of targeted therapies will be developed in the next decade, with some addressing diseases for which there is not currently a cure. To cope, the industry will need to create smarter systems for production and supply to increasingly fragmented markets, and to learn from other sectors. Concepts will need to address specific challenges presented by complex products, of processes and facilities capable of manufacture at smaller scales, and supply chains with the agility to cope with fluctuating demands and high levels of uncertainty.
Innovative bioprocessing modes, not currently feasible for large-scale manufacturing, could potentially replace traditional manufacturing routes for stratified medicines, while simultaneously reducing process development time. Pressure to reduce development costs and time, to improve manufacturing efficiency, and to control the costs of supply, will be significant and will likely become the differentiating factor for commercialisation.
We will create the technologies, skill-sets and trained personnel needed to enable UK manufacturers to deliver the promise of advanced medical precision and patient screening. The Future Targeted Healthcare Manufacturing Hub and its research and translational spokes will network with industrial users to create and apply the necessary novel methods of process development and manufacture. Hub tools will transform supply chain economics for targeted healthcare, and novel manufacturing, formulation and control technologies for stratified and personalised medicines. The Hub will herald a shift in manufacturing practice, provide the engineering infrastructure needed for sustainable healthcare. The UK economy and Society Wellbeing will gain from enhanced international competitiveness.
Planned Impact
WHO WILL BENEFIT FROM THE RESEARCH
The impact of the Future Targeted Healthcare Manufacturing Hub will extend far beyond the Hub itself with significant benefits for manufacturing companies, healthcare providers, UK citizens and the economy as a whole.
Companies
The stratified protein medicines and cellular therapies of the future cannot be made or developed using the manufacturing technologies in use for current therapies. A radical step-change and innovation to deliver the economically viable processes is required. UK companies will benefit from new process technologies designed to deliver these step changes with the necessary cost performance and speed of development. The companies will also benefit from new decision support tools that will provide a robust framework for selecting processes and manufacturing options reducing the risk of misallocating significant cost and time. The researchers trained in the Hub will have the skills and experience required by industry to transfer and implement these technologies in practice.
Healthcare Providers
It is recognised that in some diseases only 20-30% of patients are effectively treated by the standard treatment care and that there may be serious adverse reactions among those that are not. This represents a very significant wasted resource in time and materials for healthcare providers under enormous pressure to reduce costs. Targeted medicines are designed to match effective treatments to patients but this promise can only be delivered if new therapies can be developed more quickly than currently and if they have greater cost effectiveness including lower manufacturing costs than existing medicines. The processes and decision-support tools developed in the Hub will enable product manufacturers to meet these challenges.
Patients
Faster development and more economic processes for targeted therapeutics will allow the promise of precision to be realised for the patient. They will enable patients to access promptly new stratified and personalised therapies, in some cases for diseases not currently treated and they will reduce ineffective treatments and adverse side effects.
UK Economy
The UK has a strong established pharmaceutical industry and a growing number of new companies developing new protein and cellular therapies. The development of new processes and tools to guide selection of processes and manufacturing option will enable this sector to commercialise successfully their rapidly increasing research effort in targeted medicines and increase the GVA of this part of the pharmaceutical industry.
HOW WILL THEY BENEFIT FROM THE RESEARCH
The Grand Challenge research in the hub will create new process technologies to address key manufacturing challenges identified following extensive discussion with the industry. Additionally the Hub with create new decision-support tools that will be used to accelerate the development and optimisation of manufacturing processes at appropriate scales. These tools will address the complexity of new clinical trial structures and increasingly decentralised manufacturing.
User-led feasibility projects with companies of the User Group, part of the Hub's platform research, will help test and evaluate these products. The Hub will have IP arrangements designed to achieve rapid exploitation and broad uptake and will involve three Catapults to facilitate translation of the technologies developed to market. 18 PDRAS, 28 CDT, 23 PhD and 14 EngD researchers on projects aligned to the hub will be trained with the process development skills required by industry. The Hub will engage with regulators to ensure they are briefed and informed on the emerging new technologies to allow appropriate regulatory strategies to be developed and put in place.
The successful commercialisation of new targeted biological medicines by companies will strengthen the UK manufacturing community with direct impacts on people employed and UK wealth generated.
The impact of the Future Targeted Healthcare Manufacturing Hub will extend far beyond the Hub itself with significant benefits for manufacturing companies, healthcare providers, UK citizens and the economy as a whole.
Companies
The stratified protein medicines and cellular therapies of the future cannot be made or developed using the manufacturing technologies in use for current therapies. A radical step-change and innovation to deliver the economically viable processes is required. UK companies will benefit from new process technologies designed to deliver these step changes with the necessary cost performance and speed of development. The companies will also benefit from new decision support tools that will provide a robust framework for selecting processes and manufacturing options reducing the risk of misallocating significant cost and time. The researchers trained in the Hub will have the skills and experience required by industry to transfer and implement these technologies in practice.
Healthcare Providers
It is recognised that in some diseases only 20-30% of patients are effectively treated by the standard treatment care and that there may be serious adverse reactions among those that are not. This represents a very significant wasted resource in time and materials for healthcare providers under enormous pressure to reduce costs. Targeted medicines are designed to match effective treatments to patients but this promise can only be delivered if new therapies can be developed more quickly than currently and if they have greater cost effectiveness including lower manufacturing costs than existing medicines. The processes and decision-support tools developed in the Hub will enable product manufacturers to meet these challenges.
Patients
Faster development and more economic processes for targeted therapeutics will allow the promise of precision to be realised for the patient. They will enable patients to access promptly new stratified and personalised therapies, in some cases for diseases not currently treated and they will reduce ineffective treatments and adverse side effects.
UK Economy
The UK has a strong established pharmaceutical industry and a growing number of new companies developing new protein and cellular therapies. The development of new processes and tools to guide selection of processes and manufacturing option will enable this sector to commercialise successfully their rapidly increasing research effort in targeted medicines and increase the GVA of this part of the pharmaceutical industry.
HOW WILL THEY BENEFIT FROM THE RESEARCH
The Grand Challenge research in the hub will create new process technologies to address key manufacturing challenges identified following extensive discussion with the industry. Additionally the Hub with create new decision-support tools that will be used to accelerate the development and optimisation of manufacturing processes at appropriate scales. These tools will address the complexity of new clinical trial structures and increasingly decentralised manufacturing.
User-led feasibility projects with companies of the User Group, part of the Hub's platform research, will help test and evaluate these products. The Hub will have IP arrangements designed to achieve rapid exploitation and broad uptake and will involve three Catapults to facilitate translation of the technologies developed to market. 18 PDRAS, 28 CDT, 23 PhD and 14 EngD researchers on projects aligned to the hub will be trained with the process development skills required by industry. The Hub will engage with regulators to ensure they are briefed and informed on the emerging new technologies to allow appropriate regulatory strategies to be developed and put in place.
The successful commercialisation of new targeted biological medicines by companies will strengthen the UK manufacturing community with direct impacts on people employed and UK wealth generated.
Organisations
- University College London (Lead Research Organisation)
- Centre for Process Innovation (CPI) (Collaboration)
- Puridify (Collaboration)
- deltaDOT Ltd (Collaboration)
- University of Warwick (Collaboration)
- SSC Bio (Collaboration)
- Laboratory of the Government Chemist (LGC) Ltd (Collaboration)
- Lonza Group (Collaboration)
- Shanghai Pulmonary Hospital (Collaboration)
- Ipsen (Collaboration)
- Sutro Biopharma (Collaboration)
- Francis Biopharma (Collaboration)
- BIOINDUSTRY ASSOCIATION (Collaboration)
- UCB Pharma (Collaboration)
- PALL Europe (Collaboration)
- BioLogicB LLC (Collaboration)
- Cell and Gene Therapy Catapult (Collaboration)
- Cancer Research UK (Collaboration)
- Reneuron (Collaboration)
- Perceptive Engineering Ltd (Collaboration)
- Sartorius (Collaboration)
- Repligen (Collaboration)
- Eli Lilly & Company Ltd (Collaboration)
- Aglaris Ltd (Collaboration)
- Applikon Biotechnology B.V. (Collaboration)
- Autolus Limited (Collaboration)
- BIA Separations (Collaboration)
- Oxford BioMedica UK Ltd (Collaboration)
- Medicines Manufacturing Industry Partnership (Collaboration)
- Pharmaron (Collaboration)
- Cell Therapy Catapult (Collaboration)
- TrakCel (Collaboration)
- Redbud Labs (Collaboration)
- Allergan (Collaboration)
- Purolite International Ltd (Collaboration)
- Fujifilm (Japan) (Collaboration)
- AstraZeneca (Collaboration)
- United Kingdom Research and Innovation (Collaboration)
- Wyatt Technology Corporation (Collaboration)
- Orchard Therapeutics (Collaboration)
- Biopharm Services (Collaboration)
- LGC Ltd (Collaboration)
- INNOVATE UK (Collaboration)
- GlaxoSmithKline (GSK) (Collaboration)
- Cell Medica Ltd. (Collaboration)
- University College London (Collaboration)
- Autolus (Collaboration)
- 3M (United States) (Collaboration)
- West Pharmaceutical Services (Collaboration)
- National Institute for Biological Standards and Control (NIBSC) (Collaboration)
- Association of the British Pharmaceutical Industry (Collaboration)
- Ovizio Imaging Systems NV SA (Collaboration)
- IMPERIAL COLLEGE LONDON (Collaboration)
- Albumedix Ltd (Collaboration)
- Irvine Scientific (Collaboration)
- Univercells Ltd (Collaboration)
- Kuopio Center for Gene and Cell Therapy (Collaboration)
- CSL (Switzerland) (Collaboration)
- Adaptimmune (Collaboration)
- Advanced Bioprocess Services Ltd (Collaboration)
Publications
.Ogonah O
(2017)
Cell free protein synthesis: a viable option for stratified medicines manufacturing?
in Current Opinion in Chemical Engineering
Abdolvand N
(2019)
Long-Term Retinal Differentiation of Human Induced Pluripotent Stem Cells in a Continuously Perfused Microfluidic Culture Device.
in Biotechnology journal
Ahmed S
(2019)
New generation of bioreactors that advance extracellular matrix modelling and tissue engineering.
in Biotechnology letters
Amini A
(2020)
Bioprocess considerations for T-cell therapy: Investigating the impact of agitation, dissolved oxygen, and pH on T-cell expansion and differentiation.
in Biotechnology and bioengineering
Andersen C
(2021)
The Use of Platelet-Rich Plasma (PRP) for the Management of Non-union Fractures.
in Current osteoporosis reports
Aranda Hernandez J
(2022)
Microfluidic Devices as Process Development Tools for Cellular Therapy Manufacturing.
in Advances in biochemical engineering/biotechnology
Armstrong A
(2021)
Advanced control strategies for bioprocess chromatography: Challenges and opportunities for intensified processes and next generation products.
in Journal of chromatography. A
Atanasova G
(2023)
Fluid flow and mixing in a novel intermittently rotating bioreactor for CAR-T cell therapy manufacturing
in Chemical Engineering Science
| Description | The Hub has embarked on an ambitious set of strategic research targets, closely informed by its strong industry and regulatory consortium. Our focus is to develop and examine technological solutions for the rapid realization of targeted healthcare and include: pioneering studies in the utility of cell-free synthesis for just-in-time manufacture of valuable therapeutics; engineering characterization of novel bioreactor geometries for effective process development and delivery of cell and gene therapy products; as well as advanced software tools to help in decision-making for the industry. Over the past year, we have made significant advances in our scientific understanding and technology innovations, and we have working closely with our community of stakeholders to deliver user feasibility studies which are designed to test our methods, and to gauge the utility of new approaches in an industrial setting. 28 feasibility studies have been supported to date, involving individual partnerships with 21 companies and 5 academic centre departments. The results of these studies will be used to inform new research directions and to further our understanding of the evolving manufacturing landscape. This close integration with industry means that our progress makes a defined impact on manufacturing practice. Under Grand Challenge 1, novel manufacturing- and enterprise-level computational tools have been created to determine the technical, regulatory and supply chain innovation required for commercial feasibility of targeted medicines, whilst ensuring affordability. The tools span decision-making across process development, clinical manufacturing and market supply for stratified protein and personalised cell/gene therapy medicines. Under Grand Challenge 2, new technologies have been established with the potential for radical re-design of manufacture. Novel bioprocesses, analytics and control algorithms have been created that enable robust, safe and cost-effective manufacturing and formulation of stratified protein and personalised cell therapies. Together these provide the flexibility and speed to produce medicines for small patient populations or individuals in response to clinical diagnostic data. Research and Impact Highlights from GC1 include (a) Cell-free synthesis (CFS) economics: Case studies highlighted cost of goods differences and innovation required for CFS compared to traditional processes for antibody-drug conjugates (b) CAR T-cell process and supply chain economics: Decisional tools identified the key cost of goods (COG) drivers and the risk-reward trade-offs between centralised versus bedside manufacture (c) Drug development cost benchmarks: Established process development and manufacturing cost benchmarks for different attrition rates and their contribution to R&D costs for proteins and cell therapies. (d) CAR T-cell supply chain optimisation: Existing supply chains have been characterized and likely future supply chain workflows have been proposed. (e) Clinical trial scheduling under uncertainty: Developed a stochastic optimisation model that generates solutions consistently better than industry heuristics and other algorithms. (f)Regulation: Accelerated pathways for ATMPs in EU and US mapped out including post-marketing requirements and the unintended consequences on key stakeholders. (g) Reimbursement: NICE's approach to appraising CAR T-cell therapies (Yescarta® and Kymriah®) mapped out and analysed for its longer-term feasibility and budget impact. (h) Data analytics: Data mining tools have been developed for biomarker stratification to help clinicians make personalised decisions and for protein aggregation prediction. (i) Sustainability: Evaluation of the relative sustainability metrics for the manufacturing of different biological therapies. Research and Impact Highlights from GC2 include: (a)Co-formulation: Multiple case studies have established the impacts of protein co-formulation. Established core techniques as a basis for future digital formulation capability (b) Nanoencapsulation: Scalable nanoparticle manufacturing to encapsulate co-formulated proteins and fluorophores, then deliver to cells (c) Analytics: New instrument developed to monitor the degradation of proteins in mixtures. A second highly sensitive viral lasing technique also developed (d) Cell-free synthesis: Scalable in-house processes for protein and virus-like particle synthesis. Hub Specialist Working Group consultation defined objectives for Phase II, and roadmap for CFS strategy (e) CAR T-cell manufacturing and process intensification: 1st successful demonstration of CAR-T cell manufacturing in stirred-tank reactors. Intensified to reduce process time through a novel process control strategy. Demonstrated equivalent CAR-T yield with 45% reduction in process time (f) CAR T-cell predictive models: Optimal supply of primary nutrient and medium volume exchange for cell culture can be predicted by model (g) T-cell analytics: Digital Holography Microscopy provides rapid analytics to discern activated from non-activated T-cells. Microfluidic devices for personalised health manufacturing (h) Adaptive control algorithms developed: Benchmark simulator designed to test new control techniques. (i) Vector design: Novel AAV vector designs with improved manufacturability. |
| Exploitation Route | User-led feasibility studies, Spinouts, open source software |
| Sectors | Healthcare Manufacturing including Industrial Biotechology Pharmaceuticals and Medical Biotechnology |
| Description | The Hub has leveraged the expertise of our team to help set the agenda and solve key challenges in implementation of personalised medicines in the UK, through appointments on various policy committees, including the NHS Accelerated Access committee for data infrastructure. The Hub team have also provided bioprocessing insights to decision makers throughout the COVID-19 pandemic, including at the Chief Scientific Adviser's 100 Day Mission Roundtable. Public dissemination and debate of key issues, and ethics surrounding personalized medicines. Novel decisional tools have been created and applied to identify the most cost-effective manufacturing platforms, production plans and supply chain configurations for stratified protein medicines and personalised cell and gene therapies. This has helped the sector identify manufacturing and supply chain strategies that can offer cost savings for improved business performance, identify innovations and improvements required in order to have feasible business models, prioritise resource allocation for drug development and manufacturing activities and influence shifts in investment decision to focus on the most promising concepts. The long term feasibility of current regulatory and reimbursement strategies for personalised cell and gene therapies has been assessed and likely models for the future have been proposed to widen access to these therapies targeting unmet needs. Cell free synthesis has been evaluated both technically and also through cost modelling and key partner engagement, to identify current product types that would benefit, as well as areas for further innovation. Novel analytical technologies for protein formulations have been developed, patented and are being commercialised via a spin-out. In addition, it is now being explored in partnership with Pall Europe specifically for bioprocess monitoring. The option of stable coformulated proteins has been demonstrated, including using nanoparticles that target multiple proteins for delivery to cells, which paves the way for combination therapies and improves the chances of finding an acceptable formulation for each new product in industry. T-cell production in automated stirred-tank bioreactor systems will now enable a lower cost of goods for their manufacture. Novel imaging approaches have been demonstrated, including on micro fluidic platforms, for monitoring CAR-T cell quality in manufacturing. Meanwhile control strategies have enabled faster processes, which will shorten the time for delivery of personalised medicines to patients. |
| First Year Of Impact | 2018 |
| Sector | Digital/Communication/Information Technologies (including Software),Healthcare,Manufacturing, including Industrial Biotechology,Pharmaceuticals and Medical Biotechnology |
| Impact Types | Societal Policy & public services |
| Description | Advsory Board for reviewww of Defence Academy |
| Geographic Reach | National |
| Policy Influence Type | Contribution to a national consultation/review |
| Description | Attendance of EPSRC Centre for Innovative Manufacturing in Food, Advisory Board |
| Geographic Reach | National |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Description | BSI Commitee member |
| Geographic Reach | National |
| Policy Influence Type | Membership of a guideline committee |
| Description | CCMI Scale-Up and Challenges in Manufacturing of ATMP workshop |
| Geographic Reach | Europe |
| Policy Influence Type | Influenced training of practitioners or researchers |
| Description | Committee Member, Institution of Chemical Engineers Biochemical Engineering Special Interest Group |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Impact | The IChemE BESIG committee focuses on addressing and representing the Biochemical Engineering interests for the Institution of Chemical Engineers. As a member on the committee, I represent the cell and gene therapy activity which aligns with the Hub. As part of the committee, we have contributed to the Institution's BioFutures agenda which has been forwarded to government. This calls for action to boost the skills diversity of biochemical engineering graduates to prepare them for the greater diversity of roles requiring biochemical engineers. |
| Description | Contribution to All-Party Parliamentary Group on the Fourth Industrial Revolution booklet on "The Future of Manufacturing" |
| Geographic Reach | National |
| Policy Influence Type | Implementation circular/rapid advice/letter to e.g. Ministry of Health |
| URL | https://537f9414-4137-40f8-86c9-93b073010631.filesusr.com/ugd/c018a2_74d0f3563ba841a98afc70f26fdebf3... |
| Description | ESACT-UK Scientific Programme Lead |
| Geographic Reach | Europe |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Impact | ESACT-UK is the UK Society for Cell Culture Biotechnology. Dr Qasim Rafiq leads up the Scientific Programme for the society. The society promotes the communication of knowledge and experience between scientists and engineers whose work has a direct or indirect bearing on cell culture biotechnology and the products derived from it. A particular emphasis is to ensure both the inclusion, and the active participation, of less experienced scientists in industry or academic institutions. ESACT-UK actively encourages interactions between academia, governmental and policy making agencies, and the manufacturing and service industries. One product of such interactions, which involved members of ESACT-UK, is the international Good Cell Culture Practice (GCCP) Guidance Document. |
| Description | Elected Management Board Member of BBSRC BioProNet |
| Geographic Reach | National |
| Policy Influence Type | Membership of a guideline committee |
| Impact | BioProNet coordinates the entire UK academic and industrial community for Bioprocess Manufacturing, via network meetings, workshops and managed proof of concept funding. Research outputs feed into larger InnovateUK proposals. |
| Description | Feedback to the MHRA regarding the regulatory framework proposal on point-of-care manufacture |
| Geographic Reach | National |
| Policy Influence Type | Implementation circular/rapid advice/letter to e.g. Ministry of Health |
| Impact | This feedback contributed towards reinforcing the MHRA's regulatory proposal, making the agency refine, for example, its understanding of the scope and modalities of point-of-care manufacture. |
| Description | Health and Social Care Committee inquiry on Future Cancer |
| Geographic Reach | National |
| Policy Influence Type | Contribution to a national consultation/review |
| Description | House of Commons Foreign Affairs Committee inquiry on Global health security |
| Geographic Reach | National |
| Policy Influence Type | Contribution to a national consultation/review |
| URL | https://committees.parliament.uk/writtenevidence/18365/pdf/ |
| Description | House of Commons Science & Technology Inquiry on COVID-19 Lessons Learned |
| Geographic Reach | Europe |
| Policy Influence Type | Contribution to a national consultation/review |
| URL | https://committees.parliament.uk/writtenevidence/14934/pdf/ |
| Description | Hub User Feasibility Study fed into MMIP business case on "A UK Strategy for the Manufacture of GMP Viral Vectors" |
| Geographic Reach | National |
| Policy Influence Type | Contribution to a national consultation/review |
| Impact | Hub User Feasibility Study fed into MMIP business case on "A UK Strategy for the Manufacture of GMP Viral Vectors". The models predicted that switching to a scalable process at any stage of clinical trials was more cost-effective, in terms of both the total cost of development and profitability, than sticking with a non-scalable process throughout development and commercialisation. This has been used in the case for suitable viral vector capacity and capability building in the UK. |
| URL | https://www.bioindustry.org/uploads/assets/uploaded/1d1066a9-be76-44a9-b9458c9ce8c1b040.pdf |
| Description | Member, BIA (BioIndustry Association) Cell and Gene Therapy Advisory Committee (biannual) |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Impact | The Committee is linked to implementing recommendations from the MMIP Advanced Therapies Taskforce e.g. to encourage manufacturing of cell and gene therapies to stay in the UK. |
| Description | Member, BIA (BioIndustry Association) Manufacturing Advisory Committee for Bioprocessing Industries (2009 - present) |
| Geographic Reach | National |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Impact | A skills initiative was established with the 1st cohort 16-17 and the 2nd cohort 17-18. This aims to build a network of next generation bioprocess leaders, giving them an opportunity to visit each other's facilities for a day to learn about manufacturing of different medicine types. The programme to take place over 12 months. All companies and organisations taking part host a visit. |
| Description | Member, BioIndustry Association (BIA) Cell and Gene Therapy Advisory Commitee |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Impact | The BIA CGTAC is a nationally leading committee in the cell and gene therapy field which has significant national and international influence. It has been involved in implementing the MMIP Advanced Therapies Taskforce and within the committee we have developed a series of four explainer booklets and five videos that highlight and celebrate the the work taking place in UK bioscience companies to create innovative new drugs and treatments for patients. This includes a Guide to Cell and Gene Therapy and UK Excellence in the Field aimed at policy makers and the public, which was published as part of this series. |
| Description | Member, ISCT (International Society for Cellular Therapy) Business Models and Investment Subcommittee |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Impact | Cell therapy products are frequently developed and produced without incorporating cost considerations into process development, contributing to prohibitively costly products. Herein we contextualize individual process development decisions within a broad framework for cost efficient therapeutic manufacturing. This roadmap guides the analysis of Cost of Goods (COG) arising from tissue procurement, material acquisition, facility operation, production, and storage. Planning and accounting for COG at each step in the production process could reduce costs, allowing for more affordable market pricing to improve the long term viability of the cell therapy product and facilitate broader patient access to novel and transformative cell therapies. |
| Description | Membership of a guideline committee - Elected Management Board Member of BBSRC BioProNet (2015) |
| Geographic Reach | National |
| Policy Influence Type | Membership of a guideline committee |
| Impact | BioProNet is key to advising and shaping the funding opportunities between BBSRC and EPSRC |
| Description | NHS England The Innovative Medicines Fund: engagement on proposals |
| Geographic Reach | National |
| Policy Influence Type | Contribution to a national consultation/review |
| Impact | The impact of the feedback is yet to be established. However, the overall impact will improve healthcare services, patient access and quality of life and direct NHS England to make in data infrastructure, suitable for manufacturers. |
| Description | National Institute for Health and Care Excellence, The NICE methods of health technology evaluation: the case for change, Consultation: 6 November - 18 December 2020 |
| Geographic Reach | National |
| Policy Influence Type | Contribution to a national consultation/review |
| URL | https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guid... |
| Description | Response to EMA public consultation on strategy "Regulatory Science to 2025" |
| Geographic Reach | Europe |
| Policy Influence Type | Contribution to a national consultation/review |
| Impact | Hub participation in an EMA public consultation helped to shape the EMA's regulatory science strategy enabling them to decide where the future priorities and resources should be attributed. As part of the consultation, respondents were asked to select three top priorities which the EMA should consider in their strategy. Of the 3 priorities recommended by the Hub, 2 were considered in greater detail during a follow-up workshop to discuss the findings of the consultation. Under the EMA strategic goal to "Develop network-led partnerships with academia to undertake fundamental research in strategic areas of regulatory science" the Hub was cited as an example of an academic research centre with which the EMA should be collaborating (see: https://www.ema.europa.eu/en/documents/presentation/presentation-ema-regulatory-science-2025-develop-network-led-partnerships-academia-undertake_en.pdf) |
| URL | https://www.ema.europa.eu/en/about-us/how-we-work/regulatory-science-2025 |
| Description | Response to EMA public consultation on strategy "Regulatory Science to 2025" (2019) - Reimbursement Perspective |
| Geographic Reach | Europe |
| Policy Influence Type | Contribution to a national consultation/review |
| Impact | Hub participation in an EMA public consultation helped to shape the EMA's regulatory science strategy enabling them to decide where the future priorities and resources should be attributed. Key points highlighted from a reimbursement perspective: HTAs' preparedness and downstream decision-making for innovative medicines. Cost-effectiveness of emerging cell and gene therapies is based on evidence from clinical effectiveness studies, preferences regarding health outcomes and the cost of technologies. Uncertainty in the available evidence effects the estimates of incremental costs, health benefits and the decisions made about implementation of these therapies. Potential solutions to addressing uncertainty have been managed entry agreements (MEAs), which ensure the risks and benefits of technologies are shared between the payers and manufacturers. In addition to MEAs, we recommend the introduction of EU clinical registries post-launch, with input from the EMA and HTA on data requirements. Clinical registries would provide highly structured clinical data to healthcare professionals on safety and effectiveness, and can be used to compare the effectiveness of different treatments for the same disease or condition. However, we recognise that registries would require significant investment in registry design, operating data systems, training and licencing. The cost of running the registries should be factored in HTA evaluations, with further discussions on the distribution costs between the payer and manufacturers. A distinct feature of clinical trials for cell and gene therapies are that they have tended to be small, single-arm, or single-centre, early phase clinical trials. In the context of Europe's publicly-funded healthcare systems, it is evident that they are not geared to make one-off, large up-front payments for therapies with uncertainty. Amortisation and payment by performance models have emerged to as a means to financing high-cost technologies. We suggest, HTA bodies stipulate a resource impact assessment applying the annuity and payment by performance models. This criteria would serve as a tool to predict future expenditure and identify the best reimbursement model early on. Finally, we expect HTA bodies to continue authorising future cell and gene therapies with conditional approval, in which case it is important to understand how the HTA bodies plan on addressing the decommissioning of failed therapies. We suggest, the EMA and HTA bodies evaluate the safety and efficacy of the therapies in consultation with one another, to create a culture where they work in parallel to meet their objectives. This will avoid duplication of data review and expedite the pathway to commercialisation. |
| URL | https://www.ema.europa.eu/en/about-us/how-we-work/regulatory-science-2025 |
| Description | Response to the UK Research and Development Roadmap consultation |
| Geographic Reach | National |
| Policy Influence Type | Contribution to a national consultation/review |
| URL | https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/9543... |
| Description | UCL Cell and Gene Therapy MBI Course |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Influenced training of practitioners or researchers |
| Impact | The UCL Cell and Gene Therapy MBI course was designed to align with the Hub activity in mind to address the arising skills gap within the advanced therapy sector. The course is specifically aimed at industrial delegates to provide them with the necessary depth and breadth to address the manufacturing and commercialisation issues associated with these advanced therapies. Over 30 industrial delegates were present for the 3 day course representing large pharmaceutical companies (e.g. GSK), SMEs (e.g. ReNeuron) and also regulatory agencies and representatives from multiple countries including Hong Kong, Qatar and Brunei. |
| Description | Advanced Bioprocess 2 |
| Amount | £100,973 (GBP) |
| Organisation | Advanced Bioprocess Services Ltd |
| Sector | Private |
| Country | United Kingdom |
| Start | 01/2018 |
| End | 12/2018 |
| Description | Advanced Bioprocess 3 |
| Amount | £41,333 (GBP) |
| Organisation | Advanced Bioprocess Services Ltd |
| Sector | Private |
| Country | United Kingdom |
| Start | 01/2019 |
| End | 12/2019 |
| Description | Advanced Co-Formulation of Targeted Biologic Therapeutics |
| Amount | £2,000 (GBP) |
| Organisation | University of Nottingham |
| Sector | Academic/University |
| Country | United Kingdom |
| Start | 07/2018 |
| End | 07/2019 |
| Description | Artificial Intelligence-driven, Decentralized Production for Advanced Therapies in the Hospital (AIDPATH) |
| Amount | € 9,860,991 (EUR) |
| Funding ID | 101016909 |
| Organisation | European Commission |
| Sector | Public |
| Country | European Union (EU) |
| Start | 01/2021 |
| End | 12/2024 |
| Description | Autolus EngD Studentship |
| Amount | £54,000 (GBP) |
| Organisation | Autolus Limited |
| Sector | Private |
| Country | United Kingdom |
| Start | 09/2018 |
| End | 09/2022 |
| Description | CONCORD: CO-ordiNated Care Of Rare Diseases |
| Amount | £732,217 (GBP) |
| Funding ID | HS&DR/16/116/82 |
| Organisation | National Institute for Health Research |
| Sector | Public |
| Country | United Kingdom |
| Start | 05/2018 |
| End | 11/2020 |
| Description | Cell Therapy Manufacturing |
| Amount | £192,717 (GBP) |
| Organisation | Advanced Bioprocess Services Ltd |
| Sector | Private |
| Country | United Kingdom |
| Start | 01/2020 |
| End | 12/2025 |
| Description | Centre of Excellence |
| Amount | £1,393,742 (GBP) |
| Organisation | PALL Europe |
| Sector | Private |
| Country | United Kingdom |
| Start | 01/2018 |
| End | 12/2024 |
| Description | Continuous Downstream Processing of Bioproducts (CODOBIO), H2020 Marie Sklodowska-Curie Innovative Training Network Grant |
| Amount | € 3,991,008 (EUR) |
| Funding ID | 812909 |
| Organisation | European Commission H2020 |
| Sector | Public |
| Country | Belgium |
| Start | 02/2019 |
| End | 02/2023 |
| Description | Development of a stability and preservation strategy for NKT cells - Collaborate to Innovate round 2: Advanced Therapies |
| Amount | £99,998 (GBP) |
| Organisation | MedCity |
| Sector | Public |
| Country | United Kingdom |
| Start | 08/2019 |
| End | 08/2020 |
| Description | EPSRC Impact Acceleration Account (IAA) |
| Amount | £2,000 (GBP) |
| Funding ID | EP/R511638/1 |
| Organisation | Engineering and Physical Sciences Research Council (EPSRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 09/2019 |
| End | 02/2020 |
| Description | EPSRC Impact Acceleration: Establish cost of goods model for bioartificial liver machine commercialisation |
| Amount | £29,000 (GBP) |
| Organisation | Engineering and Physical Sciences Research Council (EPSRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 05/2019 |
| End | 12/2019 |
| Description | GCRF establishment of biopharmaceutical and animal vaccine production capacity in Thailand and neighbouring South East Asian countries |
| Amount | £4,090,259 (GBP) |
| Funding ID | BB/P02789X/1 |
| Organisation | Biotechnology and Biological Sciences Research Council (BBSRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 09/2017 |
| End | 12/2022 |
| Description | H&LS grant with Plasticell and UCL ICH on: Development of advanced technologies for gene therapy manufacture |
| Amount | £742,000 (GBP) |
| Funding ID | 6667 |
| Organisation | Innovate UK |
| Sector | Public |
| Country | United Kingdom |
| Start | 05/2018 |
| End | 01/2021 |
| Description | H2020 Marie Sklodowska-Curie Innovative Training Network Grant |
| Amount | £3,991,008 (GBP) |
| Funding ID | 812909 |
| Organisation | Marie Sklodowska-Curie Actions |
| Sector | Charity/Non Profit |
| Country | Global |
| Start | 02/2019 |
| End | 02/2023 |
| Description | High-throughput directed evolution to engineer thermostable therapeutic proteins |
| Amount | € 195,454 (EUR) |
| Funding ID | 795539 |
| Organisation | Marie Sklodowska-Curie Actions |
| Sector | Charity/Non Profit |
| Country | Global |
| Start | 06/2018 |
| End | 07/2020 |
| Description | Industrial Fellowship - Carlos Julia - Aglaris |
| Amount | £110,672 (GBP) |
| Organisation | Royal Commission for the Exhibition of 1851 |
| Sector | Charity/Non Profit |
| Country | United Kingdom |
| Start | 09/2018 |
| End | 09/2021 |
| Description | Industrial Fellowship - Shaun Mansfield Biovault Technical |
| Amount | £109,490 (GBP) |
| Organisation | Royal Commission for the Exhibition of 1851 |
| Sector | Charity/Non Profit |
| Country | United Kingdom |
| Start | 09/2017 |
| End | 09/2020 |
| Description | Innovate UK |
| Amount | £952,551 (GBP) |
| Funding ID | 6667 |
| Organisation | Innovate UK |
| Sector | Public |
| Country | United Kingdom |
| Start | 05/2018 |
| End | 05/2020 |
| Description | InnovateUK Collaborative R&D |
| Amount | £545,024 (GBP) |
| Funding ID | 104377 |
| Organisation | Innovate UK |
| Sector | Public |
| Country | United Kingdom |
| Start | 06/2018 |
| End | 09/2019 |
| Description | InnovateUK KTP between SureScreen Limited & University of Nottingham: Implementing technologies for manufacture of personalised nutritional supplements |
| Amount | £266,910 (GBP) |
| Funding ID | KTP011734 |
| Organisation | Innovate UK |
| Sector | Public |
| Country | United Kingdom |
| Start | 11/2019 |
| End | 11/2022 |
| Description | InnovateUK KTP with Aber Instruments - Development of a Biocapacitance Probe for Real-Time, Online ATMP Analytics |
| Amount | £232,720 (GBP) |
| Funding ID | Ref No: KTP 12049 |
| Organisation | Innovate UK |
| Sector | Public |
| Country | United Kingdom |
| Start | 05/2020 |
| End | 05/2022 |
| Description | InnovateUK KTP with Biovault Technical |
| Amount | £307,617 (GBP) |
| Funding ID | KTP011128 |
| Organisation | Innovate UK |
| Sector | Public |
| Country | United Kingdom |
| Start | 03/2019 |
| End | 04/2022 |
| Description | Knowledge Transfer Partnership |
| Amount | £286,705 (GBP) |
| Funding ID | KTP011678 |
| Organisation | Innovate UK |
| Sector | Public |
| Country | United Kingdom |
| Start | |
| Description | New Industrial Systems: Optimising Me Manufacturing Systems |
| Amount | £1,520,745 (GBP) |
| Funding ID | EP/R022534/1 |
| Organisation | Engineering and Physical Sciences Research Council (EPSRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 02/2018 |
| End | 09/2020 |
| Description | Real World Handling of Protein Drugs - Exploration, Evaluation and Education |
| Amount | € 3,139,983 (EUR) |
| Funding ID | 101007939 - RealHOPE |
| Organisation | European Commission |
| Sector | Public |
| Country | European Union (EU) |
| Start | 06/2021 |
| End | 07/2025 |
| Description | Responsive Mode Manufacturing the Future |
| Amount | £406,203 (GBP) |
| Funding ID | EP/R031649/1 |
| Organisation | Engineering and Physical Sciences Research Council (EPSRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 12/2018 |
| End | 11/2021 |
| Description | Royal Commission Industrial Studentship: Development of experimentally-integrated process models for adaptive CAR-T cell therapy manufacturing |
| Amount | £94,000 (GBP) |
| Organisation | Royal Commission for the Exhibition of 1851 |
| Sector | Charity/Non Profit |
| Country | United Kingdom |
| Start | 09/2019 |
| End | 09/2022 |
| Description | Sartorius EngD Studentship |
| Amount | £50,000 (GBP) |
| Organisation | Sartorius |
| Department | Sartorius Stedim Biotech |
| Sector | Private |
| Country | France |
| Start | 09/2017 |
| End | 09/2020 |
| Description | The Effect of Feed Quality Due to Clarification Strategy on the Design and Performance of Protein A Periodic Counter-Current Chromatography |
| Amount | £95,716 (GBP) |
| Organisation | 3M |
| Sector | Private |
| Country | United States |
| Start | 04/2017 |
| End | 11/2017 |
| Description | The Future Vaccine Manufacturing Research Hub (Vax-Hub) |
| Amount | £10,031,142 (GBP) |
| Funding ID | EP/R013756/1 |
| Organisation | Engineering and Physical Sciences Research Council (EPSRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 03/2018 |
| End | 03/2023 |
| Description | The Pluripotent Stem Cells and Engineered Cell (PSEC) Hub |
| Amount | £4,214,221 (GBP) |
| Funding ID | MR/R015724/1 |
| Organisation | Medical Research Council (MRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 05/2018 |
| End | 05/2024 |
| Description | UCL'S EPSRC IAA DISCOVERY-TO-USE |
| Amount | £41,584 (GBP) |
| Organisation | University College London |
| Sector | Academic/University |
| Country | United Kingdom |
| Start | 05/2021 |
| End | 03/2022 |
| Description | UCL-MedImmune Centre of Excellence |
| Amount | £667,000 (GBP) |
| Organisation | AstraZeneca |
| Department | MedImmune |
| Sector | Private |
| Country | United Kingdom |
| Start | 08/2017 |
| End | 12/2020 |
| Description | UKRI Future Leaders Fellowship |
| Amount | £1,167,524 (GBP) |
| Organisation | Medical Research Council (MRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 11/2020 |
| End | 11/2024 |
| Description | Unitive Design Analysis EngD Studentship |
| Amount | £40,000 (GBP) |
| Organisation | Unitive Design and Analysis, Ltd. |
| Sector | Private |
| Country | United Kingdom |
| Start | 09/2018 |
| End | 09/2022 |
| Description | University College London: - UCL'S EPSRC IAA DISCOVERY-TO-USE - Design and prototyping of a cell-free (enzymatic) reactor for plasmid DNA manufacturing in clinical applications |
| Amount | £30,000 (GBP) |
| Funding ID | 571023-100-156780 |
| Organisation | Engineering and Physical Sciences Research Council (EPSRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 03/2022 |
| End | 09/2022 |
| Description | University College London: - UCL'S MRC/EPSRC TAS - In vivo demonstration of platform pDNA production and delivery technologies for the treatment of rare genetic diseases |
| Amount | £127,651 (GBP) |
| Organisation | Medical Research Council (MRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 01/2023 |
| End | 12/2023 |
| Description | Viral Lasers for Biological Detection |
| Amount | € 100,000 (EUR) |
| Organisation | European Commission |
| Sector | Public |
| Country | European Union (EU) |
| Start | 09/2019 |
| End | 03/2020 |
| Description | Viral lasers for biological detection. |
| Amount | £60,000 (GBP) |
| Organisation | Royal Society of Edinburgh (RSE) |
| Sector | Charity/Non Profit |
| Country | United Kingdom |
| Start | 01/2020 |
| End | 01/2021 |
| Title | A fluorescence-based non-separation chromatography tool for the analytics of coformulated protein medicine |
| Description | A fluorescence life-time spectroscopy associated with chromatrography system. Proteins, aggregates or fragments that are co-eluted can be resolved based on their respective fluorescence life time. |
| Type Of Material | Technology assay or reagent |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | Two key experiments were conducted to validate the system in the bioprocessing of protein medicine: 1) A coformulation of a full-size mAb and a fragment Fab, which were different in size but the degradation products during stress experiment were overlapping on the chromatogram. 2) A coformulation of two full-size mAbs, which have similar size, were co-eluted. The result showed this fluorescence tool can resolve different species in coformulated protein therapeutics, even when they, or their degradation product, were co-eluted in chromatographic separation. This paves the way to future application of this tool to the inline analytics of compound biopharmaceutical modalities. |
| Title | An ODE Model for Optimal Operation of T Cell Culture in Suspension Systems |
| Description | Application of ODE modelling framework to provide better understanding of T-cell manufacturing process optimisation and the risk involved. The modelling approach can be applied, with very limited process data, to define medium exchange process operation limits in a stirred tank culture format for example Ambr 15 bioreactor. This approach can be evolved to provide further process insight with nutrient concentrations and an optimized cytokine provision in T cell culture. |
| Type Of Material | Technology assay or reagent |
| Year Produced | 2020 |
| Provided To Others? | No |
| Impact | 1.A dynamic model was built to link efficient feed scenarios to population selective effects and product quality and to Integrate the use of statistical model based population detection to identify the most efficient method for achieving product quality and intensification goals. 2.Employing the ODE model identified secondary inhibitory factors in T cell culture system that can be used in further improvement of system intensification. |
| Title | Cell-free pDNA production |
| Description | Through UFS with CRUK techniques for producing pDNA enzymatically (i.e. cell-free) have been developed and continue to be developed. The method details are available to CRUK, after further development and data collection it is intended to publish finalised methods. A description of this protocol has been submitted to UCLB to be considered for a patent application. It is also the subject of a funding application for development into an automated production device and for an EngD project. |
| Type Of Material | Technology assay or reagent |
| Year Produced | 2020 |
| Provided To Others? | Yes |
| Impact | The development process has allowed CRUK BDU to begin developing their in-house capabilities to analyse pDNA. Further impact and publications anticipated. |
| Title | PBMC from healthy donor to isolate T cells |
| Description | Isolated T cells were used in optimisation of automated expansion of T cells in Ambr 15 bioreactor to regenerate data for our ODE model. |
| Type Of Material | Cell line |
| Year Produced | 2018 |
| Provided To Others? | No |
| Impact | A manuscript on "Application of a Simple Unstructured Kinetic Model to Support T-Cell Therapy Manufacture" that is in its final stage and ready to be submitted. |
| Title | Transient transfection of 293F Suspension producer cell line optimisation in house. rAAV purification by iodixanol gradient and caracterisation of the final product by ddPCR in progress. |
| Description | - Transient transfection of 293F-cells suspension pcell line optimisation with the aim of selecting enginnered rAAV producer cell line stably transfected clone of 293F cells. The purpose of this cell culture and engineering strategy is the avoid the cost of repetitive transient trasfection method, to increase the yield of rAAV productyion by scaling up the cell expansion in a bioreactor. - rAAV purification by iodixanol gradient, a rapid method of rAAV purification - ddPCR, a potent tool of |
| Type Of Material | Technology assay or reagent |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | Utilise a design-of-experiment (DOE) methodology to optimize rAAV production in a 293F suspension cell system. Develop a DOE-optimized protocol, to possibly achieve unpurified yields approaching 3 × 1014 viral genomes (VGs)/L of cell culture. The impact of this DOE-driven approach would allow one to evaluate the impact of multiple interdependent factors on a given output. Scale up and reduce the cost of rAAV production. |
| Title | development of UCL CFPS platform |
| Description | UCL developed their CFPS platform including reaction mix and method for the scalable production of high specific activity extract. This work was conducted by Noelle Colant and Dr Olotu Ogonah. |
| Type Of Material | Technology assay or reagent |
| Year Produced | 2020 |
| Provided To Others? | Yes |
| Impact | A feasibility study by Noelle Colant with Fujifilm Diosynth Biosciences has looked at using the UCL platform for their plasmids. The sensitivity of the CFPS react to the plasmid purification process was identified. *Soon to be submitted for publication by Noelle Colant |
| URL | https://pubmed.ncbi.nlm.nih.gov/32761750/ |
| Title | DNA sequences encoding mammalian expression of putative transient transfection-sensor (Trensor) genes |
| Description | Sequence data evidencing synthesis and assembly of three plasmids encoding genes intended to be induced when cells undergo transient transfection or are exposed to small molecules that enhance transient transfection performance. Please open the file: "READ ME Trensor Seq 170723.pdf" for an explanation of the plasmid design and assembly, and description of each of the sequencing files. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2023 |
| Provided To Others? | Yes |
| URL | https://figshare.com/articles/dataset/_strong_DNA_sequences_encoding_mammalian_expression_of_putativ... |
| Title | A decision making tool for CDx clinical trial strategies selection |
| Description | Computational models for CDx clinical Trial and manufacturing process which can explore the impact of different CDx clinical trial strategies on potential market size, process development cost and future profitable prediction |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | The computational models can be applied for decision making support on CDx clinical trial strategy selection |
| Title | A mechanistic model of erythroblast growth inhibition providing a framework for optimisation of cell therapy manufacturing |
| Description | Manufacture of Red Blood Cell based products in vitro requires highly efficient erythroblast culture for economic viability. It has previously been shown that efficiency of erythroblast culture in scalable bioreactors is not primarily limited by mass transfer, availability of medium components, or commonly recognised inhibitory metabolites or cytokines. We have developed a dynamic mechanistic model that describes an autocrine feedback loop in which a cell-derived factor accumulates in culture medium resulting in reversible erythroblast growth inhibition. Cells exhibited two phases of growth: a relatively uninhibited followed by an inhibited phase. Cell cycle analysis during inhibition identified slight accumulation of cells in S phase, distinct from the G1 accumulation anticipated in growth factor or nutrient deprivation. Substantial donor to donor growth rate variability (mean 0.047 h-1, standard deviation 0.008 h-1) required the growth rate parameter to be refitted for different donors. The model could then be used to predict growth behaviour with full medium exchange, but showed some reduced predictive ability after partial medium exchange. The model could predict the growth inflexion point over a range of phenotypic maturities from early to late maturity erythroblasts; however the secondary phase of growth differed substantially with less inhibition observed in more mature cells. The model provided a framework to optimise culture economics based on cost of production time and input consumables. It also provided a framework to evaluate the benefits of biological process engineering in medium design or cell modification vs. operational optimisation depending on the specific cost scenario of a process developer. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2018 |
| Provided To Others? | Yes |
| Impact | We have previously reported a modelling approach developed to provide a framework for optimisation of cell therapy manufacturing.This is an ordinary differential equation-based approach to describing key process mechanisms in a low parameter (and generally unstructured) form that directly relate to process operation. We are investigating the application of this framework to rapidly provide insight to T-cell manufacturing process optimisation and how the approach can be applied, with very limited process data, to define medium exchange process operation limits in a stirred tank culture format. |
| Title | Activity and Stability data 321 EKL variants |
| Description | 321 directed evolution derived bovine enterokinase variants. Measured in clarified lysates for activity and retained activity after a heat shock (stability). Measured from cells grown. at both 30C and 37C. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2022 |
| Provided To Others? | Yes |
| URL | https://rdr.ucl.ac.uk/articles/dataset/Activity_and_Stability_data_321_EKL_variants/20477127 |
| Title | Cash-flow Engine for Biopharmaceuticals and Cell &Gene Therapies |
| Description | A cash-flow model was created in an object-oriented environment using Python. The model is an evolution of the previous drug development spreadsheet model. The model is capable of simulating any research, development and commercialisation pathway in order to evaluate the potential cost and value of any drug/therapy over its entire lifecycle. |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2020 |
| Provided To Others? | No |
| Impact | The model has been used to perform the analysis for the user feasibility study signed between UCL and the BioIndustry Association. The key take home message from the analysis was that for in-vivo lenti- and adeno-associated viral vector therapies, the introduction of a scalable manufacturing process at very early stage in the development pathway is the most profitable approach. |
| Title | Cell-free synthesis Micro-24 Microbioreactor |
| Description | Several data-sets of 19-24 observations (repeats) each with varying process parameters such as length of reaction, pH, Temperature and DO. They contain final titre and aggregation % measurements and on-line pH, Temperature and DO measurements. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | The data-sets were useful for analyses which involved process characterization, optimization, and comparability between experiments |
| URL | https://github.com/CarlosADuranVIllalobos/CFPS-Multivariate-Statistical-Data-Analysis |
| Title | Cost of development model |
| Description | A cash-flow spreadsheet model capturing different costs occurring during research, development, regulatory approval and commercialisation of a new therapeutic drug. |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | The model offer the ability to estimate the expected net present value of different manufacturing and business strategies and identify the optimum. |
| Title | Develop a decision making tool for personalised lung cancer recurrence and survivability prediction using Matlab |
| Description | This model can integrate the genomic, clinical and demographic data of 511 lung adenocarcinoma patients and 487 lung squamous-cell carcinoma patients has been collected from The Cancer Genome Atlas. For 15 selected oncology genes, copy number variation as complement of mutation information was added in this work. Comparing the accuracy and benefits of three advanced machine-learning algorithms: decision tree methods, neural networks and vector support machines, the tree models reveal the most important predictors among genomic information (e.g. KRAS, EGFR, TP53...), clinical status (e.g. TMN stage and radiotherapy) and demographics (e.g. age and gender) and how they influence the prediction of recurrence and survivability for both early stage LUAD and LUSC. |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | The models reveal the most important predictors among genomic information (e.g. KRAS, EGFR, TP53...), clinical status (e.g. TMN stage and radiotherapy) and demographics (e.g. age and gender) and how they influence the prediction of recurrence and survivability for both early stage LUAD and LUSC. The machine learning models have the potential to help clinicians to make personalised decisions on aspects such as follow-up timeline and to assist with personalised planning of future social and care needs |
| Title | Develop a predictive tool for rapid therapeutic protein thermal stability analysis using Matlab |
| Description | Predictive model using Neural Network to predict protein aggregation levels at early stage. |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | The tool reveals that the temperature-dependence of native fluorescence spectra contains a high-degree of previously hidden information relating to protein dynamics and stability. This could lead to rapid screening of therapeutic protein variants and formulations based on spectroscopic measurements under non-denaturing temperatures only. |
| Title | Machine learning application in personalised lung cancer recurrence and survivability prediction |
| Description | With the rise of personalised and precision medicine, there is a growing trend towards machine learning applications for prognosis prediction. Building reliable prediction models of cancer outcomes in everyday clinical practice is still a hurdle. This paper aims to integrate genomic, clinical, diagnostic and demographic data to generate a full picture of patients in order to develop a risk prediction model for the overall survival and recurrence status for non-small cell lung cancer. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2019 |
| Provided To Others? | Yes |
| Impact | Exploring The Cancer Genome Atlas (TCGA) for biomarker collection for non-small cell lung cancer (NSCLC) and integration the genomic, clinical, diagnostic and demographic data to generate a full picture of patients in order to develop a risk prediction model for the overall survival and recurrence status for non-small cell lung cancer. |
| Title | Mixed Integer Linear Programming model for supply chain optimisation of CAR T cell therapies |
| Description | Two functions are selected as candidate minimisation objectives; namely the total cost and the average network response time, reflecting the urgency of the treatment. The total network cost is associated with the capital, fixed operational (leukapheresis and in-house quality control expenses), manufacturing and transportation cost. The responsiveness of the network is measured through the average lead time for the production and delivery of a therapy, which is the sum of the time a treatment spends at every stage of the supply chain. For the cost minimisation scenarios, an upper bound on the average response time (ART) is introduced (ART=U, where U corresponds to the maximum time allowed before the therapy is be administered to the patient). A set of network and transportation constraints, ensures that only feasible connections are established inside the supply chain network. As regulatory authorities require manufacturers of biological medicinal products to conduct comparability studies for the introduction of new manufacturing facilities, an upper bound constraint is introduced to prevent the establishment of more than two production sites. Sequencing constraints and material balances at each supply chain node impose the correct sequence of events inside the supply chain for every treatment. Finally, capacity constraints on the treatment centres and the manufacturing sites enforce upper bound limitations on the scheduling of therapies. Publication to become available in the 2nd trimester of 2020 - paper submitted and accepted |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | The model allows economic performance of each network evaluated through the achievable average cost per therapy. The results demonstrate that the cost significantly decreased with an increase in patient demand. The high demand scenarios presented reduced operational costs, due to higher manufacturing capacity utilisation. In addition, there is a clear cost trade-off between the supply chain responsiveness and economies of scale. Strict time constraints resulted in higher infrastructure and transportation costs, decreasing the profit gap per therapy. Finally, the capital costs are identified as the major cost contributor in the production of CAR-T cell therapies, while transportation costs only correspond to ~ 2% of the total therapy cost. |
| Title | Multivariate statistical / Neural network Models of Cell-Free synthesis |
| Description | These data-based models estimate protein titre from process parameters measurements (Such as DO, Temperature, pH, etc.). |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | It was possible to compare the quality of predictions from different modelling techniques. |
| URL | https://github.com/CarlosADuranVIllalobos/CFPS-Multivariate-Statistical-Data-Analysis |
| Title | Multivariate statistical / Neural network Models of T-cell expansion |
| Description | These data-based models estimate cell growth and fold-expansion from process parameters measurements (Such as DO, Temperature, Glucose consumption, Lactate production, pH, etc.). |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | It was possible to compare the quality of predictions from different modelling techniques. |
| Title | Supplementary Information Files for Application of a simple unstructured kinetic and cost of goods models to support T-cell therapy manufacture |
| Description | Supplementary Information Files for Application of a simple unstructured kinetic and cost of goods models to support T-cell therapy manufactureManufacturing of cell therapy products requires sufficient understanding of the cell culture variables and associated mechanisms for adequate control and risk analysis. The aim of this study was to apply an unstructured ordinary differential equationbased model for prediction of T-cell bioprocess outcomes as a function of process input parameters. A series of models were developed to represent the growth of T-cells as a function of time, culture volumes, cell densities, and glucose concentration using data from the Ambr®15 stirred bioreactor system. The models were sufficiently representative of the process to predict the glucose and volume provision required to maintain cell growth rate and quantitatively defined the relationship between glucose concentration, cell growth rate, and glucose utilization rate. The models demonstrated that although glucose is a limiting factor in batch supplied medium, a delivery rate of glucose at significantly less than the maximal specific consumption rate (0.05 mg 1 ? 106 cell h1 ) will adequately sustain cell growth due to a lower glucose Monod constant determining glucose consumption rate relative to the glucose Monod constant determining cell growth rate. The resultant volume and exchange requirements were used as inputs to an operational BioSolve cost model to suggest a cost-effective T-cell manufacturing process with minimum cost of goods per million cells produced and optimal volumetric productivity in a manufacturing settings. These findings highlight the potential of a simple unstructured model of T-cell growth in a stirred tank system to provide a framework for control and optimization of bioprocesses for manufacture. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| URL | https://repository.lboro.ac.uk/articles/dataset/Supplementary_Information_Files_for_Application_of_a... |
| Title | T-cell expansion (wells and flasks) |
| Description | Several data-sets of 7-24 observations (repeats) each. They contain final cell growth, final fold expansion, off-line flow cytometry and on-line process measurements such as pH, Temperature and DO in 100ml flasks. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | The data-sets were useful for analyses which involved process characterization, optimization, and comparability between experiments |
| Title | Techno-economic engine for autologous cell therapies |
| Description | An object-oriented model to simulate technical and financial aspects in bioprocessing for autologous cell therapies. The model offers the ability to simulate the manufacture of CAR-T therapies and evaluate multiple manufacturing strategies considering different process flowsheets. |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2019 |
| Provided To Others? | No |
| Impact | Alternative technologies (e.g integrated systems, rocking motion bioreactors, hollow fibre bioreactors) can be rapidly evaluated under technical and financial criteria and determine their fit into a manufacturing facility. Additionally, key cost driver can be identified along with the necessary process improvements to design a feasible and cost-effective manufacturing process. |
| Title | Techno-economic engine for biopharmaceuticals |
| Description | An object-oriented model to simulate technical and financial aspects in bioprocessing for therapeutic proteins. The model offers the ability to simulate the manufacture of different therapeutic proteins (e.g. monoclonal antibodies, antibody drug conjugates, fusion proteins etc.) and evaluate multiple manufacturing strategies considering different process flowsheets (cell culture, cell-free synthesis, chromatographic purification, column-free purification etc.) and modes of operation (batch, fed-batch, perfusion, hybrids). Additionally, the model offers the option to simulate multiple process flowsheets in the same manufacturing facility. |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2018 |
| Provided To Others? | No |
| Impact | Alternative technologies (e.g cell-free synthesis) can be rapidly evaluated under technical and financial criteria and determine their fit into a manufacturing facility. Additionally, key cost driver can be identified along with the necessary process improvements to design a feasible and cost-effective manufacturing process. |
| Title | Techno-economic engine for mRNA vaccines |
| Description | An object-oriented model to simulate technical and financial aspects in bioprocessing for mRNA vaccines. The model offers the ability to evaluate multiple manufacturing strategies considering different process flowsheets. Additionally, the model offers the option to simulate multiple process flowsheets in the same manufacturing facility. |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2021 |
| Provided To Others? | No |
| Impact | Alternative technologies (e.g standalone and integrated unit operations) can be rapidly evaluated under technical and financial criteria and determine their fit into a manufacturing facility. Additionally, key cost driver can be identified along with the necessary process improvements to design a feasible and cost-effective manufacturing process. |
| Title | Techno-economic engine for viral vectors |
| Description | An object-oriented model to simulate technical and financial aspects in bioprocessing for viral vectors. The model offers the ability to simulate the manufacture of different viral vectors (e.g. lenti, adeno & adeno-associated vectors) and evaluate multiple manufacturing strategies considering different process flowsheets (adherent, suspension, chromatographic purification, column-free purification etc.) and modes of operation (batch, fed-batch, perfusion, hybrids). |
| Type Of Material | Computer model/algorithm |
| Year Produced | 2021 |
| Provided To Others? | No |
| Impact | Alternative technologies (e.g chromatography and ultracentrifugation) can be rapidly evaluated under technical and financial criteria and determine their fit into a manufacturing facility. Additionally, key cost driver can be identified along with the necessary process improvements to design a feasible and cost-effective manufacturing process. |
| Description | 3M (FTHM Hub Partner) |
| Organisation | 3M |
| Department | 3M UK & Ireland |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15,000 per year from 2019 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to societ |
| Start Year | 2019 |
| Description | AAV materials from Pharmaron |
| Organisation | Pharmaron |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Input of PDRA and PI time in meetings (2 hours each per month, over 9 months): 36 hours total. PDRA time on research: 90 days total. |
| Collaborator Contribution | Input of consulting time from two company researchers (2 hours each per month, over 9 months): 36 hours total. This defined the research project, provided input on protocols and assays used in industry to be replicated at UCL. AAV viral vector supplies for the project. ELISA kit supply has lowered the cost-burden to UCL for AAV analysis. Assays carried out at the company site on samples from UCL have been essential where UCL does not have the facilities. |
| Impact | Input on AAV manufacturing has helped UCL establish in-house manufacturing capabilities for Hub research. AAV viral vector supplies for the project have been critical for benchmarking analytical instrumentation at UCL, and for carrying out novel formulation research. |
| Start Year | 2021 |
| Description | ALLCAR19 Trial, Cancer Research UK & UCL Cancer Trials Centre |
| Organisation | University College London |
| Department | Cancer Trials Centre |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Health economics analysis plan added to ALLCART19, trial protocol: Evaluate the cost and cost-effectiveness of second generation CAR T therapy to the NHS in patients with (i) Diffuse large B-cell lymphoma (DLBCL) versus salvage chemotherapy (nice.org.uk/guidance/ta559) (ii) Chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL) allogeneic transplantation (iii) Indolent non-Hodgkin's lymphoma (B-NHL) versus Primary outcomes: (i) Health care resources utilised and direct medical costs (ii) Health related quality of life (HRQoL) measured by the EuroQol 5-dimension 3-level (EQ-5D-3L) and the EORTC QLQ-C30 |
| Collaborator Contribution | This is a multi-centre, open label, non-randomised Phase I trial. Data for the health economics analysis will be supplemented with data from the trial. The aims of the trial are evaluate adverse events experienced with second generation CD19 CAR T-cells: 1. To evaluate the feasibility of generating autologous CD19 Chimeric Antigen Receptor (CAR) T-cell therapy from patients with resistant or relapsed (r/r) adult B-ALL, r/r B-NHL and in r/r CLL/SLL. 2. To evaluate the safety of administering CD19 CAR T-cell therapy to patients with r/r B-ALL, in r/r B-NHL and in r/r CLL/SLL. 3. In B-ALL patients only, to evaluate the efficacy and duration of re-sponse to CD19 CAR T-cells. The protocol with the health economics has been submitted to ethics and Medical Healthcare Product Research Agency (MHRA). |
| Impact | This will be a multi-disciplinary collaboration between the FTHM Hub and clinicians at UCL. |
| Start Year | 2020 |
| Description | Access to HDX MS at LGC |
| Organisation | LGC Ltd |
| Country | Global |
| Sector | Private |
| PI Contribution | UCL produces protein formulations to be further analysed at LGC. We have also helped LGC to develop the capability for HDX on solid state (freeze-dried) materials. |
| Collaborator Contribution | LGC have provided access to their Mass spectrometry facility, particularly to carry out HDX and peptide mapping of proteins and their formulations. |
| Impact | LGC have trained two PhD students - both funded by the EPSRC CDT, and also one PDRA from the EPSRC Hub. They have also provided access to their facilities to carry out a large body HDX peptide mapping for GCSF and IgG formulations. |
| Start Year | 2016 |
| Description | Adaptimmune Therapeutics (FTHM Hub Partner) |
| Organisation | Adaptimmune |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £7000.00 per year from 2019 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2019 |
| Description | Advanced Bioprocess Services |
| Organisation | Advanced Bioprocess Services Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | We have partnered with Advanced Bioprocess Services to deliver technology and process development expertise to clinical stage therapeutic product companies. These have included pre-clinical bioreactor process development and modelling for several Boston (US) based blood products companies, and process development for leading UK based companies in neural stem cell and immunotherapies. Our contribution has been specifically in novel process model development, novel methods to improve process efficiency, and technology design. |
| Collaborator Contribution | Advanced Bioprocess Services manage the interface with companies seeking process development and provide extensive high value reagents and consumables for project work, either directly or via other collaborating companies, as well as providing access to the most relevant and protected industrial processes for development of our research. Since 2016 Advanced Bioprocess Services have directly funded 258,000 GBP of research and impact activity through the University as of end of 2019 |
| Impact | Novel industrially applied processes |
| Start Year | 2016 |
| Description | Aglaris (FTHM Hub Partner) |
| Organisation | Aglaris Ltd |
| Country | Spain |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year from 2018 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Sponsored a Hub User Feasibility Study in collaboration with the University of Manchester Spoke (this has been report in a separate P&C record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2018 |
| Description | Albumedix (FTHM Hub Partner) |
| Organisation | Albumedix Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £3000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Sponsor of multiple separate user feasibility studies with Hub and Spoke University partners (these have been reported in a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Allergan (FTHM Hub Partner) |
| Organisation | Allergan |
| Country | Global |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Applikon Biotechnology (FTHM Hub Partner) |
| Organisation | Applikon Biotechnology B.V. |
| Country | Netherlands |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | New partnership in 2021, information to follow |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2021 |
| Description | AstraZeneca (FTHM Hub Partner) |
| Organisation | AstraZeneca |
| Department | MedImmune |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £7000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1500 per day contributed Provision of advice and guidance on the overall direction and progress of the Hub research programme ensuring it is appropriate to their medium to long term needs while satisfying the remit of the Hub, via Advisory Board, valued @£1500 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Autolus (FTHM Hub Partner) |
| Organisation | Autolus Limited |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Provision of advice and guidance on the overall direction and progress of the Hub research programme ensuring it is appropriate to their medium to long term needs while satisfying the remit of the Hub, via Advisory Board, valued at £1000 per day contributed Provision of expert input on cell therapy manufacture and analytics |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | BIA Separations (FTHM Hub Partner) |
| Organisation | BIA Separations |
| Country | Slovenia |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation at Hub meetings and working groups, valued @£2000.00 per day contributed Equipment, PATfixTM system, valued at £83,500 Conference registration fees, valued at £3000 Provision of training, valued at £5000 |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Big data analysis and decision-support for personalised diagnosis and treatment of lung cancer |
| Organisation | Shanghai Pulmonary Hospital |
| Country | China |
| Sector | Hospitals |
| PI Contribution | Apply novel big data techniques to integrate and analyze all data from the database and repositories in order to explore and identify effective biomarkers related to lung cancer such as imaging biomarkers (CT, PET, MRI) and molecular biomarkers (plasma, serum, cerebrospinal fluid, gene expression). These biomarkers can act as indicators of different forms or subtypes of lung cancer. Novel causal relationship analysis (e.g. decision tree, multivariate regression) will be applied to find common factors and causes of variation which will enable a better understanding of the root causes of lung cancer and rapid diagnosis of subtypes and stages. Association rule analysis (e.g. Apriori, Eclat) will be used to explore the link between the biomarkers and response to treatment so that the treatment can be tailored for specific patients according to personal response, clinical effectiveness and adverse reactions. |
| Collaborator Contribution | Shanghai Pulmonary Hospital is the leading pulmonary hospital in China that provides treatment for over 140,000 patients and conducts about 7,000 cases of lung resection per year. They have a well-established clinical and diagnostic database with complete follow-up information records for 95% patients and large repositories of lung cancer pathology specimen and blood specimen bank repositories. |
| Impact | Conference poster of Machine learning in personalized prognosis prediction of lung cancer on ISMB, Switzerland, 2019 Journal paper of Machine learning application in personalised lung cancer recurrence and survivability prediction (submitted to Scientific Report) |
| Start Year | 2017 |
| Description | BioIndustry Association (FTHM Hub Partner) |
| Organisation | BioIndustry Association |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Conference & Training course registration fees, annually (e.g. BioProcess UK, UK Bioscience Forum) valued @ £320 per head Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Provision of advice and guidance alignment of Hub with sector and national biotech strategies, valued @1000 per day contributed Organising committee for partnered-Hub events (1 per year, e.g. manufacturing ATMPs in 2018, Viral Vecs in 2019), valued @1000 per day contributed Dissemination using BIA media channels/database of bioscience companies, valued @ 3000 per year Assistance in identifying user demonstrated feasibility projects designed to illustrate the impact of the Hub research outputs, valued @1000 per day contributed Sponsored a user feasibility project in partnership with UCL and with member-companies of BIA- Manufacturing Advisory Group (BIA-MAC, reported in a separate C&P record) Invited webinar speaker (May 2020), invited keynote speaker (Nov 2020) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Biologic B Consulting (FTHM Hub Partner) |
| Organisation | BioLogicB LLC |
| Country | United States |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings, working groups, dedicated consultation meetings, incl international travel, valued @£2500.00 per day contributed Provision of expert input on future directions for the development and manufacture of stratified biologics and cell therapies Provision of advice and guidance on the overall direction and progress of the Hub research programme, as a national resource and international network/profile (e.g. NIIMBLE) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Biopharm Services (FTHM Hub Partner) |
| Organisation | Biopharm Services |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Provision of Software, BioSolve Process valued at £28,000 per year, from 2017 Provision of Sofware, BioSolve Connect valued at £40,000 plus £12,500 per year from 2017 |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | CSL Behring (FTHM Hub Partner) |
| Organisation | CSL Behring |
| Country | United States |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual Cash contribution from 2021. Provision of advice and guidance on the overall direction and progress of the Hub via participation in Hub meetings and working groups. |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society. |
| Start Year | 2021 |
| Description | Cancer Research UK (FTHM Hub Partner) |
| Organisation | Cancer Research UK |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Co-organised a Hub event, Cell Therapy Technology Showcase event, valued at £1000 per day contributed Sponsored a user feasibility project in partnership with UCL around cell-free synthesis (reported in a separate C&P record) Assistance with Webinar speaker (Sep 2020) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2019 |
| Description | Cell & Gene Therapy Catapult (FTHM Hub Partner) |
| Organisation | Cell Therapy Catapult |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Provision of expert advice and guidance on the Healthcare Reimbursement workstream via 1:1 meetings with team members, valued @£500 Assistance in identifying and prioritising user demonstrated projects designed to illustrate the impact of the Hub research outputs via Translation and Impact Committee, valued @ £1000 per day contributed Provision of advice and guidance on the overall direction and progress of the Hub research programme ensuring it is appropriate to their medium to long term needs while satisfying the remit of the Hub, via Advisory Board, valued @£1000 per day contributed Conference & Training course registration fees (e.g.Cell and Gene Therapy Manufacturing Workshop) valued @ £500 Sponsored a user feasibility project in partnership with Loughborough University (reported in a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Cell Medica (FTHM Hub Partner) |
| Organisation | Cell Medica Ltd. |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year, collected for their first year (2019) Provision of advice and guidance on the overall direction and progress of the Hub, via participationat Hub meetings and working groups, valued @£1000.00 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2019 |
| Description | Centre for Process Innovation (FTHM Hub Partner) |
| Organisation | Centre for Process Innovation (CPI) |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Assistance in identifying and prioritising user demonstrated projects designed to illustrate the impact of the Hub research outputs via Translation and Impact Committee, valued @£1000 per day contributed Provision of advice and guidance on the overall direction and progress of the Hub research programme ensuring it is appropriate to their medium to long term needs while satisfying the remit of the Hub, via Advisory Board, valued @ 1000 per day contributed Sponsored the Cell-Free Synthesis Specialist Working Group meeting at Darlington site, valued @£2000 |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to societ |
| Start Year | 2017 |
| Description | Characterisation of degradations in biotherapeutics coformulation |
| Organisation | AstraZeneca |
| Department | MedImmune |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | The co-formulation of biotherapeutics has been researched as a form of drug combination product which brings conveniences of storage and administration. We study the analytics and control strategy for degradation in coformulated therapeutic proteins, primarily antibodies, using a range of biophysical techniques. |
| Collaborator Contribution | MedImmune provides three antibodies under the MTA framework. The materials provided by MedImmune will be mixed into ratios for aggregation study in our team. |
| Impact | Aggregation kinetics of these MedImmune antibodies is studied at the moment. The prelimarily result shows a concentration-dependent kinetics of aggregation for some of these antibodies. Another therapeutic proteins which is previously shown to be aggregation-inhibitory will be mixed with MedImmune antibodies to test if the aggregation can be prevented. |
| Start Year | 2018 |
| Description | Collaboration on phenotyping of immunotherapeutic T/CAR-T cell products by Raman spectroscopy |
| Organisation | University College London |
| Department | Biochemical Engineering |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Optimise recently installed (BioScience DivRaman spectroscopy for live single cell analysis. Co-supervise MSc student affilated with FTHM Hub 2.3a project on characterization of immune cells by Raman spectroscopy. Participate in acquisition of Raman spectra of immunotherapeutic T cells. Multivariate analysis of T cells Raman spectra alowing to distinquish non-activated and activated human T cells by its unique biochemical content. |
| Collaborator Contribution | Access to new Raman facilities. Participate in acquisition of limited sets of T cells Raman spectra. |
| Impact | Non-activated and activated human T cells were distinquished by Raman spectroscopy combined with chemometrics methods - i.e. by its unique biochemical content. |
| Start Year | 2019 |
| Description | Collaboration on phenotyping of immunotherapeutic T/CAR-T cell products by Raman spectroscopy |
| Organisation | University College London |
| Department | Department of Cell and Developmental Biology |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Optimise recently installed (BioScience DivRaman spectroscopy for live single cell analysis. Co-supervise MSc student affilated with FTHM Hub 2.3a project on characterization of immune cells by Raman spectroscopy. Participate in acquisition of Raman spectra of immunotherapeutic T cells. Multivariate analysis of T cells Raman spectra alowing to distinquish non-activated and activated human T cells by its unique biochemical content. |
| Collaborator Contribution | Access to new Raman facilities. Participate in acquisition of limited sets of T cells Raman spectra. |
| Impact | Non-activated and activated human T cells were distinquished by Raman spectroscopy combined with chemometrics methods - i.e. by its unique biochemical content. |
| Start Year | 2019 |
| Description | Collaboration with Cell Medica |
| Organisation | Cell Medica Ltd. |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | We have provided Cell Medica with relevant expertise in the scale-up and manufacture of iNKTs as well as techniques for the scalable cryopreservation of iNKT cell therapies. |
| Collaborator Contribution | Cell Medica have provided a significant amount of consumables for research work at UCL, in excess of £15,000 of consumable items. |
| Impact | A key output will be a paper that draws on the key research findings that have emanated from the study. |
| Start Year | 2019 |
| Description | Collaboration with Sartorius for CAR-T manufacture |
| Organisation | Sartorius |
| Country | Germany |
| Sector | Private |
| PI Contribution | We have established a CAR-T manufacturing process that works for the ambr250 and the Rocking Motion Bioreactor. This has led to publications and webinars hosted by Sartorius promoting the work. We have demonstrated successful expansion and subsequent isolation and characterisation of functionally-effective CAR-T cells using the Sartorius platforms. |
| Collaborator Contribution | Provision of the ambr250 system with perfusion capability and the Rocking Motion bioreactor platform. This also includes all associated consumables to run the systems. |
| Impact | 2 x Publications in Biotechnology and Bioengineering and Biotechnology Journal 2 x Webinars 3 x Conference Presentations |
| Start Year | 2019 |
| Description | Collaboration with Sartorius on ambr250 use for Cell and Gene Therapy |
| Organisation | Sartorius |
| Department | Sartorius Stedim Biotech |
| Country | France |
| Sector | Private |
| PI Contribution | We (the FTHM Hub) have provided significant expertise and knowledge to the development of the new cell and gene therapy amb250 bioreactor vessel. We have also demonstrated and published on data highlighting it's use for cell and gene therapy applications, including T-cell production. |
| Collaborator Contribution | Sartorius have provided a 3-year loan of the ambr250 bioreactor system (value of loan is £50,000) which is likely to be extended for an additional period of time. They have also provided all of the consumables required for the project, including bioreactor vessels, filters and tubing sets free of charge. The estimated cost of this for the 3 year period is in excess of £50,000. |
| Impact | (1) A new bioreactor vessel has been released by Sartorius with direct input and involvement from academics at UCL. (2) A paper has been published in Biotechnology and Bioengineering which demonstrates the use of the ambr250 for T-cell production. (3) A white paper has been published by Sartorius for the use of the ambr250 for cell and gene therapy applications. (4) 8 presentations both nationally and internationally have been delivered on this work. (5) 2 more papers are in submission showing the use of the system for CAR-T and scalability between ambr15, ambr250 and the 1-L Stirred-tank systems. |
| Start Year | 2017 |
| Description | Cost model for automated T cell bio processing and manufacturing with Biopharm services |
| Organisation | Biopharm Services |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Our contribution was to show the application of ODE framework modelling in rapidly providing an insight to T-cell manufacturing process optimisation. We described how the approach can be applied, with very limited process data, to define medium exchange process operation limits in a stirred tank culture format. We further showed how the approach can be evolved to provide further process insight as more analytical data, such as nutrient concentrations, becomes available. Finally, we evaluate the process optimisation and risk understanding that can be gained from such an approach. |
| Collaborator Contribution | Biopharm Services contributed by carried out a cost model for variety of feeding scenarios in automated expansion of T cell in a manufacturing setting for example a 2L bioreactor. |
| Impact | Finalizing a manuscript on "Application of a Simple Unstructured Kinetic Model to Support T-Cell Therapy Manufacture "which is currently under the review by Biopharm services team. |
| Start Year | 2020 |
| Description | Eli Lilly (FTHM Hub Partner) |
| Organisation | Eli Lilly & Company Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£2000 per day contributed Keynote speaker (Nov 2020) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | FUJIFILM Irvine Scientific collaboration |
| Organisation | Irvine Scientific |
| Country | United States |
| Sector | Private |
| PI Contribution | Postdoctoral expertise, intellectual input. |
| Collaborator Contribution | Intellectual input, materials and reagents. |
| Impact | -The viral titre from serum-free, chemically-defined, suspension system is comparable to that of the conventional serum-containing, adherent system. -Healthy donor CAR-T cells can be effectively grown in serum-free, chemically-defined conditions. -The media in which CAR-T cells are grown may affect their killing capability in vitro. This collaboration is not multi-disciplinary. |
| Start Year | 2019 |
| Description | Francis Biopharma Ltd (FTHM Hub Partner) |
| Organisation | Francis Biopharma |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via meetings with the Hub leadership valued @£2000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | FujiFilm Diosynth Biotechnologies (FTHM Hub Partner) |
| Organisation | Fujifilm |
| Department | Fujifilm Diosynth Biotechnologies |
| Country | United States |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £7000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Sponsored a user feasibility project in partnership with UCL (reported in a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | GSK (FTHM Hub Partner) |
| Organisation | GlaxoSmithKline (GSK) |
| Country | Global |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Provision of advice and guidance on the overall direction and progress of Hub workstreams via consultation with individual team members valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Holographic Imaging |
| Organisation | Ovizio Imaging Systems NV SA |
| Country | Belgium |
| Sector | Private |
| PI Contribution | We provided the T-cell work, and made sure to get good quality images for processing. We demonstrated how the activation status of a T-cell can be detected using holographic imaging. This has been presented at conferences. |
| Collaborator Contribution | A free of charge trial of qMod DHM camera and OsOne image processing software. |
| Impact | Poster "Label-free recognition of non-activated and activated human primary T cells by Quantitative Phase Imaging" at BioMAN 2019 Summit ("Technology and Innovation for Sustainable Manufacturing of Advanced Therapy Medicinal Products", Cambridge, US, 11-12 December 2019). Talk at "Could key cell attributes be defined and monitored label-free for a Quality Control of manufacturing a safe and potent immunotherapeutic cell products" for Cell and Gene Therapy Innovations 2020 Summit (Berlin, 5-6 February 2020) Accepted abstract "Label-free recognition of non-activated and activated T cells on a chip by Quantitative Phase Imaging" for EUROPTRODE 2020 conference (Poland, April 2020 but postponed to July 2020, then to March 2021 and now postponed again |
| Start Year | 2019 |
| Description | Hub-Autolus User feasibility study: Techno-economic analysis and supply chain optimisation for the assessment of the "fresh-in/cryo-out" business model under uncertainty |
| Organisation | Autolus |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Provided the modes to simulate supply chain logistics (Imperial), the factory floor scheduling (Warwick), and process economics (UCL) for autologous CART therapies. |
| Collaborator Contribution | Autolus provided data to build/inform the models, and insights on relevant scenarios to analyse. |
| Impact | This collaboration is still ongoing. Preliminary results on the comparison between fresh and cryogenic transportation have been obtained and identified potential benefits associated with cryogenic transportation mode due to improved flexibility and enhanced scheduling of the manufacturing facility. Once completed the supply chain model will provide useful information on the optimal supply chain configurations to minimise the therapy cost, as well as help to identify trade-offs between average cost and average vein-to-vein time. The effect of key uncertainties (e.g. manufacturing time) on the overall supply chain configuration will also be assessed. |
| Start Year | 2021 |
| Description | Hub-Autolus User feasibility study: Techno-economic analysis and supply chain optimisation for the assessment of the "fresh-in/cryo-out" business model under uncertainty |
| Organisation | Autolus |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Provided the modes to simulate the factory floor scheduling (Warwick), process economics (UCL), and the supply chain logistics (Imperial) for autologous CART therapies. |
| Collaborator Contribution | Autolus provided the data and information to build the model |
| Impact | At the end of this study, the industrial partner will have: 1. an easy-to-use and fast tool to plan the workforce and resources on the factory floor 2. a tool to react to the realisation of uncertainties, e.i., patients' drop out, expansion uncertainty, and delay in leuk delivery 3. a tool to plan new patients according to the latest resource availability |
| Start Year | 2021 |
| Description | Hub-Autolus User feasibility study: Techno-economic analysis and supply chain optimisation for the assessment of the "fresh-in/cryo-out" business model under uncertainty |
| Organisation | Imperial College London |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Provided the modes to simulate the factory floor scheduling (Warwick), process economics (UCL), and the supply chain logistics (Imperial) for autologous CART therapies. |
| Collaborator Contribution | Autolus provided the data and information to build the model |
| Impact | At the end of this study, the industrial partner will have: 1. an easy-to-use and fast tool to plan the workforce and resources on the factory floor 2. a tool to react to the realisation of uncertainties, e.i., patients' drop out, expansion uncertainty, and delay in leuk delivery 3. a tool to plan new patients according to the latest resource availability |
| Start Year | 2021 |
| Description | Hub-Autolus User feasibility study: Techno-economic analysis and supply chain optimisation for the assessment of the "fresh-in/cryo-out" business model under uncertainty |
| Organisation | University College London |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Provided the modes to simulate the factory floor scheduling (Warwick), process economics (UCL), and the supply chain logistics (Imperial) for autologous CART therapies. |
| Collaborator Contribution | Autolus provided the data and information to build the model |
| Impact | At the end of this study, the industrial partner will have: 1. an easy-to-use and fast tool to plan the workforce and resources on the factory floor 2. a tool to react to the realisation of uncertainties, e.i., patients' drop out, expansion uncertainty, and delay in leuk delivery 3. a tool to plan new patients according to the latest resource availability |
| Start Year | 2021 |
| Description | Hub-Autolus User feasibility study: Techno-economic analysis and supply chain optimisation for the assessment of the "fresh-in/cryo-out" business model under uncertainty |
| Organisation | University College London |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Provided the modes to simulate supply chain logistics (Imperial), the factory floor scheduling (Warwick), and process economics (UCL) for autologous CART therapies. |
| Collaborator Contribution | Autolus provided data to build/inform the models, and insights on relevant scenarios to analyse. |
| Impact | This collaboration is still ongoing. Preliminary results on the comparison between fresh and cryogenic transportation have been obtained and identified potential benefits associated with cryogenic transportation mode due to improved flexibility and enhanced scheduling of the manufacturing facility. Once completed the supply chain model will provide useful information on the optimal supply chain configurations to minimise the therapy cost, as well as help to identify trade-offs between average cost and average vein-to-vein time. The effect of key uncertainties (e.g. manufacturing time) on the overall supply chain configuration will also be assessed. |
| Start Year | 2021 |
| Description | Hub-Autolus User feasibility study: Techno-economic analysis and supply chain optimisation for the assessment of the "fresh-in/cryo-out" business model under uncertainty |
| Organisation | University of Warwick |
| Department | Warwick Business School |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Provided the modes to simulate supply chain logistics (Imperial), the factory floor scheduling (Warwick), and process economics (UCL) for autologous CART therapies. |
| Collaborator Contribution | Autolus provided data to build/inform the models, and insights on relevant scenarios to analyse. |
| Impact | This collaboration is still ongoing. Preliminary results on the comparison between fresh and cryogenic transportation have been obtained and identified potential benefits associated with cryogenic transportation mode due to improved flexibility and enhanced scheduling of the manufacturing facility. Once completed the supply chain model will provide useful information on the optimal supply chain configurations to minimise the therapy cost, as well as help to identify trade-offs between average cost and average vein-to-vein time. The effect of key uncertainties (e.g. manufacturing time) on the overall supply chain configuration will also be assessed. |
| Start Year | 2021 |
| Description | Hub-Autolus User feasibility study: Techno-economic analysis for the assessment of the "fresh-in/cryo-out" business model under uncertainty |
| Organisation | Autolus |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Provided the models to simulate the process economics for autologous CART therapies. |
| Collaborator Contribution | Autolus provided the data to populate the models. Helped to formulate the scenarios for analysis. |
| Impact | The study is still active. Once completed it is expected to achieve the following: Techno-economics for fresh-in/cryo-out: • Identify key COG drivers and innovation opportunities across the value chain to materially reduce costs per patient dosed by >20% without compromise to quality and production risk • Evaluate extent of global coverage with centralized and regional site configurations • Evaluate impact of uncertainties on likelihoods of meeting throughputs and COG targets Scheduling and supply chain logistics for fresh-in/cryo-out: • Evaluate impact of different objectives (e.g. profitability) and constraints (e.g. vein-to-delivery) on the optimal supply chain configurations across networks involving multiple hospitals and manufacturing sites and viral vector suppliers • Evaluate impact of different objectives (e.g. cost v time) on the optimal factory floor scheduling including leukapheresate arrival patterns • Develop efficient and robust supply chain and scheduling logistics that account for uncertainties (e.g. patient-to-patient variability, transport disruptions) with flexible real-time control processes • Evaluate the benefit of QC in parallel to transportation |
| Start Year | 2021 |
| Description | Industry partnership for materials from UCB Pharma |
| Organisation | UCB Pharma |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | We analysed the aggregation behaviour of a Fab protein obtained from UCB Pharma under a wide range of conditions. This has provided general information on the aggregation mechanisms, formulations and stabilising factors in Fab molecules, useful for therapeutic formulations and bioprocessing. |
| Collaborator Contribution | UCB Pharma provided an E. coli strain that produces the A33 Fab fragment. They also provided advice for its expression and analysis. |
| Impact | Scientific outputs on formulation of Fab and understanding of aggregation mechanisms. The access to this material has also enabled us to develop novel analytical techniques in other grants. The partnership has also led to three CASE-PhD collaborations with UCB in 2017. |
| Start Year | 2011 |
| Description | Ipsen (FTHM Hub Partner) |
| Organisation | Ipsen |
| Country | Global |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £7000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Provision of advice and guidance on workstream-related collaborative funding bid, via consultation with senior academic team valued @£1000 per day contributed Sponsored a user feasibility project in partnership with UCL (reported in a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Knowledge Transfer Network (KTN, FTHM Hub Partner) |
| Organisation | Knowledge Transfer Network |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Provision of advice and guidance on the overall direction and progress of the Hub research programme ensuring it is appropriate to their medium to long term needs while satisfying the remit of the Hub, via Advisory Board, valued @£1000 per day contributed Support for co-sponsored networking events, valued @ £3000 per year (1) Vaccine Development Workshop, 6th September 2017, London (2) https://ktn-uk.co.uk/news/manufacturing-atmps-moving-from-approvable-to-commercial-success (3) https://ktn-uk.co.uk/news/viral-vectors-for-vaccines-and-gene-delivery Dissemination using database of bioscience companies, access to proprietary roadmap, valued @ £2000 pear year |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Kuopio Centre for Cell and Gene Therapy (FTHM Hub Partner) |
| Organisation | Kuopio Center for Gene and Cell Therapy |
| Country | Finland |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2020 |
| Description | LGC (FTHM Hub Partner) |
| Organisation | Laboratory of the Government Chemist (LGC) Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Access/exploitation of mass spectrometry capabilities and instrumentation, including training and supervision (collaborating with UCL, reported as a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Lonza (FTHM Hub Partner) |
| Organisation | Lonza Group |
| Department | Lonza Biologics |
| Country | United States |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1500 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Manchester/KCT Multivariate data analysis of bioreactor measurements of process parameters and end-point qualities (viral titter and cell growth). |
| Organisation | Kuopio Center for Gene and Cell Therapy |
| Country | Finland |
| Sector | Private |
| PI Contribution | Multivariate statistical data analysis of experimental data. This study will seeked to optimise the trajectory (-ies) of one or more Critical Process Parameters (CPPs) found to have an important effect in product quality. |
| Collaborator Contribution | -Manufacturing data (e.g. yields, concentrations, measurable CPPs, etc.). -Feedback and technical support |
| Impact | No impact yet |
| Start Year | 2020 |
| Description | Materials and facility access from NIBSC |
| Organisation | National Institute for Biological Standards and Control (NIBSC) |
| Country | United Kingdom |
| PI Contribution | We analysed the aggregation behaviour, and stability of a GCSF protein and mutants of this, obtained from NIBSC under a wide range of formulations. This has provided general information on the aggregation mechanisms, formulations and stabilising factors in GCSF molecules, useful for therapeutic formulations and bioprocessing. |
| Collaborator Contribution | NIBSC provided an E. coli strain that produces the GCSF. They also provided advice for its expression and analysis. They also provided access to NMR, pilot-scale freeze dryers, Karl Fischer analysis, biological potency assays, and Mass spectrometry. |
| Impact | This partnership has involved one EPSRC EngD, one BBSRC PhD, and two EPSRC CDT PhD students, formal partnership and strategic advice for the EPSRC Formulation project, Centre for Innovative Manufacturing and Future Targeted Healthcare Manufacturing Hub, as well as attendance by NIBSC at Hub events and workshops. The partnership is multi-disciplinary, bringing together protein biophysics (UCL), protein engineering (UCL), protein aggregation (UCL), freeze-drying (NIBSC), biological assays (NIBSC), NMR (NIBSC) and Mass spectrometry (NIBSC). Outputs therefore include, 3 graduated PhD/EngDs, 1 PhD currently running, 3 PDRAs receiving training and carrying out work in NIBSC facilities, 5 co-authored publications. |
| Start Year | 2007 |
| Description | Materials from Medimmune/AZ |
| Organisation | AstraZeneca |
| Department | MedImmune |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | We have analysed the antibody samples using novel analytics |
| Collaborator Contribution | Donation of five antibody and bispecific samples. |
| Impact | Molecules have enabled an assessment of analytical techniques we have developed. Molecules have also been used to elucidate new mechanisms involved in protein aggregation in medical formulations. This will enable improved future formulations of antibody therapies. |
| Start Year | 2018 |
| Description | Medicines Manufacturing Industry Partnership (MMIP, FTHM Hub Partner) |
| Organisation | Medicines Manufacturing Industry Partnership |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on alignment of Hub with sector and national biotech strategy, valued at £1000 per day contributed Additional contributions captured within the C&P records for Knowledge Transfer Network (KTN) , Association of Biopharmaceutical Industry (ABPI) and BioIndustry Association (BIA) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | NIBSC (FTHM Hub Partner) |
| Organisation | National Institute for Biological Standards and Control (NIBSC) |
| Country | United Kingdom |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Orchard Therapeutics (FTHM Hub Partner) |
| Organisation | Orchard Therapeutics |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | further information to follow |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2020 |
| Description | Oxford BioMedica (FTHM Hub Partner) |
| Organisation | Oxford BioMedica UK Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£2000 per day contributed Assistance in identifying and prioritising user demonstrated projects designed to illustrate the impact of the Hub research outputs via Translation and Impact Committee Sponsored a user feasibility project in partnership with UCL & BIA-Manufacturing Advisory Committee (reported in a separate C&P record) Keynote speaker (Nov 2020) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Pall (FTHM Hub Partner) |
| Organisation | PALL Europe |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups,valued @£1000.00 per day contributed Assistance in identifying and prioritising user demonstrated projects designed to illustrate the impact of the Hub research outputs via Translation and Impact Committee, valued @£1000.00 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Pall Europe collaboration |
| Organisation | PALL Europe |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Evaluating samples supplied by Pall in the new instrumentation. |
| Collaborator Contribution | Supply of antibody samples of different grades for analysis. |
| Impact | Led to a follow on EngD project in collaboration with Pall Europe |
| Start Year | 2017 |
| Description | Perceptive Engineering (FTHM Hub Partner) |
| Organisation | Perceptive Engineering Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000.00 per day contributed Access to Software, valued at £30,000, and associated training, valued at £3,000. Webinar speaker (June 2020) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Policy brief on point-of-care manufacture |
| Organisation | United Kingdom Research and Innovation |
| Country | United Kingdom |
| Sector | Public |
| PI Contribution | We are leading the preparation of a policy brief focusing on point-of-care manufacture. |
| Collaborator Contribution | They are helping us refine and enhance the policy brief. |
| Impact | The collaboration will result in a policy brief. Involved in the collaboration are social scientists and a NHS-based pharmacist. |
| Start Year | 2022 |
| Description | Puridify (FTHM Hub Partner) |
| Organisation | Puridify |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000.00 per day contributed Time filming a stakeholder interview for the Hub promotional video, valued at £1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Purolite (FTHM Hub Partner) |
| Organisation | Purolite International Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £7000 per year from 2017 (waived in 2018) Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Time in 1:1 discussions with members of the senior academic team, valued @£1000 per day contributed Sponsored a user feasibility project in partnership with UCL (reported in a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | ReNeuron (FTHM Hub Partner) |
| Organisation | Reneuron |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year from 2017 1:1 discussions with the senior acadeimic team, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Repligen (FTHM Hub Partner) |
| Organisation | Repligen |
| Country | United States |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15,000 per hear from 2021. Provision of advice and guidance on the overall direction and progress of the Hub via participation in Hub meetings and working groups, valued @14000 per year. |
| Impact | Working with a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society. |
| Start Year | 2021 |
| Description | Ribosomal concentrator reactor AFS with CRUK |
| Organisation | Cancer Research UK |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | UCL initiated the idea for this feasibility study. UCL will develop a detailed reactor design, build a prototype using the UCL workshop and test the prototype using in-house model proteins and in-house extract production methodology. |
| Collaborator Contribution | CRUK will provide: • An in-kind contribution up to £50K for the project for additional resource and ongoing support between CRUK and the UCL Hub. • Technical support; • Know-how in the translation of this technology to GMP. • Translation of extract production to scale / GMP. This includes allied support for the project as well as the large-scale testing within their Process Development team (4 scientists/snr scientists and a Process Development Manager). This support can involve production of starting material by development using scale-down bioreactor models and large scale-bioreactor operations to produce the extracts/systems. This includes expertise to provide starting material and operation of the platform in manufacturing. This will also create opportunities for UCL researchers to embed in a GMP manufacturing environment and train on the project and at scale. • Resource and analytical assay support from their QC Analytics team (3 scientists, 1 apprentice, 1 QC Manager). This includes all assays and assay development, analysis and reporting. |
| Impact | TBC |
| Start Year | 2021 |
| Description | SSC Bio (FTHM Hub Partner) |
| Organisation | SSC Bio |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£2000 per day contributed Provision of expert input on healthcare regulation and reimbursement, NICE, valued @ £2000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Sartorius (FTHM Hub Partner) |
| Organisation | Sartorius |
| Department | Sartorius Stedim UK Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15,000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1500 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Stability of coformulated biotherapeutics in the presence of serum albumin |
| Organisation | Albumedix Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | The co-formulation of biotherapeutics has been researched as a form of drug combination product which brings conveniences of storage and administration. However, the mixture of therapeutic proteins could lead to co-aggregation and reduced shelf life. Human serum albumin (HSA) is a widely used protein stabilising biologics in many formulations. We investigate whether HSA can aid the stability of biotheraputics in co-formulations. |
| Collaborator Contribution | Albumedix provides the HSA material under MTA framework for us to make analysis. |
| Impact | The mixture of HSA and antibodies has been created under a few pH conditions. The prelimarily data shows HSA cannot be separated using SEC-HPLC from antibodies. Therefore, other form of separation such as HIC and RP-HPLC are under investigation. |
| Start Year | 2017 |
| Description | Sutro Biopharma (FTHM Hub Partner) |
| Organisation | Sutro Biopharma |
| Country | United States |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £2000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£2000 per day contributed Provision of expert input on cell-free synthesis route for personalised medicines Sponsored a user feasibility project in partnership with UCL (reported in a separate C&P record) Keynote speaker (May 2019) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Testing a novel microfluidic mixing approach |
| Organisation | Redbud Labs |
| Country | United States |
| Sector | Private |
| PI Contribution | We tested the microfluidic mixer with fluorescent particle. The next step was to use cells and see whether vigorous and/or gentle ways of mixing cell suspension solutions of human cells can be achieved |
| Collaborator Contribution | Redbud Labs provided a functioning set-up together with 10 microfluidic chips for testing |
| Impact | The mixing principle was tested but we could not finalise due to the restrictions on lab access. |
| Start Year | 2019 |
| Description | The Association of the British Pharmaceutical Industry (ABPI, FTHM Hub Partner) |
| Organisation | Association of the British Pharmaceutical Industry |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via attendance at Hub meetings and working groups, valued @£1000.00 per day contributed. Webinar speaker, (Sept 2020), Keynote speaker (Nov 2020). Provision of advice and guidance on the overall direction and progress of the Hub research programme ensuring it is appropriate to their medium to long term needs while satisfying the remit of the Hub, via Advisory Board |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Towards the development of a robust scalable platform for the production of Stratified medicines using Cell Free Protein Synthesis: training and knowledge transfer. |
| Organisation | Sutro Biopharma |
| Country | United States |
| Sector | Private |
| PI Contribution | After ann analysis of the published literature we (UCL) recognised the need to accelerate our Cell Free Protein Synthesis capabilities through an acceleration of our technical/hands-on skills and knowledge transfer/organisational learning processes. We recognised that one of the HUB industrial partners is already a leader in this field. Thus a representative of Sutro Biopharma was contacted and a project proposal put forward with very clearly defined outcomes, outputs and benefits. This proposal was accepted and a 1 month secondment for a member of the HUB technical staff to Sutro's South San Francisco research centre took place in the summer of 2018. IN addition to the secondment an agreement was reached where UCL researchers were allowed to use propriety Sutro developed reagents in the UCL Bloomsbury laboratories for the process of staff training/process development. |
| Collaborator Contribution | Sutro Biopharma are the world leaders in the use of cell free protein synthesis for the production of biologicals for thearupitic use at an industrial scale. Here at UCL we are working towards the development of a robust scalable platform for the production of Stratified medicines using Cell Free Protein Synthesis (CFPS). CFPS is a new area of research to us; in order to meet the agreed program objectives and timelines it was imperative for the UCL scientists to rapidly become profecient in the use of this system. Although there is significant amount of data/information published in this area most are from academic sources, and of somewhat limited application to us. Through this colaboration with Sutro Biopharma we were able to rapidly upskill our team members through hands on training methods training as well as acceleratino the organisational learning process through knowledge transfer. We have estimate that the knowledge and training gained through this secondment/colaboration has accelerated our process development timelines by 9 - 12 months. |
| Impact | We have estimate that the knowledge and training gained through this secondment/collaboration has accelerated our process development timelines by 9 - 12 months. Specifically, we have developed a scalable, robust method for the production of a key propriety reagent (extract), the insight gained have allowed a deeper understanding on possible additional genetic/molecular biology modifications/tweeks needed to improve process yields, and the knowledge and 'tricks' gained through this collaboration has enabeled us to develop 'trade secrets' which have directly led to higher process efficiencies/yields. |
| Start Year | 2017 |
| Description | TrakCel (FTHM Hub Partner) |
| Organisation | TrakCel |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £1000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1500 per day contributed Sponsored a user feasibility project in partnership with Imperial College (reported in a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | UCL-BIA User Feasibility Study: Economic evaluation of switching to a scalable GMP process for the manufacture of viral vectors at different stages of the drug development pathway |
| Organisation | BioIndustry Association |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | We (the FTHM hub) developed and linked the models (a CART, a Viral vector and a drug development model) to simulate the cost of goods, the cost of drug development and the net present value of in vivo and ex vivo therapies at different commercial demands. We interviewed other industrial specialist along with academics and clinicians to help us establish a representative drug development pathway and all associated costs except manufacturing costs which we simulated. We performed the simulations, the analysis and the visualisation of the results. |
| Collaborator Contribution | BIA and OXB proposed the context and the key research question for the user feasibility study. BIA and OXB helped to fine-tune the models, participate in teleconferences and sanity check the results. Contribution valued @£2000 per day. |
| Impact | • Evaluated the cost of drug development (i.e. process development, technology transfer, clinical manufacturing, etc.) and the profitability (net present value) of different therapies assuming a switch to a scalable process at different stages in the drug development pathway. • The analysis demonstrated the need for viral vector capacity using scalable manufacturing processes to supply clinical and commercial demand. The initial findings of this work have been used to support the ongoing development of a joint industry-academia business case that holistically illustrates the benefits of adopting future-proofed scalable viral vector manufacturing processes and platforms for the development of ATMPs. This work provides supporting data for a key deliverable from the advanced therapies workstream of the Medicines Manufacturing Industry Partnership (MMIP). Overall it will be used to further outline to policymakers and government the importance of investment and enabling policy work to secure a long term future of ATMP manufacture in the UK. The expertise of the UCL team, coupled with the use of objective modelling tools ensure that a robust set of data and findings are now available to support this important work. It also helps to generically illustrate the importance of investing in future-proofed scalable manufacturing processes and platforms for this group of important and emerging new medicines - the ATMPs - which are one of the fastest growing and most heavily invested-in areas of pharmaceutical development at the current time globally. |
| Start Year | 2019 |
| Description | UCL-BIA User Feasibility Study: Economic evaluation of switching to a scalable GMP process for the manufacture of viral vectors at different stages of the drug development pathway |
| Organisation | Oxford BioMedica UK Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | We (the FTHM hub) developed and linked the models (a CART, a Viral vector and a drug development model) to simulate the cost of goods, the cost of drug development and the net present value of in vivo and ex vivo therapies at different commercial demands. We interviewed other industrial specialist along with academics and clinicians to help us establish a representative drug development pathway and all associated costs except manufacturing costs which we simulated. We performed the simulations, the analysis and the visualisation of the results. |
| Collaborator Contribution | BIA and OXB proposed the context and the key research question for the user feasibility study. BIA and OXB helped to fine-tune the models, participate in teleconferences and sanity check the results. Contribution valued @£2000 per day. |
| Impact | • Evaluated the cost of drug development (i.e. process development, technology transfer, clinical manufacturing, etc.) and the profitability (net present value) of different therapies assuming a switch to a scalable process at different stages in the drug development pathway. • The analysis demonstrated the need for viral vector capacity using scalable manufacturing processes to supply clinical and commercial demand. The initial findings of this work have been used to support the ongoing development of a joint industry-academia business case that holistically illustrates the benefits of adopting future-proofed scalable viral vector manufacturing processes and platforms for the development of ATMPs. This work provides supporting data for a key deliverable from the advanced therapies workstream of the Medicines Manufacturing Industry Partnership (MMIP). Overall it will be used to further outline to policymakers and government the importance of investment and enabling policy work to secure a long term future of ATMP manufacture in the UK. The expertise of the UCL team, coupled with the use of objective modelling tools ensure that a robust set of data and findings are now available to support this important work. It also helps to generically illustrate the importance of investing in future-proofed scalable manufacturing processes and platforms for this group of important and emerging new medicines - the ATMPs - which are one of the fastest growing and most heavily invested-in areas of pharmaceutical development at the current time globally. |
| Start Year | 2019 |
| Description | UCL-Ipsen User Feasibility Study: Techno-economic evaluation of a cell-free synthesis system for the expression of highly potent recombinant proteins |
| Organisation | Ipsen |
| Country | Global |
| Sector | Private |
| PI Contribution | We (the FTHM hub) developed and the model to simulate the different process configurations relevant to Ipsen comparing their platform process to a cell-free synthesis process. We performed the simulations, the analysis and the visualisation of the results. |
| Collaborator Contribution | Ipsen proposed the context and the key research questions of the user feasibility study. Ipsen provided all necessary data to feed into the model. Ipsen validated the initial results and provided constructive feedback to help fine-tune the model and finalise the scenarios. Input from Ipsen staff valued at £1000 per day. |
| Impact | • Benchmarked the cost of goods (COG) of Ipsen's current manufacturing platform process. • Evaluated the COG using a cell-free synthesis process. • Evaluated in-house and outsourcing options for the supply of the necessary extract for the CFS process. • Evaluated the impact of alternative purification strategies on the COG (e.g. introduction of an affinity chromatography resin for the capture step that would also cause the removal of the intermediate purification chromatography step). • Evaluated and compared the cost-effectiveness and operational feasibility of the platform process and the CFS process for a multi-product facility at different dose sizes. |
| Start Year | 2019 |
| Description | UCL-Purolite User Feasibility Study: Bringing down Cost of Goods in mAb downstream processing |
| Organisation | Purolite International Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Provided the model for the analysis. Provided preliminary scenarios for analysis. |
| Collaborator Contribution | Provided the concept. Provided preliminary data. |
| Impact | Still active. |
| Start Year | 2020 |
| Description | UCL-Purolite User Feasibility Study: Bringing down Cost of Goods in mAb downstream processing |
| Organisation | Purolite International Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Provided the model for the analysis. Provided preliminary scenarios for analysis. |
| Collaborator Contribution | Provided the concept. Provided preliminary data. |
| Impact | Still active. |
| Start Year | 2020 |
| Description | UCL-Univercells User Feasibility Study: Economic evaluation of CFS in personalized medicine |
| Organisation | Univercells Ltd |
| Country | Belgium |
| Sector | Private |
| PI Contribution | Provided the model to perform the simulations. Designed the preliminary scenarios for analysis. |
| Collaborator Contribution | Provided the concept for evaluation. Provided preliminary data. |
| Impact | The project has been cancelled. |
| Start Year | 2020 |
| Description | UCL-Univercells User Feasibility Study: Economic evaluation of integrated mRNA vaccine platforms |
| Organisation | Univercells Ltd |
| Country | Belgium |
| Sector | Private |
| PI Contribution | Provided the model to simulate the different mRNA process flowsheets. Mapped out the mRNA process flowsheets. Gathered process and facility assumptions to populate the model. Formulated the scenarios for analysis. |
| Collaborator Contribution | Provided data on Univercells unit operations. Provided constructive feedback to help build the scenarios for analysis. |
| Impact | The study is still active. Once completed it is expected to provide insights in the following questions: • Cost of goods analysis on Univercells platform vs typical processes for mRNA vaccines. o What is the size and type of process equipment required in the Box? o What are the requirements in materials? o What are the key factors affecting the size and the materials requirements? o What are the key cost drivers? • Net present value analysis of different business models (i.e. from a vendors or an innovators perspective). o How reimbursement would work? There are 3 parties involved; the drug innovator, vendor and the hospital. What is the allocation of responsibilities and revenue? |
| Start Year | 2021 |
| Description | UCL-Univercells User Feasibility Study: Economic evaluation of scalable and adherent cell culture platforms for the manufacture of viral vectors |
| Organisation | Univercells Ltd |
| Country | Belgium |
| Sector | Private |
| PI Contribution | Provided the model to simulate the different LV and AAV process flowsheets. Mapped out the LV and AAV process flowsheets. Gathered process and facility assumptions to populate the model. Formulated the scenarios for analysis. |
| Collaborator Contribution | Provided data on Univercells unit operations. Provided constructive feedback to help build the scenarios for analysis. |
| Impact | The study is still active. Once completed it is expected to provide insights in the following questions: • Cost of goods analysis on Univercells adherent platform vs alternative adherent and suspension processes for lentiviral vectors. o What is the size and type of process equipment required in the platform? o What are the requirements in materials? o What are the key factors affecting the size and the materials requirements? o What are the key cost drivers? • Net present value analysis of manufacturing flowsheets. o Which flowsheet is more profitable? |
| Start Year | 2021 |
| Description | UFS - Historical batch analysis and trajectory optimisation for T-cell expansion process control |
| Organisation | Aglaris Ltd |
| Country | Spain |
| Sector | Private |
| PI Contribution | - Multivariate statistical data analysis of T-cell manufacturing data. Based on the historical data analysis, this study will seeked to optimise the trajectory (-ies) of one or more Critical Process Parameters (CPPs) found to have an important effect in product quality. These were carried out by a Design of Experiments (DoE) in collaboration with Aglaris. |
| Collaborator Contribution | Total value of £8,300 - Manufacturing data (e.g. yields, concentrations, measurable CPPs, etc.). - Feedback and technical support from the T-cell manufacturing team. - Application of the DoE strategy on the manufacturing site and the collection of further process data. Contribution based on input from Aglaris: Labour costs £4,000, materials £3,500, overheads (20% labour costs) £800 |
| Impact | In review - Multivariate statistical data analysis journal article indicating the ability of regression models to estimate product quality Multi-disciplinary collaboration: - Research expertise within the HUB focuses on Process Control at the School of Electrical and Electronic Engineering in The University of Manchester. - Reserach expertise within Aglaris focuses on Biomedical Engineering at the R&D System Automation Engineering in Aglaris Ltd. |
| Start Year | 2018 |
| Description | Univercells (FTHM Hub Partner) |
| Organisation | Univercells Ltd |
| Country | Belgium |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £2000 per year from 2019 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed Sponsored a user feasibility project in partnership with UCL(reported in a separate C&P record) |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2019 |
| Description | User Feasibility Study Mechanistic modelling for immunotherapy manufacture |
| Organisation | Cell and Gene Therapy Catapult |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Professor Rob Thomas' group in Loughborough is highly active in this area generating mechanistic models for industrial cell culture, initially focusing on erythroblast growth. A new direction for their group is applying the same methodologies for T cell cultures, with the hope to impact the lucrative immunotherapy industry. Loughborough contribution is to develop and apply the mechanistic modelling approach for adaptive process control of immunotherapy manufacture, to increase batch success, reduce final product variability as well as CoGs. |
| Collaborator Contribution | CGTC have a steady supply of donor apheresis material used to drive core projects funded through Innovate UK. This material in feed our exemplar CAR T immunotherapy process, and is subjected to deep characterization of the starting material as well as at various stages during processing; (LC-MS, Raman spectroscopy, broad spectrum flow cytometry etc.). CGT will supply characterisation data from their Core projects, to drive the mechanistic modelling-based control strategy development. Finally this strategy will be applied to CGT exemplar Centralised Control project where they have a PC-driven, bioprocess feedback control test-bed, to prove concept of mechanistic modelling-based adaptive control of immunotherapy manufacture. |
| Impact | not yet. |
| Start Year | 2019 |
| Description | User Feasibility Study with Albumedix |
| Organisation | Albumedix Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | We have been working with Albumedix to formulate nanoparticles and microparticles using their recombinant human serum albumin as a scaffold. We have been using the microparticles to solubilise poorly soluble small molecule drugs within the albumin scaffiolds. |
| Collaborator Contribution | Albumedix have provided the albumin required for the experiments. They have also offered the time of their scientists and we are carrying some stability studies in their laboratories. |
| Impact | None as yet. |
| Start Year | 2019 |
| Description | User Feasibility Study with Cancer Research UK |
| Organisation | Cancer Research UK |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | Bringing experience with cell-free synthesis and in-house developed protocols / materials for cell-free extract and reaction mix preparation. 3 months of FT from a post doctoral researcher (Beatrice Melinek). UCL have committed to continuing to collaborate with CRUK on this project (assuming a successful feasibility study). |
| Collaborator Contribution | CRUK originated the suggested area of collaboration. CRUK provided laboratory space / facilities for experimental work, adhoc technical input, conducted analytics, and will drive continued development of the technology to GMP / clinical trial stages (assuming a successful feasibility study). |
| Impact | Four possible strategies for cell-free pDNA production were derived. The below lists initial results to date: The PDRA has been installed and trained to work in the CRUK BDU process development environment. The benchmark cell-free pDNA production method (based on an existing patent) has been successfully replicated; One of the possible alternative strategies tested, showed successful pDNA amplification. An adaptation of the reaction to include use of a dialysis cassette substantially increased titres, such that few hundred microliters of reaction gives a yield equivalent to a 2 liter shake flask of cell-based pDNA production. CRUK developed their pDNA analytics capacities. The data will be used for a funding bid to RiHN's RDM grant. |
| Start Year | 2019 |
| Description | User Feasibility Study with FujiFilm Diosynth |
| Organisation | Fujifilm |
| Department | Fujifilm Diosynth Biotechnologies, UK |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | 3 months of a PhD candidate's (Noelle Colant's) time. Using the CFS platform developed in-house at UCL to trial expression of "difficult to express" plasmids at Fujifilm Diosynth, and applying in-house developed expertise to troubleshoot. |
| Collaborator Contribution | Access to facilities for conducting experiments. Use of in-house plasmids. Some experimentation towards troubleshooting. |
| Impact | Identification of an inappropriate plasmid purification kit, which is incompatible with cell-free. |
| Start Year | 2019 |
| Description | User Feasibility Study with West Pharma - Performance Comparability of Cyclic Olefin Polymer Vials and Ethylene Vinyl Acetate Bags for Storage of T Cells |
| Organisation | West Pharmaceutical Services |
| Country | United States |
| Sector | Private |
| PI Contribution | This user feasibility study allowed us (the FTHM Hub) to undertake novel bioprocessing research investigating the comparability of using cryovials for the endstage cryopreservation versus cryobags. Experimental work has been completed for the study. Access to the expertise and cell material of UCL through the Hub has enabled the following key learnings: 1) The data showed similar recovery of T cells, in terms of viability, growth kinetics, immunophenotype and cell exhaustion, whether stored in vials or bags. This highlights that rigid vials may be a suitable container for cell therapies that can be used without the risks of dead volume and breakage that accompanies storage in bags. 2) While the thermodynamic behavior of the two vessels is quite different, with vials taking an average of 6x longer to thaw than bags, the fundamental cell characteristics evaluated do not appear to be affected by the extended warming. If confirmed, this enhanced stability in a rigid vial could be shown to be advantageous for cell therapy users struggling with handling, transport, or cold chain logistics by providing a longer time window to transfer between processing steps. |
| Collaborator Contribution | West Pharmaceutical Services provided access to consumables and the cyclic olefin vials used for the study. They also provided the context and key research questions for this study and, through regular consultations, ensured that the research questions and experimental goals remained on track. |
| Impact | Presentations at major national and international conferences. A white paper on the use of vials vs cryobags and adoption of protocols and processes internally within West Pharmaceutical Services. |
| Start Year | 2019 |
| Description | User feasibility study with Albumedix |
| Organisation | Albumedix Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Mathematical model to assess the impact of cryopreservation on the supply chain. |
| Collaborator Contribution | Expert knowledge, research questions and data equal to 2,000GBP provided in kind. |
| Impact | Beta versions of the mathematical models & preliminary results on supply chain flexibility/resilience. |
| Start Year | 2020 |
| Description | User feasibility study with TrakCel |
| Organisation | TrakCel |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Identification of bottlenecks, challenges and opportunities of the current supply chain network are identified and addressed. Alternative solutions towards time & cost minimisation, risk mitigation and seamless scalability will be proposed in the space of clinical and/or manufacturing site distribution. Development, simulation and assessment of the following Mixed Integer Linear Programming Problems: • Classic supply chain structure (hospital-manufacturing-hospital) • Outsourcing versus in-house QC analysis • Potential of intermediate storage/cryopreservation facilties |
| Collaborator Contribution | Expert insight, data sets and results assessment. Trakcel helped to develop the initial proposal with its key research questions. During the study, they provided datasets and contributed with their Expert insight and assessment of resullts to the fine-tuning of the model. Input from Trakcel valued at £1500 per day [total of £12000]. |
| Impact | 1. Moschou D. Papathanasiou M.M, Lakelin M., Shah, N., "Investment Planning in Personalised Medicine", European Symposium on Computer Aided Process Engineering (ESCAPE) 30 2. Papathanasiou M.M, Stamatis C. Lakelin M, Farid S., Tichener-Hooker N., Shah N., "Autologous CAR T-cell therapies supply chain: Challenges and Opportunities?", l submitted at Cancer Gene Therapy Nature 3. Papathanasiou M.M, Lakelin M., Shah N., "Enabling Precision Healthcare through Patient-Centric Supply Chain Networks", AIChE Annual Meeting 2019 |
| Start Year | 2018 |
| Description | WestPharma (FTHM Hub Partner) |
| Organisation | West Pharmaceutical Services |
| Country | United States |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Annual cash contribution: £15,000 per year from 2017 Provision of advice and guidance on the overall direction and progress of the Hub, via participation in Hub meetings and working groups, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | Wyatt UK (FTHM Hub Partner) |
| Organisation | Wyatt Technology Corporation |
| Department | Wyatt Technology UK Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of input to the Hub as an original core partner, valued @£1000 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to society |
| Start Year | 2017 |
| Description | deltaDOT (FTHM Hub Partner) |
| Organisation | deltaDOT Ltd |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | Collaborating Organisation and Partnership under the Future Targeted Healthcare Manufacturing Hub |
| Collaborator Contribution | Provision of advice and guidance on the overall direction and progress of the Hub, via participationat Hub meetings and working groups, valued @£1500 per day contributed |
| Impact | Working within a network of users and academics to deliver the vision: to address the manufacturing, business and regulatory challenges to ensure that new targeted biological medicines can be developed quickly and manufactured at a cost affordable to societ |
| Start Year | 2017 |
| Company Name | Roxijen Limited |
| Description | |
| Year Established | 2020 |
| Impact | none yet |
| Company Name | SAFI Biosolutions UK Limited |
| Description | |
| Year Established | 2021 |
| Impact | Established an economic manufacturing platform for therapeutic blood products |
| Company Name | Young OWL Microfluidics Limited |
| Description | |
| Year Established | 2021 |
| Impact | N/A; recently formed |
| Description | 1-Day Teaching Seminar in Microfluidics |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Postgraduate students |
| Results and Impact | Together with an industrial partner in microfabrication, I taught a one day seminar in microfluidics ahead of the Lab on Chip conference in Mumbai, India, 2018. |
| Year(s) Of Engagement Activity | 2018 |
| URL | https://selectbiosciences.com/conferences/index.aspx?conf=MLOAC18 |
| Description | 1st International Training Course - Application of microbioreactors (MBR) in bioprocess development. September 24-28, 2018, TU Braunschweig, Germany http://www.eurombr.nu/ |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Postgraduate students |
| Results and Impact | 5-day training course with lecture programme and laboratory hands-on Topics: - Microfabrication - Microfluidics, Transport phenoma, Fluid flow - Sensors and Inline-Analytics - Enzyme immobilisation - Biocatalysis - Cultivation - Modeling and Design of microfluidic processes |
| Year(s) Of Engagement Activity | 2018 |
| URL | http://www.eurombr.nu/ |
| Description | A specialist working group (SWG) on cell free synthesis (CFS). |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Professional Practitioners |
| Results and Impact | A specialist working group (SWG) on cell free synthesis (CFS). The group meets twice a year to discuss the challenges of using CFS as a platform for biotherapeutic manufacture to meet the needs of targeted healthcare. This is an important activity within the EPSRC Future Targeted Healthcare Manufacturing hub (https://www.ucl.ac.uk/biochemical-engineering/research/hub). Bringing together supplier and user companies to consider the challenges and possibilities in the area. The scope is from the experimental challenges to costing and regulatory issues. |
| Year(s) Of Engagement Activity | 2017,2018,2019 |
| Description | AIChE 2022 annual meeting |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Oral presentation given at AIChE 2022 annual meeting. |
| Year(s) Of Engagement Activity | 2022 |
| Description | AIChE Annual Meeting 2020 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Presentation at the AIChE Annual Meeting 2020 |
| Year(s) Of Engagement Activity | 2020 |
| Description | AN ODE MODEL FOR OPTIMAL OPERATION OF T-CELL CULTURE IN SUSPENSION SYSTEM |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | My talk at WATRMC covered the results of our Quality by Design approach to process control of cell therapy manufacture that addresses autologous cell therapies variability in patient-specific source material through first principles models.. Ambr15 platform was used to validate the ODE model with respect to input variability including; cell seeding density, feeding ration, frequency and glucose concentration in bulk medium. Our results demonstrated a simple unstructured model of T-cell growth in a stirred tank system which provides framework for operation with respect to timings and volume of medium delivery to maintain an uninhibited growth. Such a model can also indicate how glucose delivery can be controlled to sustain cell growth while selectively affect the growth of specific sub-population of T cells. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Application of Digital Twins and Advanced Process Control to Improve Manufacture of Targeted Healthcare Products - Webinar session in partnership with Perceptive Engineering |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | FTHM Hub Webinar Session in partnership with Perceptive Engineering. Delivered information and generated discussion and debate on topic of emerging sector interest. 55 Registrations, 36 Attendees. |
| Year(s) Of Engagement Activity | 2020 |
| Description | BioMAN 2019 Summit. Technology and Innovation for Sustainable Manufacturing of Advanced Therapy Medicinal Products. Cambridge USA, 11-12 December 2019 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | The Summit was related to Technology and Innovation for Sustainable Manufacturing of Advanced Therapy Medicinal Products. It was orginised by MIT Centre for Biomedical Innovation. Top people from industry and academia (including Harvard University and MIT as a majour players on this Summit) discussed the current state and future directions of Manufacturing of Advanced Therapy Medicinal Products. Presenting a poster "Label-free recognition of non-activated and activated human T cells by Quantative Phase Imging" and further overall discussions over non-invasive methods for cell Quality Control of manufacturing of safe and potent immunotherapeutic cell products. A particular interest was a presentation of newly created SMART-CAMP MIT/Singapour Interdisciplinary research group regarding their activities in developing of Critical Analytics for manufacturing of Cell Therapy products. |
| Year(s) Of Engagement Activity | 2019 |
| Description | BioProNet Bioprocess Intensification Symposium |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | BioProNET Bioprocess Intensification Symposium 4th July 2018 The continued growth of the biopharmaceutical industry is being challenged by a greater number and variety of products than in the past. The industry is also increasingly concerned with the affordability of these products. These key factors means process intensification has become a critical objective. The goal is higher productivity processes to enable small process trains, resulting in cost-effective, lean, and agile manufacturing facilities. The symposium assembles 8 leaders from the biopharmaceutical industry and academia who will present their latest findings and opinions on the subject. By bringing together scientists and engineers from academia and industry who are actively engaged in bioprocess intensification the event will provide forum for lively debate. |
| Year(s) Of Engagement Activity | 2018 |
| URL | http://biopronetuk.org/biopronet-funded-collaboration-building-workshops/ |
| Description | Bioprocess Asia Conference |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | BPA is an international conference and I am part of the Scientific Advisory Committee |
| Year(s) Of Engagement Activity | 2018 |
| Description | Bioprocessing Skills School (STARS) |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | Ran half-day session entitled "Innovative therapies but what about the societal and economic implications", introducing health economic concepts and thinking to participants. |
| Year(s) Of Engagement Activity | 2017 |
| Description | CGTI and BILS summit 2022 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Oral presentation at the Cell and Gene Therapies Innovation (CGTI) summit. Attended the Bio Innovation Leaders summit (BILS). |
| Year(s) Of Engagement Activity | 2022 |
| Description | CGTI and BILS summit 2023 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Attended CGTI and BILS summit. Engaged with industrial representatives of the cell and gene therapy space. |
| Year(s) Of Engagement Activity | 2023 |
| Description | Cell and Gene Innovation Summit 2020. Liders Inspire action. Berlin 5-6 February 2020 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | The Summit was related to Innovations in Cell and Gene Therapy. Top people from industry and academia presented and discussed different topics in Cell and Gene Therapy with particular focus on innovations. As a speaker of the Summit I presented a 30-minutes talk on a topic "Could key cell attributes be defined and monitored label-free for Quality Control of manufacturing a safe and potent immunotherapeutic cell products". Topis covered included application of Quantative Phase Imaging and Raman spectroscopy as analytical methods for label free cell recognition, detection of non-activated and activated human T cells by Phase Imaging on a chip, monitoring of viral loading in cells by Quantative Phase Imaging. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Cell-free roadmap |
| Form Of Engagement Activity | A magazine, newsletter or online publication |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Based on a series of workshops and input from users at the CFPS SWG a white paper was produced, published in Bioprocess International, on the subject of the technical hurdles to cell-free distributed manufacture. The aim of publishing in a trade magazine was to widen the awareness of cell-free synthesis and its potential applications, and encourage wider contribution to the debate on how the future development of this technology might look. This article was further presented to the USC, followed by questions and comments. Was awarded runner up in BPI Reader's Choice Awards. |
| Year(s) Of Engagement Activity | 2020 |
| URL | https://bioprocessintl.com/upstream-processing/expression-platforms/toward-a-roadmap-for-cell-free-s... |
| Description | Chair for the Young Members Forum of London and South East Coast IChemE |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Professional Practitioners |
| Results and Impact | Since October 2018 I am chairing the Young Members Forum of London and South East Coast IChemE organisign events that are related and targeted to: - Undergraduate Chemical Engineering students - Postgraduate Chemical Engineering students - Chemical Engineering professionals I am actively involved in initiating and supporting networking activities, educative pilot plant tours and social activities amongst the group members and beyond. |
| Year(s) Of Engagement Activity | 2018,2019 |
| Description | Clarke Society Panel Discussion: ''Responsible' Innovation; True or Gimmick? |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Professional Practitioners |
| Results and Impact | The UCL postdoctoral society of Biochemical Engineering hosted a series of events entitled 'An Afternoon of Innovation' to inform, inspire and celebrate innovation of all disciplines. This was funded by a EPSRC IAA Knowledge Exchange fund. The event on the 15th Nov 2019 looked at innovation from the perspective of 'True' Innovation and the Science-Society Interface. The event was open to UCL and the Public. The keynote speaker for this event was Mr David Rowan (Founding Editor-In-Chief of WIRED UK). The panel discussion focused on the concept of 'Responsible Innovation' and entitled - ''Responsible' Innovation; True or Gimmick? - Driving Innovation towards Societal Good.' The Panel host was Mr Jim Dawton (Director at Impeller Ventures). Paul Dalby was one of the four panel members. |
| Year(s) Of Engagement Activity | 2019 |
| Description | Co-chair at AIChE Annual Meeting 2019 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Chaired the Process Intensification through Process Systems Engineering session ( CAST Division (10A05)) at the AIChE Annual Meeting 2019 |
| Year(s) Of Engagement Activity | 2019 |
| URL | https://www.aiche.org/conferences/aiche-annual-meeting/2019/proceeding/session/process-intensificati... |
| Description | Commercialization of ATMPs |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Postgraduate students |
| Results and Impact | 2 hour Lecture on Regenerative Medicine Commercialisation and Variability- using flow cytometry as an example. Tours of facility foloowed by Design Exercise worshop on assessment of the feasibility for the company to move into cell therapy manufacture. Each team assessed the feasibility of producing either an allogeneic or autologous therapy at different doses and provided recommendation on the upstream cell culture technology. The session included case studies and group discussion. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Conference Presentation "Compartmentalisation of Transcription and Translation in Cell-Free Protein Synthesis Using a Membrane Reactor Design" |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | An in person presentation for the conference PEGS Europe "Compartmentalisation of Transcription and Translation in Cell-Free Protein Synthesis Using a Membrane Reactor Design" |
| Year(s) Of Engagement Activity | 2022 |
| Description | Decisional Tools Specialist Working Group |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | The Decisional Tools Specialist Working Group (SWG) has met x2 a year since the Hub launch, The SWG discusses the use of Hub Decisional Tools to aid decision-making in the development, manufacture and commercialisation of autologous cell therapies and stratified therapies and includes invited user expert presentations. The aim of this specialist working group meeting is to: Assess whether decisional tools research plans are addressing the key challenges within stratified protein therapies and personalised cell therapies. Identify any emerging areas, or current weaknesses in the proposal. Identify key materials and resources required for the projects proposed. Evaluate potential risks to the success of the decisional tools workpackages within the Hub. |
| Year(s) Of Engagement Activity | 2017,2018 |
| Description | Developing therapeutic monoclonal antibodies for COVID-19 at pandemic speed - Webinar session in partnership with Vir Biotechnology |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Shared information and generated discussion and debate around a topic of significant international importance. Registrations 113, Attendees 72 |
| Year(s) Of Engagement Activity | 2020 |
| Description | Dolomite News |
| Form Of Engagement Activity | A magazine, newsletter or online publication |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Featured in dolomites news article detailing the application of microfluidics for the synthesis of nano and microparticles to augment therapeutic delivery and biosensing. |
| Year(s) Of Engagement Activity | 2019 |
| URL | https://www.dolomite-microfluidics.com/news/fluorescent-biosensors-as-tools-for-drug-therapeutics/ |
| Description | ESCAPE 30 Conference |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Presentation at the virtual 30th European Symposium on Computer Aided Process Engineering |
| Year(s) Of Engagement Activity | 2020 |
| Description | ESCAPE32 conference |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Poster presentation at the ESCAPE 32 conference. |
| Year(s) Of Engagement Activity | 2022 |
| Description | Exploring the shift towards point-of-care and decentralised manufacturing |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Participation in roundtable at the Advanced Therapies Europe conference. Royal Lancaster, London, UK |
| Year(s) Of Engagement Activity | 2022 |
| Description | FTHM Hub ATMP Manufacturing, Regulation and Reimbursement Specialist Working Group, 29th September 2022 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Third sector organisations |
| Results and Impact | Updates on 'Cost-effectiveness, Reimbursement and Value of Cell Therapies'. |
| Year(s) Of Engagement Activity | 2022 |
| Description | FTHM Hub Bi-annual Newsletter (digital) |
| Form Of Engagement Activity | A magazine, newsletter or online publication |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Connecting with the FTHM Hub partner community and wider stakeholders to inform them about various news items, e.g. team news, publications, user news, feasibility study project news, recent meetings and events, forthcoming meetings and events, policy impact news. |
| Year(s) Of Engagement Activity | 2017,2018,2019,2020,2021,2022 |
| URL | https://mailchi.mp/0c0f5fd887a8/future-targeted-healthcare-manufacturing-hub-newsletter-feb2022 |
| Description | FTHM Hub LinkedIn Group |
| Form Of Engagement Activity | Engagement focused website, blog or social media channel |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | A private LinkedIn group to connect group members with news and updates from the team and activities. |
| Year(s) Of Engagement Activity | 2017,2018,2019,2020,2021,2022 |
| URL | https://www.linkedin.com/groups/4629426/ |
| Description | FTHM Hub Session at the World Advanced Therapy Congress |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | On behalf of the FTHM Hub, Dr Rafiq organised two dedicated sessions at the World Advanced Therapy Congress Meeting in May 2019. This included 3 talks from FTHM Hub users as part of a "Future Leaders" session, including Rhian Carter from Oxford BioMedica and Emma Chan from Autolus, and also organised a specific session on Hub research which included presentations from Hub Researchers and Investigators. Included presentations for Hub researchers and users (Autolus, CRUK, Oxford BioMedica). |
| Year(s) Of Engagement Activity | 2019 |
| Description | FTHM Hub Twitter Feed @FutureHealthHub |
| Form Of Engagement Activity | Engagement focused website, blog or social media channel |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Public/other audiences |
| Results and Impact | News, updates and brief information about the Hub (e.g. team, articles, events, associated interest) to promote the Hub in the public domain, both nationally and internationally |
| Year(s) Of Engagement Activity | 2017,2018,2019,2020,2021 |
| URL | http://www.twitter.com/futurehealthhub |
| Description | FTHM Hub Website |
| Form Of Engagement Activity | Engagement focused website, blog or social media channel |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Public/other audiences |
| Results and Impact | A public facing website informing people about what the FTHM Hub is and information on elements of the project e.g. vision and mission, the academic team, industry and sector partners, feasibility study collaboration mechanisms, events schedule. Hosts an additional page that provides Hub Users with project updates and outputs (e.g. meeting minutes, slide packs, reports) |
| Year(s) Of Engagement Activity | 2017,2018,2019,2020,2021,2022 |
| URL | https://www.ucl.ac.uk/biochemical-engineering/research/research-and-training-centres/future-targeted... |
| Description | FTHM Hub/NHS England Meeting - Industrial Economics, 14th January 2020 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Other audiences |
| Results and Impact | Follow-up meeting with NHS England Medicines Analysis Strategy and Policy Team - Summary and Future Interactions Presentation given to the Hub by NHS England on the challenges when deciding what to pay for a new treatment? Conclusion: Health Opportunity Cost is a vital consideration for HTA and society. Nishma Patel to liaise with John Spoors to discuss future collaborations. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Formulation Specialist Working Group |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The formulation Specialist Working Group meets every 6 months (twice in 2017, once in 2018 so far) to enable industry, regulatory agency, government laboratory and academic experts to discuss the arising challenges, and potential solutions on a 5-10 year horizon, in the area of formulation for stratified proteins, and cell and gene therapies. Over 30 different users have attended to date, and the meetings have refined the Hub research agenda, scoped out topics for review articles, and engaged the regulatory agencies which raises their awareness of future challenges that they will also need to address. |
| Year(s) Of Engagement Activity | 2017 |
| Description | Formulation Specialist Working Group - 2018 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Professional Practitioners |
| Results and Impact | 1. Assess the current second level plan for formulation work proposed within the Hub, in terms of whether it is addressing the key challenges within stratified protein therapy. 2. Identify any emerging areas, or current weaknesses in the proposal. 3. Identify key materials and resources required for the project proposed. 4. Evaluate potential risks to the success of the formulation workpackages within the Hub. |
| Year(s) Of Engagement Activity | 2018 |
| Description | Future Food Formulations Factories |
| Form Of Engagement Activity | A magazine, newsletter or online publication |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Schools |
| Results and Impact | University of Nottingham Student Magazine Article. With the advent of nanotechnology and the food industry looking for novel applications to maximise the benefits of food nutrition, there have been key advancements in the nutraceutical market. The researchers at the University of Nottingham's School of Pharmacy have an in depth understanding of the fundamental formulation parameters. We are now enhancing particle formulation by preparing custom designed polymers. These polymers permit targeted delivery of therapeutics and nutraceuticals to specific cellular systems. We believe that the research being conducted at our school is paving the way to transform food security of an ever-growing world population with a limited number of resources |
| Year(s) Of Engagement Activity | 2018 |
| URL | https://impactnottingham.com/2018/04/future-food-factories/ |
| Description | Health Economics Workshop, 12th February 2019 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Other audiences |
| Results and Impact | 1/2 day course 1) Introduction to economic evaluation in health care (Steve Morris) 2) How to conduct an economic evaluation (Nishma Patel) 3) Case study - highly specialised technologies (Nishma Patel) |
| Year(s) Of Engagement Activity | 2018,2019 |
| Description | Healthcare Specialist Working Group |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Policymakers/politicians |
| Results and Impact | Healthcare Specialist Working Group involving round table discussions with health care providers, policy makers and industry about reimbursement of personalised cell therapies and stratified medicines. |
| Year(s) Of Engagement Activity | 2017,2018 |
| Description | Healthcare Specialist Working Group, 10th May 2019 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | This meeting explored the future direction for the Healthcare SWG: • How do managed access agreements (MAA) unlock access to highly specialised technologies (HST)? What do we know about how expensive they are to administer? Who bears the cost? • What changes to manufacturing processes for cell and gene therapies will affect the costs to the NHS and benefits to patients of these therapies? What are the likely pathways to impact? • For CAR-T cell therapy to extend beyond early clinical phase one trials, cryopreservation is essential. How will this impact on the NHS? What are the likely costs and benefits? |
| Year(s) Of Engagement Activity | 2019 |
| Description | Healthcare Specialist Working Group, 11th October 2019 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | The objective of this meeting were to understand how real-world data is accessed or generated and used to produce or generate real-world evidence (registries) See whether use is aligned with international standards. Particpants explored the core principles that govern how real-world data and how the data could be credibly used, with the potential to developing 'international standards' to support decisions in healthcare systems. Key message were the how existing databases could be updated for the purposes of OBR is possible, or through part automation. However, it were evident that this approach were fragmented and costly. The participants explored pros and cons of building a new system, or integrating existing sources into a new platform. |
| Year(s) Of Engagement Activity | 2019 |
| Description | Healthcare Specialist Working Group, 16th October 2018 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The focus of this meeting was on the challenges in obtaining evidence for regulatory approval and reimbursement of cell and gene therapies. The National Institute for Health and Care Excellence (NICE) rejected the use of Novartis's CAR-T therapy Kymriah in adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in the NHS. The draft guidance concluded that Kymriah was not cost-effective and highlighted concerns about the data and evidence received. Similarly, Gilead's CAR-T therapy Yescarta (axicabtagene ciloleucel) for diffuse large B-cell lymphoma and primary mediastinal B-cell lymphoma was also rejected. What were the driving factors behind these rejections? What implications do they have for how we measure the cost-effectiveness of cell and gene therapies? Roundtable discussion on healthcare reimbursement issues: 1. What are the expectations of regulators and payers for data and evidence in approval and coverage decisions? 2. How can reimbursement decisions about targeted healthcare be handled to accelerate adoption of targeted healthcare? 3. When treatments are recommended for use in the NHS by NICE, commissioners are legally required to provide patients with access to these medicines within three months of the positive recommendation. What barriers to implementing the recommendations should be expected and how can these be overcome? 4. Economic evaluations are conducted to assess the cost-effectiveness of targeted health care. How should these barriers be incorporated in the evaluation? |
| Year(s) Of Engagement Activity | 2018 |
| Description | Healthcare Specialist Working Group, 1st November 2017 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | In this SWG we aimed at sharing knowledge, experience of different stakeholder perspectives in order to facilitate the progress of the integration of marketing approval and reimbursement. Roundtable discussion on healthcare reimbursement issues: 1. What are the main challenges in the reimbursement of personalised cell therapies and stratified medicines? 2. What models of drug pricing and reimbursement strategies are currently used and are likely to be used in future for personalised cell therapies and in stratified medicine? 3. What implications does this have for manufacturing? 4. What research questions should the Hub be addressing |
| Year(s) Of Engagement Activity | 2017 |
| Description | Healthcare Specialist Working Group, 25th April 2018 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The second Healthcare Specialist Working Group explores current realities and future challenges and opportunities in the areas of reimbursement and regulation of targeted proteins and cell-based therapies. The working group are introduced by a summary of the outcomes of the previous meeting and of further issues suggested by the work of the two postdoctoral researchers working on reimbursement and regulations. Roundtable discussion on healthcare reimbursement issues: 1. What are the expectations of reimbursement agencies and payers for data and evidence in approval and coverage decisions for targeted health care, and how and why should these differ from other innovations? 2. Once a product has been authorised, what are the costs of collecting evidence required for adaptive or conditional regulatory approvals? Who bears these costs? 3. When targeted health care innovations are recommended for use in the NHS what barriers are typically faced to implement them? How can these be overcome? 4. Economic evaluations are conducted to assess the cost-effectiveness of tar |
| Year(s) Of Engagement Activity | 2018 |
| Description | Hub presentation to the MHRA Innovation Office |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | Hub researchers provided an overview of research key findings to members of the Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Office and discussed future collaborative activities. As a result Hub invited to participate in an MHRA workshop on point-of-care manufacture regulatory frameworks. |
| Year(s) Of Engagement Activity | 2018,2020 |
| Description | Industry-Academia Networking Event on Application of Artificial Intelligence & Big Data for Early Cancer Detection |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | The activity was related to meet people from Academia and from Industry on Application of AI and Big Data. It is annual event international event on front edge techniques like AI/Big Data used in Medicine and in Academia research for Early Cancer Detection. I was there to understand potential of Quantative Phase Microscopy combined with mashine learning techniques for early cancer detection and the routes how to build up AI apropriate algorithms. |
| Year(s) Of Engagement Activity | 2019 |
| Description | International Health Economics Association (iHEA) Cost-Effectiveness of Chimeric Antigen Receptor (CAR) T-Cell Therapies for Blood Cancers: A Systematic Review, 8 - 12 July 2023 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Third sector organisations |
| Results and Impact | Cost-effectiveness of Chimeric Antigen Receptor (CAR) T-cell Therapies for Blood Cancers: A Systematic Review Patel N1, Farid S2, Gomes M1 1Department of Applied Health Research, University College London, London, United Kingdom 2Farid S. Department of Biochemical Engineering, University College London, United Kingdom Background: Chimeric antigen receptor (CAR) T-cell therapy is an area of rapid development, showing the promise of curing blood cancers. Since 2018, six CAR T-cell therapy products have been granted recommendation for use in patients with (i) B-cell acute lymphoblastic leukaemia (ALL) (ii) diffuse large B-cell lymphoma (DLBCL) (iii) mantle cell lymphoma and (iv) multiple myeloma. Implementation of these products includes complex and novel engineering, manufacturing, and delivery processes, which means very high costs per patient and a threat to the sustainability of healthcare systems. While health gains may justify such high costs, it is currently unclear the extent to which the overall cost-effectiveness of these therapies is determined by the therapy cost, magnitude of health benefits or cost-effectiveness threshold. Aim: This review aims to review existing evidence on the cost-effectiveness of the six CAR T-cell therapies across different international jurisdictions. Methods: We conducted a systematic review using PubMed, Scopus and Web of Science databases to identify economic evaluations published until 29 November 2022. The methodological quality of each study was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS), 2022 edition. Studies were included if they were full economic evaluations (within-trial and model-based). Abstracts, non-incremental cost, editorials, redacted reports, reviews, and budget impact studies were excluded from the review. Extracted data was grouped according to type of CAR T-cell therapy and summarised narratively. All estimates of cost and cost-effectiveness were converted to current prices, applying the US exchange rate (November 2022). Results: The review included 29 full cost-effectiveness studies: tisagenlecleucel (n = 14), axicabtagene ciloleucel (n = 6), axicabtagene ciloleucel with tisagenlecleucel (n = 3), brexucabtagene autoleucel (n = 4), and lisocabtagene maraleucel (n = 2). Incremental costs varied considerably between $US1407 and $US606,010, whereas QALYs gained ranged from 0.81 to 10.77 over a lifetime horizon. The highest incremental QALY was reported for tisagenlecleucel in the Netherlands, Singapore and Spain; 10.77, 9.87 and 8.97 for use in paediatric, relapsed/refractory B-cell acute lymphoblastic leukaemia (ALL). The highest incremental cost ($US606,010) was associated with the use of axicabtagene ciloleucel therapy in Canada for diffuse large B-cell lymphoma (DLBCL) in Canada and US (US$439,500), and brexucabtagene autoleucel for mantle cell lymphoma in the UK (US$367,423). The main cost-driver of the total cost were attributed to the price of the CAR T-cell therapy. Cost-per-QALY ratios ranged from US$2,306 to US$1,615,000, resulting in a probability of being cost-effective between 16% and 100%, given reported willingness to pay thresholds between US$46,598 and US$423,000. Quality assessment showed that 95% of the studies were of good quality according to CHEERS checklist. Conclusions: To our knowledge, this is the most up-to-date literature review on the cost-effectiveness of existing CAR T-cell therapies. It finds uncertainty was not limited to evidence generation but extended to the affordability of CAR T-cell therapies. Existing reimbursement models for CAR T-cell therapies are not fit for propose. This review highlights the need for robust evidence to address considerable uncertainty in the cost and effectiveness data given the magnitude of differences in cost-effectiveness estimates. |
| Year(s) Of Engagement Activity | 2023 |
| Description | International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Cost-Effectiveness of Chimeric Antigen Receptor (CAR) T-Cell Therapies for Blood Cancers: A Systematic Review, 7 - 10 May 2023 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Third sector organisations |
| Results and Impact | Cost-effectiveness of Chimeric Antigen Receptor (CAR) T-cell Therapies for Blood Cancers: A Systematic Review Patel N1, Farid S2, Gomes M1 1Department of Applied Health Research, University College London, London, United Kingdom 2Farid S. Department of Biochemical Engineering, University College London, United Kingdom Background: Chimeric antigen receptor (CAR) T-cell therapy is an area of rapid development, showing the promise of curing blood cancers. Since 2018, six CAR T-cell therapy products have been granted recommendation for use in patients with (i) B-cell acute lymphoblastic leukaemia (ALL) (ii) diffuse large B-cell lymphoma (DLBCL) (iii) mantle cell lymphoma and (iv) multiple myeloma. Implementation of these products includes complex and novel engineering, manufacturing, and delivery processes, which means very high costs per patient and a threat to the sustainability of healthcare systems. While health gains may justify such high costs, it is currently unclear the extent to which the overall cost-effectiveness of these therapies is determined by the therapy cost, magnitude of health benefits or cost-effectiveness threshold. Aim: This review aims to review existing evidence on the cost-effectiveness of the six CAR T-cell therapies across different international jurisdictions. Methods: We conducted a systematic review using PubMed, Scopus and Web of Science databases to identify economic evaluations published until 29 November 2022. The methodological quality of each study was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS), 2022 edition. Studies were included if they were full economic evaluations (within-trial and model-based). Abstracts, non-incremental cost, editorials, redacted reports, reviews, and budget impact studies were excluded from the review. Extracted data was grouped according to type of CAR T-cell therapy and summarised narratively. All estimates of cost and cost-effectiveness were converted to current prices, applying the US exchange rate (November 2022). Results: The review included 29 full cost-effectiveness studies: tisagenlecleucel (n = 14), axicabtagene ciloleucel (n = 6), axicabtagene ciloleucel with tisagenlecleucel (n = 3), brexucabtagene autoleucel (n = 4), and lisocabtagene maraleucel (n = 2). Incremental costs varied considerably between $US1407 and $US606,010, whereas QALYs gained ranged from 0.81 to 10.77 over a lifetime horizon. The highest incremental QALY was reported for tisagenlecleucel in the Netherlands, Singapore and Spain; 10.77, 9.87 and 8.97 for use in paediatric, relapsed/refractory B-cell acute lymphoblastic leukaemia (ALL). The highest incremental cost ($US606,010) was associated with the use of axicabtagene ciloleucel therapy in Canada for diffuse large B-cell lymphoma (DLBCL) in Canada and US (US$439,500), and brexucabtagene autoleucel for mantle cell lymphoma in the UK (US$367,423). The main cost-driver of the total cost were attributed to the price of the CAR T-cell therapy. Cost-per-QALY ratios ranged from US$2,306 to US$1,615,000, resulting in a probability of being cost-effective between 16% and 100%, given reported willingness to pay thresholds between US$46,598 and US$423,000. Quality assessment showed that 95% of the studies were of good quality according to CHEERS checklist. Conclusions: To our knowledge, this is the most up-to-date literature review on the cost-effectiveness of existing CAR T-cell therapies. It finds uncertainty was not limited to evidence generation but extended to the affordability of CAR T-cell therapies. Existing reimbursement models for CAR T-cell therapies are not fit for propose. This review highlights the need for robust evidence to address considerable uncertainty in the cost and effectiveness data given the magnitude of differences in cost-effectiveness estimates. |
| Year(s) Of Engagement Activity | 2023 |
| Description | Invited Interview |
| Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | "Advances in enabling smart technologies across the cell therapy supply chain", Cell and Gene Therapy Insights |
| Year(s) Of Engagement Activity | 2018 |
| URL | https://insights.bio/cell-and-gene-therapy-insights/journal/articles/advances-in-enabling-smart-tech... |
| Description | Invited Seminar |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Invited seminar at the Texas A&M Energy Institute with title "Pharma 4.0: Advancing the current state of the art through Process Systems Engineering approaches". More than 50 people attended, coming from all academic levels (UG to faculty), as well as a few industrial practitioners. Most of the interested was raised around the unique challenges faced in the design and optimisation of the supply chain of CAR T cell therapies. My talk initiated further conversations with members of the faculty and I am now exploring how those can move forward. |
| Year(s) Of Engagement Activity | 2018 |
| URL | https://energy.tamu.edu/events/energy-institute-lecture-series-dr-maria-m-papathanasiou/ |
| Description | Joint UCL and Cancer Research UK Technology Showcase |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | CRUK-Lonza Technology Showcase: Dr Qasim Rafiq, as part of the Future Targeted Healthcare Manufacturing Hub, organised and hosted an event for Hub users to showcase a novel technology developed by Hub Partner (Lonza) for personalised therapy manufacture prior to the general launch. This was the launch of their Cocoon system for manufacturing of autologous therapies and over 60 industrial, commercial and postgraduate users attended. Good technical discussions were had and it was a good opportunity for the user community to engage directly with the technology developers, gain understanding on the new system and showcase the latest technology available for cell and gene therapy. |
| Year(s) Of Engagement Activity | 2019 |
| Description | Lecture at skills workshop |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | Presented workshop at STARS Bioprocessing Skills School. "Innovative therapies but what about the societal and economic implications: Building a compelling case - making the health economics stack up." Darlington, September 2018. |
| Year(s) Of Engagement Activity | 2018 |
| URL | http://biopronetuk.org/stars-school-2018-embedding-the-industrial-perspective/ |
| Description | Lecture on cell therapies |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Postgraduate students |
| Results and Impact | Lecture on cell therapies in the framework of the Advanced Bioprocess Engineering course for the MSc Advanced Chemical Engineering, Department of Chemical Engineering, Imperial College |
| Year(s) Of Engagement Activity | 2018,2019 |
| Description | Looking to the Future: Phase II of the Hub - Webinar Session in partnership with the BioIndustryAssociation |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | FTHM Hub Webinar Session in partnership with Dr Paul Varley, representing the BioIndustry Association and speaking on the BIA Antibodies COVID-19 workstream. Provided further opportunity for Hub Users to hear about the Hub's planned Research Programme for phase II. Brought information and generated discussion and debate on topic of critical international interest. 67 Registrations, 50 Attendees. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Manufacturing ATMPs: moving from approvable to commercial success |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The Hub presentation on our CAR T research for this KTN event/workshop |
| Year(s) Of Engagement Activity | 2018 |
| Description | Manufacturing ATMPs: moving from approvable to commercial success |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The Hub and KTN partnered to deliver this meeting enabling industry and academia engagement and collaboration, and showcasing of the relevant translational research being undertaken by the Hub in collaboration with industry. The focus of this meeting was on the current UK innovation landscape for advanced therapies, in support of enabling gene and cell therapy products to go from approvable to commercially successful; translational research projects in support of manufacturing readiness, a future looking session regarding key challenges from clinical, regulatory and skills perspectives. One of the purposes of this meeting was to promote and give an updated view of the Hub to organisations outside of the current consortium. Approx 77% of the attendees were from outside companies & organisations and we got 4 new enquiries about becoming Hub members. |
| Year(s) Of Engagement Activity | 2018 |
| URL | https://ktn-uk.co.uk/events/manufacturing-atmps-moving-from-approvable-to-commercial-success |
| Description | Manufacturing personalised medicines: overcoming the affordability barrier (Policy Dialogue Event organised by Dr Penny Carmichael), 5th February 2020 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | ***Event organised by Dr Penny Carmichael*** ***Reimbursement led by Nishma Patel*** The Hub were awarded £2,000 funding from a UCL-held EPSRC Impact Acceleration account. The aim of the funding is to accelerate the impact of UCL's EPSRC-funded research outputs by supporting knowledge exchange. To make use of this funding, an event is being scheduled around Hub work on manufacturing personalised CAR T-cell therapies. The event brought together researchers with and invited group of policy-makers, clinicians and industry stakeholders providing an opportunity to explore questions around the long-term sustainability of delivering personalised CAR T-cell (and other ex-vivo cell and gene) therapies to NHS patients. The following objectives were set for the day: (1) Provide Hub researchers with an opportunity to build relationships with the policy-making and health provider communities and to raise the profile of the Hub with these stakeholders; (2) To agree the main challenges for CAR T delivery in the UK now - opportunity to highlight manufacturing issues to policy-makers/healthcare providers; and (3) To agree to what extent these challenges are already being met and what other steps might be required My role was to lead sessions on the challenges of implementing CAR Ts in the area of reimbursement. By the end of the session, with the groups I agreed what the top two challenges were, and think about how these challenges might be overcome. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Meeting with NHS England representatives on industrial economics |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | Dissemination of Hub research to NHS England Medicines Analysis Strategy and Policy Team and discussion of industrial economics. Team then participated in Hub policy facing event held in February 2020. |
| Year(s) Of Engagement Activity | 2020 |
| Description | NHS England Accelerated Access Collaborative (ACC),Data Infrastructure Workstream - 16th April 2021, 30th September 2021, 18th January 2022 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Third sector organisations |
| Results and Impact | The scope of the meeting: 1. There are many key points relating to data generation and collection along the development and adoption journey for new technologies, however two key points to build a new data collection infrastructures around are the point when a technology is undergoing regulatory review by the MHRA and the point when NICE is undertaking a technology appraisal. 2. Focussing building data collection infrastructures around these two key points will allow organisations that input at those stages to collectively identify data collection needs. |
| Year(s) Of Engagement Activity | 2010,2021 |
| Description | NHS England Accelerated Access Collaborative (ACC),Data Infrastructure Workstream, 13th July 2020 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | The scope of the meeting: 1. There are many key points relating to data generation and collection along the development and adoption journey for new technologies, however two key points to build a new data collection infrastructures around are the point when a technology is undergoing regulatory review by the MHRA and the point when NICE is undertaking a technology appraisal. 2. Focussing building data collection infrastructures around these two key points will allow organisations that input at those stages to collectively identify data collection needs. |
| Year(s) Of Engagement Activity | 2020 |
| Description | NHS England Accelerated Access Collaborative (ACC),Data Infrastructure Workstream, 6th April 2020 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The scope of the meeting: 1. There are many key points relating to data generation and collection along the development and adoption journey for new technologies, however two key points to build a new data collection infrastructures around are the point when a technology is undergoing regulatory review by the MHRA and the point when NICE is undertaking a technology appraisal. 2. Focussing building data collection infrastructures around these two key points will allow organisations that input at those stages to collectively identify data collection needs. |
| Year(s) Of Engagement Activity | 2020 |
| URL | https://www.england.nhs.uk/aac/what-we-do/what-innovations-do-we-support/ |
| Description | NHS England Accelerated Access Collaborative, 11th February 2020 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Other audiences |
| Results and Impact | This AAC address issues the National Health Service is confronting; how best to access innovation for the benefit of patients and to improve health care efficiency. The approach to accessing innovation in the NHS has become increasingly challenging; creating frustration for innovators who see the NHS as an interesting environment for demonstrating the value of their products, for patients who often have to wait long periods of time before life-saving therapies are available, and for clinicians who are frustrated by the multiple barriers to both approval and adoption. One work stream is looking at ATMPs and easing their adoption in the NHS. We discussed the possibility of Hub members contributing to the following work streams: 1. Standardisation of system requirements (Suzy, Qasim & Irina) 2. Data infrastructure (Nishma/Steve) 3. NICE methods review (Nishma/Steve) 4. Exploration of reimbursement approaches (Nishma/Steve) 5. Supporting data generation opportunities (Irina) |
| Year(s) Of Engagement Activity | 2020 |
| Description | NHS England Medicines Analysis Strategy and Policy Team, 22nd November 2019 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Other audiences |
| Results and Impact | Key points raised from the meeting: • Since ATMPs often receive market authorisation through accelerated regulatory pathways, when they reach the Health Technology Assessment (HTA), information gaps exist. There is a tension between the political pressure to push for ATMP access early in product life-cycle and robust and fair HTAs. • To cope with the novelty of ATMPs and the high price-tag, people have jumped straight to "we need a pricing model" without thinking about the steps needed for market access. What we need to do is step back and think about the following, stressing the unmet need: o What is the disease? o What is the burden? o How does it affect the patient? o Why is it different to any other disease? o Is it best for the patient and NHS? Using this kind of thinking, a commercial framework needs to be drafted for ATMPs. • There is still financial uncertainty with current pricing and payments models. NHS England suggested that the Hub use the evidence already gathered, but supplement this with information from EU5 markets and invite experts from each to form an advisory board and they can translate the information and provide at least qualitative summaries. Could also consider inviting someone from Canada. • Since pharma is global, products can be brought to market in one region, e.g. the US and then pressure is on other regions to provide patients with access to the therapy, but there may have been improper consideration of (using gene therapy for macular degeneration as an example): o Add on costs e.g. genetic counselling o Gene testing and failures o Efficacy waning (depends on the disease) o Patient population with high prevalence and low incidence o Uncertain clinical evidence o The opportunity cost of preventing a new product coming through o Irrecoverable costs off loaded surplus prices into patent prices • It is not clear that all these costs for the extra levels of service provision necessary for ATMPs are adequately being captured. ATMPs will also require ramping up of services at both a national and regional level to meet the infrastructure and technical implementation requirements. • An area of concern was the provision first-generation ATMPs precluding patients from receiving more advanced next-generation therapies when they become available. • Voluntary Pharmaceutical Pricing Regulation Scheme (PPRS) has changed - we'd normally expect a better value proposition in exchange for the complexity of the product e.g. Italian system (treatments thought to be initially effective came with huge risks) |
| Year(s) Of Engagement Activity | 2019 |
| Description | News interview for Royal Society fo Chemistry (Chemistry World) on Nobel Prize Awards to Frances Arnold and Greg Winter in 2018 |
| Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Media (as a channel to the public) |
| Results and Impact | Interviewed to comment on the Nobel Prize Awards to Frances Arnold and Greg Winter in 2018. Quoted in an article online and also in Chemistry World magazine published by the Royal Society of Chemistry. https://www.chemistryworld.com/features/how-chemical-evolution-took-the-2018-chemistry-nobel-prize/3009626.article |
| Year(s) Of Engagement Activity | 2018 |
| Description | News interview for the Guardian on Nobel Prize Awards to Frances Arnold and Greg Winter in 2019 |
| Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Media (as a channel to the public) |
| Results and Impact | News interview for the Guardian on Nobel Prize Awards to Frances Arnold and Greg Winter in 2019. Quoted in article printed in the Guardian and online. |
| Year(s) Of Engagement Activity | 2018 |
| Description | Online: FDA Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) Meeting #70 September 2-3, 2021 Toxicity Risks of Adeno-associated Virus (AAV) Vectors for Gene Therapy (GT) |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Purpose to learn about the Biology of rAAV and I have learned about integration of rAAV in the genome: - rAAVvectors can stably integrate into the mouse genome (=10%) - The vast majority of integrated vectors appear to be grossly rearranged - The integration mechanism is unknown -Strong promoter/enhancer combinations increase hepatocellular carcinoma (HCC) frequency - Low frequency of HCC in adult animals - Low frequency of HCC following CNS-directed gene therapy |
| Year(s) Of Engagement Activity | 2021 |
| Description | Organised a policy dialogue on the affordability of personalised medicines |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | Event description: CAR T-cell therapies and other personalised medicines offer the prospect of vastly improved outcomes for patients, but will require radical changes in the way that drugs are manufactured; moving from centralised, large-scale production of off-the-shelf therapies to individual batches, possibly at the bedside. How might the costs of production be reduced so that personalised treatments are affordable? Will new business and financing models be needed to make these therapies available on the NHS? And how might the idea of 'bedside manufacture' be realised? We would like to invite you to attend a policy dialogue hosted by UCL's Future Targeted Healthcare Manufacturing Hub (the Hub) to explore these questions and more. During the event, we will consider what innovations in manufacturing, NHS service delivery, regulation and reimbursement are required to maximise the value that the NHS spends on drugs each year and ensure the best outcomes for patients. The event will be Chaired by Dr Mark Carver (former Senior VP, R&D and Innovation, FUJIFILM Diosynth Biotechnologies) and take place on 5th February 2020 between 10:00 - 15:30 at the Institution of Mechanical Engineers in Westminster, London. Speakers: Professor Suzy Farid, UCL Department of Biochemical Engineering, will provide an overview of the Hub's work on CAR T-cell therapies, including the cost-of-goods for producing CAR Ts and the challenges presented for pricing and reimbursement. Professor Farid will also reflect on future developments and opportunities for personalised medicines. Nina Pinwill, NHS England, will recount lessons learnt from the initial implementations of CAR T treatments. Dr Claire Roddie, UCL Cancer Institute, will share her experiences as a leader in the UCL CAR T programme and will set out a vision of how patients will be benefitted by personalised therapies in the future. The session offers invited attendees from Government, industry, the third sector and academia an opportunity to convene and collectively examine the current and future challenges of delivering personalised medicines in three interactive sessions. |
| Year(s) Of Engagement Activity | 2020 |
| URL | https://www.eventbrite.co.uk/e/manufacturing-personalised-medicines-overcoming-the-affordability-bar... |
| Description | Organising and chairing a session for an ENABLE satellite event: Key elements to bridge the gap between academic, clinical and industry collaborations |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Postgraduate students |
| Results and Impact | The Early Career Network for the Personalised Medicines and Cancer Domains organised a virtual half-day workshop promoting the career development of early-career researchers. Our workshop covered elements such as patient and public involvement (PPI), intellectual property (IP), and developing ideas from bench to business, as they are becoming key when applying for research funding, but also for academic career promotion. If a research proposal does not include a well-structured PPI component, or the impact of research studies in terms of possible IP elements derived from it is not well-defined, the possibility of success considerably decreases. Moreover, being able to properly pitch your ideas is key to secure funding or obtaining the position you applied for. We also should not forget the impact our research could have on public policy. Our workshop provided the attendees with a broad but carefully chosen overview of these components and some advice to easily implement them in their own research projects. It is important for scientists to develop these skills at early stages of their career, through appropriate training. We would also have a networking session to encourage further discussion between participants. |
| Year(s) Of Engagement Activity | 2021 |
| Description | PSE2021+ conference |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Oral presentation given online at the PSE2021+ conference. |
| Year(s) Of Engagement Activity | 2022 |
| Description | Participation in Innovation 2 Market course at London Business School |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Postgraduate students |
| Results and Impact | A colleague and I joined the I2M course, with the aim of building a commercialisation strategy for the cell-free pDNA production protocol and device which I have begun developing during this postdoc. We have made significant progress already, and learnt a substantial amount about the process of commercialisation. |
| Year(s) Of Engagement Activity | 2022,2023 |
| Description | Participation in the NHS AAC workstream on roadmapping development on ATMPs |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | The NHS Accelerated Access Collaborative was established to expedite patient access to new and innovative medicines and digital technologies. Through a series of meetings, Hub researchers fed-into the a workstream of the programme which is delivering a roadmap for ATMP developers navigating the complex requirements for approval of a new therapy |
| Year(s) Of Engagement Activity | 2020 |
| Description | Personalised medicine made in hospitals can revolutionise the way diseases are treated: the challenge now will be implementing it |
| Form Of Engagement Activity | Engagement focused website, blog or social media channel |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Public/other audiences |
| Results and Impact | Jargon-free article about point-of-care manufacture. |
| Year(s) Of Engagement Activity | 2022 |
| URL | https://theconversation.com/personalised-medicine-made-in-hospitals-can-revolutionise-the-way-diseas... |
| Description | PodCast on COVID-19 research |
| Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Interviewed by the Podcast series Never Lick the Spoon, Institute for Molecular Science and Engineering at Imperial College London on my research on supply chain optimisation for COVID-19 vaccines. |
| Year(s) Of Engagement Activity | 2020 |
| URL | https://neverlickthespoon.podbean.com/e/episode-9-getting-rid-of-covid-part-ii/ |
| Description | Policy brief on biomanufacturing |
| Form Of Engagement Activity | Engagement focused website, blog or social media channel |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | Policy brief published on the UK biomanufacturing industry highlighting the Hub's expertise in this area and strategic fit within a national investment portfolio |
| Year(s) Of Engagement Activity | 2020 |
| URL | https://www.ucl.ac.uk/steapp/sites/steapp/files/biomanufacturing_briefing_jan_2020.pdf |
| Description | Policy engagement scoping meetings |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | A series of scoping meetings were held with policymakers with the aim to: 1. Identify areas where Hub research may align with/impact current policy-making questions and concerns; and 2. Raise the profile of the Hub within the policy-making community. The following key meetings were held: 1. 20th June 2019: HM Treasury's Health and Social Care team, to provide an overview of the Hub research and identify areas of mutual interest; 2. 1st July 2019: Office for Life Sciences Investment and Engagement Team, to provide an overview of the Hub research and identify areas of mutual interest; 3. 19th September 2019: a follow-on meeting was held with the HM Treasury Health and Social Care team to discuss Hub research on the cost-of-goods for CAR T manufacture.; 4. 30th October: Commercial Medicines Directorate NHS England, to provide an overview of the Hub research and identify areas of mutual interest; 5. 22nd November 2019: NHS England Medicines Analysis Strategy/Policy Team, to provide an overview of the Hub research and identify areas of mutual interest; This series of scoping meetings led to the development of a network of policy professionals with whom to engage on Hub research. Additionally, the cost-of-goods for CAR T manufacture and how this varies under different scenarios emerged as an area of particular interest to the individuals with whom we met. As a result, a 'policy dialogue' meeting is being held in February 2020 which will convene a range of policy and industry stakeholders to consider the business and reimbursement challenges for CAR T manufacture and implementation in the NHS. |
| Year(s) Of Engagement Activity | 2019 |
| Description | Poster Presentation, 15th May 2018 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | This meeting was an opportunity to update users on the progress of Hub research and to network with other potential researchers and industry partners. |
| Year(s) Of Engagement Activity | 2018 |
| Description | Poster Presentation, 7th November 2017 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | Poster presentation part of the User Steering Committee meeting in 7th November 2017. Used to engage with collaborators and industry attendees. |
| Year(s) Of Engagement Activity | 2017 |
| Description | Presentation for Cell-Free Synthesis Specialist Working Group Webinar |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | Presented outcomes from the user feasibility study with CRUK. FujiFilm Diosynth contacted us regarding potential collaboration to extend this research. |
| Year(s) Of Engagement Activity | 2021 |
| Description | Presentation to the NHSE&I Accelerated Access Collaborative ATMP project leads |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | A meeting was held between Hub researchers and the ATMP project leads for the NHS Accelerated Access Collaborative which aims to expedite access for NHS patients for new innovations in medicines and digital technologies. Hub researchers shared key findings from the project, resulting in invitations to contribute to the NHS AAC's working groups on ATMPs. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Presented at PEGs Boston (Virtual) |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Presented a pre-recorded conference paper entitled "A rational approach to designing cell-free synthesis based biomolecule platform screening processes" and participated in a live Q&A with other panelists in the "difficult to express proteins" series. |
| Year(s) Of Engagement Activity | 2021 |
| Description | Recovery of Biological Products Board Conference Series . |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Chair of Recovery of Biological Products Board Conference Series . 2011- 2012 Now member of Scientific Board. The Recovery Conference Series is the premier international forum for the presentation and discussion of the status, direction and trends in the recovery of biological products of therapeutic, diagnostic and industrial value to the society. . Awarding Body - Recovery of Biological Products Board Conference Series, Name of Scheme - Recovery of Biological Products |
| Year(s) Of Engagement Activity | 2012,2014,2016,2018 |
| Description | Regulation, Reimbursement and Policy Specialist Working Group, 16th April 2021 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | This session offered insights on the new research agenda of the Regulation, Reimbursement and Policy Group for Phase II of the Future Targeted Healthcare Manufacturing Hub (2020-23). We updated colleagues, partners and collaborators on our main findings to date and invite participants to reflect on our main workplans moving forward, with a view to establishing long-lasting collaborations. Some of our forthcoming priorities include: evaluating the regulatory challenges for point-of-care manufacture; exploring innovations in pricing and payment mechanisms for advanced therapies; mapping regulatory alignment, standardization and compliance challenges resulting from Brexit. |
| Year(s) Of Engagement Activity | 2021 |
| Description | Regulation, Reimbursement and Policy Specialist Working Group, 22nd October 2021 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Third sector organisations |
| Results and Impact | The current Covid-19 pandemic has brought about some lessons that will hopefully improve the ways in which therapies are assessed, innovation fostered, and regulatory frameworks designed. These needs seem to be particularly high for cutting-edge therapies such as CAR-T cells, as different players need to quickly learn how to develop, manufacture, and regulate them. This Specialist Working Group aims to carry out this discussion, relying on the participation of professionals involved in the development, study, and regulation of advanced therapies. During our session, we will address two major innovations in personalized therapy development, delivery, reimbursement, and regulation: - The clinical results of the Phase I novel CD19 CAR and their implications on current and future reimbursement models: Chimeric Antigen Receptor (CAR) T-cell therapy offers potential curative effects in hematology and oncology. The two major adverse effects of CAR T-cell therapy are cytokine release syndrome (CRS) and neurotoxicity. The occurrence of mild or severe adverse effects during hospitalisation requires additional treatment and prolonged hospital length of stay. Optimal management can lead to greater health outcomes for patients and potential cost savings to the NHS. Together with the ALLCAR19 trial team (UCL Cancer Trials Centre) we explore the clinical results of the phase I novel CD19 CAR developed to reduce adverse events. - The latest Point-of-Care Manufacture Regulatory Framework Proposal put forward by the MHRA, its implications on key players such as hospitals, and the development of future standards and regulatory guidelines to support this regulatory proposal. It is important to explore the ways in which innovation is being promoted in personalized therapies such as CAR-Ts, asking whether these innovation pathways will lead to therapies that can be easily accessed and properly managed by the regulatory authorities. At the same time, it is important to consider recent trends and projects in therapy development, such as the prospects for somewhat decentralized, or even highly decentralized, manufacturing schemes. These new approaches will necessarily require the involvement of players (such as hospitals) which have traditionally not been present in manufacturing activities and supply chains. For this reason, new regulatory approaches have been proposed, as in the case of the MHRA's regulatory framework proposal for point-of-care manufacture. Through three talks and two discussion sessions, this event will address these issues, opening up a space for the expressions of insights and sharing of relevant experiences. This meeting informed existing research and how to take the next phase of the research forward. |
| Year(s) Of Engagement Activity | 2021 |
| Description | Regulation, Reimbursement and Policy Specialist Working Group, 29th April 2022 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Third sector organisations |
| Results and Impact | We are delighted to invite you to this SWG session, where we will be addressing two critical issues emerging from our ongoing research and engagement: 1. Institutional and infrastructure changes and challenges facing hospitals willing to manufacture ATMPs at point-of-care. What kind of infrastructure and institutional shifts are and can be implemented in hospitals willing to manufacture ATMPs at point-of-care? This question is particularly important considering the UK Medicines and Healthcare products Regulatory Agency's (MHRA) new framework proposal aimed at making it easier, from a regulatory perspective, to manage point-of-care manufacturing initiatives. In the next years, the MHRA expects to see some manufacturing systems that will involve large numbers of hospitals. In ATMPs, manufacture will probably continue to happen in a few hospitals, although in a more consistent way, and probably also at larger scale. It is then important to see if we can learn lessons from the infrastructure and institutional changes that have been adopted by some hospitals and can favour the implementation of ATMP point-of-care manufacture. This section will involve a review of, and a discussion about, these changes and their implications. 2. Stakeholder preferences for pricing and reimbursing ATMPs. As more high-cost ATMPs enter the market, both in the UK and worldwide, manufacturers and healthcare systems need to identify affordable ways to price and pay for therapies. To understand what is important to stakeholders when negotiating pricing and reimbursement, it is important to understand stakeholder preferences. In this section, we will explore stakeholder preferences in reimbursement and pricing for ATMPs. |
| Year(s) Of Engagement Activity | 2022 |
| Description | Regulatory clusters for therapy manufacturing clusters: highly automated cell and gene therapy production and its regulatory implications |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Presentation at the World Conference on Stem Cells and Regenerative Medicine, Paris, France |
| Year(s) Of Engagement Activity | 2023 |
| Description | Reimbursement, Market Access Approval and Evidence for Cell and Gene Therapies (poster presentation). User Steering, Committee 5th November 2019 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The poster reports outcomes from three case studies and (i) examined the impact of limited clinical evidence on the cost-effectiveness of cell and gene therapies and emerging reimbursement/payment models for the following: • Strimvelis • Kymriah (Tisagenlecleucel) • Yescarta (Axicabtagene Ciloleucel) |
| Year(s) Of Engagement Activity | 2019 |
| Description | Reimbursement, Regulation & Portfolio Management. User Steering Committee 21st May 2019 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | Cell and Gene therapy products entail complex research, development, manufacturing and delivery. These complexities are reflected in the processes for portfolio planning, regulatory approval and reimbursement. At the stage of portfolio planning, manufacturers need to determine the optimal development sequence and resource utilisation for hedging products, to maximise the overall projected profits during the market exclusivity period. To authorise access to these products regulators must assess the quality of the product and its risk benefit profile, while healthcare providers/insurers need to assess its affordability. We described the challenges and success of cell and gene therapies, using Strimvelis and Kymriah as our case studies. |
| Year(s) Of Engagement Activity | 2019 |
| Description | Royal Society of Chemistry Conference Organisation 2017 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | I organised a conference for the Royal Society of Chemistry Biotechnology Group titled "Biotransformations: From Science to Industrial Application", in London. This was attended by approx 70 delegates from across academia and industry, internationally. The conference enabled discussions on the sustainable use of biomass for production of high value chemicals and biologics, including regulatory issues. |
| Year(s) Of Engagement Activity | 2017 |
| Description | STEM Outreach |
| Form Of Engagement Activity | A broadcast e.g. TV/radio/film/podcast (other than news/press) |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Schools |
| Results and Impact | TechWeCan initiatice lead by PwC and RAEng Education Description as taken from the website: "this lesson is to explain how technology is used during pandemics and to prevent spread of viruses. the lessons also broadens the student's knowledge of careers available designing, creating and using tech for pandemics" |
| Year(s) Of Engagement Activity | 2021 |
| URL | https://weclass.techwecan.org/portal/courseDetails?Web=140 |
| Description | Series of meetings with UCL CAR T programme, Dr Claire Roddie and UCL CAR -T trial team, 2nd July 2020 - 19th October 2020 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Professional Practitioners |
| Results and Impact | Discussion on the data collection and data variables needed to undertake health economics analysis. The final proposal was submitted to the ALLCAR19, CR UK & UCL Cancer Trials team in October 2020. The proposal objectives are: (1) To identify the cost and cost-effectiveness of ALLCAR19 to the NHS for patients who receive CAR T-cell therapy (2) To calculate the difference in costs by type of adverse event (3) Evaluate the cost associated with adverse events of CAR T-cell therapy with main comparators. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Spring school on 3D-printing of columns, devices and stationary materials |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | This workshop originates in response to the increasingly significant role of Additive Manufacturing (AM, or 3D printing) in the separation sciences. This three day course was elaborate on current work and future prospects of 3D printing. It covered the full pipeline to produce separation devices, from conceptual design to CAD modelling to manufacture using 3D printers to post-processing, evaluation and troubleshooting. Lectures on 3D printing methods, CAD design, and applications in the separation sciences will be combined with hands-on tutorials with CAD design software and practicums on 3D printers. |
| Year(s) Of Engagement Activity | 2018 |
| URL | https://3dprintchromatographycolumns.wordpress.com/ |
| Description | T-cell Processing Specialist Working Group |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | The T-cell Processing Specialist Working Group (SWG) has met twice a year since the launch of the Hub. The focus of the T-cell Processing SWG is to discuss the the research activity and strategic direction of the T-cell autologous cell therapy development activity within the Hub, engaging users of their perspectives, as a platform for dissemination and ensure that the research plans are addressing key challenges within the sector. With input and presentation from Hub users (including professional practitioners from industry and the clinic) it is also used to highlight the state of the art in the sector and identify emerging areas for research exploitation and areas that need addressing. It is also designed to identify potential risks or additional work that could be undertaken in the T-cell processing work packages. |
| Year(s) Of Engagement Activity | 2017,2018 |
| Description | Teaching assistance on Vaccines Bioprocessing course |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Postgraduate students |
| Results and Impact | Prepared and recorded a set of lectures on novel vaccine technologies for UCL Biochemical Engineering Vaccines Bioprocessing course, and answered questions in a live session. The novel vaccine technologies included a number I am familiar with through my research activities. I also assisted in managing live sessions and marking coursework. |
| Year(s) Of Engagement Activity | 2020,2021 |
| URL | https://www.ucl.ac.uk/biochemical-engineering/prospective-students/msc_modules/bengg028 |
| Description | Technology governance and regulatory fragmentation in advanced therapies:the UK's clinical network for CAR-T cell therapies targeting resistant cancers |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Oral presentation in the International Conference of the European Consortium for Political Research, University of Innsbruck, Austria |
| Year(s) Of Engagement Activity | 2022 |
| Description | Tele-conference with AstraZeneca for feasibility study |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Brainstorm on feasibility study collaborations |
| Year(s) Of Engagement Activity | 2020,2021 |
| Description | Tele-conference with Insmed |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | Using our continuous process model to help Insmed to solve practical problem. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Tele-conference with Medimmune on feasibility study |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Discussion the possibility of collaboration on continuous chromatography real-time analysis and control. |
| Year(s) Of Engagement Activity | 2019 |
| Description | The Conversation article |
| Form Of Engagement Activity | A magazine, newsletter or online publication |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Public/other audiences |
| Results and Impact | A widely read article for The Conversation written in collaboration with a colleague from the Vax Hub: "Coronavirus: how the pharma industry is changing to produce a vaccine on time". The article was republished by a number of media sites. Stephen and myself have participated in a few interviews on the subject of vaccines production. |
| Year(s) Of Engagement Activity | 2020 |
| URL | https://theconversation.com/coronavirus-how-the-pharma-industry-is-changing-to-produce-a-vaccine-on-... |
| Description | The Engineer Interview |
| Form Of Engagement Activity | A press release, press conference or response to a media enquiry/interview |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Interviewed by The Engineer on my research on supply chain optimisation for COVID-19 vaccines. |
| Year(s) Of Engagement Activity | 2020 |
| URL | https://www.theengineer.co.uk/scaling-up-uk-vaccine-manufacture/ |
| Description | The Role of Patients in Drug Development, Regulatory Approval and Reimbursement Decisions - Webinar session in partnership with ABPI, UoOxford/HERC and NHS England Accelerated Access Collaborative (AAC) |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Brought together practitioners from a range of stakeholder groups. Shared expertise and information and generated discussion and debate on topic of sector importance |
| Year(s) Of Engagement Activity | 2020 |
| Description | The ethics of personalised medicines manufacture and use |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | Ran a workshop to identify the ethical issues related to manufacturing personalised medicines and formulate specific research questions on ethical issues related to the use of data and AI, and on access to medicines in resource limited settings. Additionally, the Hub team had the aim to identify those with research experience and expertise in ethics and create the opportunity for collaboration via short, funded feasibility studies, which will be informed by the findings of this workshop. |
| Year(s) Of Engagement Activity | 2021 |
| Description | Towards Regulatory Scenarios for Proteins and Cell Therapies: mapping the regulatory drivers and decision nodes |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | On February the 15th 2018, at the Bioengineering Department of UCL, as part of the activity of the Grand Challenge 1.2c - regulatory strategies- Giovanni De Grandis and Arthur Petersen convened a workshop bringing together junior and senior staff from the hub and some high-profile experts in biopharmaceutical regulations and policy. Through a combination of group work and presentations, the workshop performed a small-scale horizon scanning exercise, aimed at identifying regulatory challenges and opportunities for the hub. |
| Year(s) Of Engagement Activity | 2018 |
| Description | Trends in Viral Vector Processing - Webinar session in partnership with Cobra Biologics |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | FTHM Hub Webinar Session in partnership with Cobra Biologics. Delivered information and generated discussion and debate on topic of significant sector interest. 83 Registrations. |
| Year(s) Of Engagement Activity | 2020 |
| Description | UCL 3D Printing in Healthcare Research Symposium |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Postgraduate students |
| Results and Impact | Bioprinting background, skills, facilities and contacts who worked in UCL in the field. |
| Year(s) Of Engagement Activity | 2018 |
| Description | UCL Cancer Institute, UCL CAR T programme, Dr Claire Roddie and Professor Karl Peggs, 10th October 2019 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Professional Practitioners |
| Results and Impact | Discussion on the reimbursement and health technology assessment of cell and gene therapies, including CAR T-cell therapy. We explored the potential of a collaboration using existing trial data for a feasibility study. A proposal were submitted to the group in January 2020. The proposal objectives are: (1) To identify the cost to the NHS for patients who receive CAR T-cell therapy and have adverse events (2) To calculate the difference in costs by type of adverse event (3) Evaluate the cost associated with adverse events of CAR T-cell therapy with blinatumomab |
| Year(s) Of Engagement Activity | 2019 |
| Description | UCL Early Careers Research (ECN) Network Personalised Medicine Domain. Bridging the gap between academic, clinical and industry collaboration: impact across the research lifecycle, Monday 10th May 2021 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Postgraduate students |
| Results and Impact | Organise and lead the workshop is to promote the career development of early-career researchers, covering elements such as patient and public involvement (PPI), intellectual property (IP), and developing ideas from bench to business.Bridging the gap between academic, clinical and industry collaboration: impact across the research lifecycle. As a part of ENABLE 2021 satellite events, this virtual half-day workshop aimed to promote the career development of early-career researchers working the the personalised medicine field. The workshop covered elements such as patient and public involvement (PPI), intellectual property (IP), and developing ideas from bench to business, as they are becoming key when applying for research funding, but also for academic career promotion. The workshop provided attendees with a broad but carefully chosen overview of these components and some advice to easily implement them in their own research projects. The workshop asked volunteers to deliver a short pitches during the pitching session, providing opportunities for presenters, colleagues, and the trainer to provide constructive feedback on your presentation style. |
| Year(s) Of Engagement Activity | 2021 |
| Description | User Steering Committee Meeting |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | This is a meeting of the whole Hub consortium - academic team, members from the Hub's Industrial and non-corporate partner organizations, independent members of the Hub Advisory Board. The purpose of this bi-annual meeting is to provide an update on the progress of Hub research and (once a year) an update of the research in the Centres for Doctoral Training (CDT) aligned to the Hub. The format includes formal presentations, poster sessions, round-table workshop discussions and structured networking elements. |
| Year(s) Of Engagement Activity | 2017,2018,2019,2020,2021 |
| Description | User Steering Committee meeting, 15th May 2018 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The User Steering Committee is an important part of the hub. It is was an opportunity to identify how companies can help the project by provision of my project in terms of: advice, data, equipment or access to equipment, resources materials, future evaluation of experimental techniques, or participation in user feasibility or adaptation studies. |
| Year(s) Of Engagement Activity | 2018 |
| Description | User Steering Committee meeting, 7th November 2017 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The User Steering Committee is an important part of the hub. It is was an opportunity to identify how companies can help the project by provision of my project in terms of: advice, data, equipment or access to equipment, resources materials, future evaluation of experimental techniques, or participation in user feasibility or adaptation studies. |
| Year(s) Of Engagement Activity | 2017 |
| Description | User Steering Committee meetings |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | The User Steering Committee meeting was an excellent platform to engage industry and academics to highlight our latest research work being done in the hub. The benefit of this kind of meeting is that work groups/researchers can have interdisciplinary interactions with other scientists and provide a fresh perspective while everyone learns through the interaction. We've had overwhelmingly positive feedback from everyone involved. |
| Year(s) Of Engagement Activity | 2016,2017,2018 |
| Description | User feasibility study engagement with Albumedix |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Industry/Business |
| Results and Impact | Identification of the key challenge in lenti-/AAV viral vector production (from lab to industry scale) Potentially leading to innovation in the formulation & bioprocessing of viral vector that brings extra stability enhancement of the products. |
| Year(s) Of Engagement Activity | 2020 |
| Description | User interviews on Walter Low from Polpharma Biologics regarding to CDx parameters assumption |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Asking help for CDx parameters assumption |
| Year(s) Of Engagement Activity | 2020 |
| Description | Vaccines Development and Manufacturing Workshop |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | The Hub co-hosted a workshop on vaccine manufacturing and development with the KTN on 6 September 2017 at UCL. The event brought together more than 90 delegates from the vaccine development and manufacturing community in human health in the UK. Presentations were given on the latest innovative technologies, current UK capabilities, Department of Health investments to date and the work of the UK Vaccine network. It highlighted potential funding opportunities through the Industrial Strategy Challenge Fund. |
| Year(s) Of Engagement Activity | 2017 |
| URL | https://ktn-uk.co.uk/news/the-future-of-vaccines-development-and-manufacture |
| Description | Viral Vectors for Vaccines and Gene Delivery, 9th Sept 2019 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | This one-day meeting was jointly organised by this Hub, the Future Vaccine Manufacturing Research Hub (Vax-Hub) and the Knowledge Transfer Network and offered a programme of invited talks, a poster session and an interactive panel discussion. The meeting bought together ~110 participants from industry, academia, government, sector organisations, to inform and discuss the common manufacturing and analytical challenges across the major viral vector products. |
| Year(s) Of Engagement Activity | 2019 |
| URL | https://ktn-uk.co.uk/news/viral-vectors-for-vaccines-and-gene-delivery/ |
| Description | We must improve accessibility and affordability of biologics through innovation, Webinar Session with Jorg Thommes PhD |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | FTHM Hub Webinar Session with invited speaker Dr Jorg Thommes, Executive Vice President of Pharmaceutical Sciences and Operations, Repertoire Immune Medicines, Cambridge MA, USA |
| Year(s) Of Engagement Activity | 2022 |
| Description | Workshop "Ideas to Impact", Entrepreneurship Essentials for Researchers (SPERO) |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Industry/Business |
| Results and Impact | A nice workshop on Entrepreneurship Essentials for Researchers (SPERO) |
| Year(s) Of Engagement Activity | 2018 |
| Description | Workshop in Bioproceesing |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Postgraduate students |
| Results and Impact | A workshop and lecturing session were designed for Nottingham University Master students who visited CBE centre at Loughborough university. |
| Year(s) Of Engagement Activity | 2020 |
| Description | Workshop on Sustainable Practices in Personalised Medicine Manufacture |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Policymakers/politicians |
| Results and Impact | The aim of the workshop was to obtain a clear understanding of the current and future environmental sustainability challenges in personalised biological medicine manufacturing and understand which of these should be prioritised in the Hub's next programme of research once the current Hub draws to a close at the end of 2023. The workshop brought together 17 participants from the following organisations: The Assocation of the British Pharmaceutical Industry, AstraZeneca, The Cell and Gene Therapy Catapult, The Knowledge Transfer Network, The Engineering and Physical sciences Research Council (observer), Innovate UK, NHS England, Sartorius, Univercells, and University College London. The Hub will use the ideas generated in this workshop to inform the activities in their research proposal for the 'Hub 2', which will continue and build on activities from the first research cycle. |
| Year(s) Of Engagement Activity | 2022 |
| URL | https://www.ucl.ac.uk/steapp/sites/steapp/files/sustainable_practices_in_pm_manufacture_hub_workshop... |
| Description | Workshop on point-of-care manufacture |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Policymakers/politicians |
| Results and Impact | This workshop discussed the challenges and potentialities of point-of-care manufacture. It was organised in the framework of the renewal of the UK legislation, with a new regulatory framework being proposed by the Medicines and Healthcare products Regulatory Agency (MHRA). The event had a presentation by an MHRA representative and participation of other MHRA members. |
| Year(s) Of Engagement Activity | 2021 |
| URL | https://www.ucl.ac.uk/steapp/collaborate/policy-impact-unit/current-projects/workshop-report-regulat... |
| Description | Workshop- User Steering Committee, 6th November 2018 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Industry/Business |
| Results and Impact | As high-priced personalised healthcare moves towards a pivotal change, healthcare providers like the NHS are faced with affordability challenges and pressures of resource allocations. We explore the financial challenges ahead for the healthcare payers and products developers. We looked at some recent initiatives and decisions by European regulators (EMA) and British health technology assessors (NICE) and buyers (NHS). There is currently misalignment about what counts as sufficient evidence for clinical effectiveness. Clearly different goals need to be balanced: for instance offering more therapeutic options to patients in need, promoting innovation in therapeutic areas with unmet needs, allocating limited healthcare resources fairly and efficiently. But can a balance acceptable to all stakeholders be found? Can new products benefit patients and reward developers if requirements are not harmonised? We ran two workshops with users to bring their perspective to this important discussion and explored the following: 1. How do accelerated pathways affect the production process 2. Can the evidence requirements for effectiveness of the EMA and national HTA bodies and payers be harmonized? 3. How can manufacturers and NICE work together to formulate reimbursement models? 4. How can we bring down the cost of goods and services to align them with the NHS budget? |
| Year(s) Of Engagement Activity | 2018 |
| Description | co-Chair AIChE Annual Meeting 2020 & 2021 |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | Chairing 2 sessions at the AIChE Annual meeting 2020 and 3 sessions at the AIChE Annual meeting 2021 |
| Year(s) Of Engagement Activity | 2020,2021 |
| Description | presentation the poster "Label free recognition of non-activated and activated T cells by Quantitative Phase Imaging on a chip" and discussion it in the workgroup of the Europtrode 2021 conference on innovations in chemical & biochemical optical sensing (Warsaw, 28.11--01-12.2021) |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | The poster was presented on-line in alone room for a conference attendants, which sparked questions and discussions in related subject areas |
| Year(s) Of Engagement Activity | 2021 |
| URL | http://europtrode2020.eu/ |