Biomodifying technologies and experimental space: organisational and regulatory implications for the translation and valuation of health research.

Lead Research Organisation: University of Oxford
Department Name: Law Faculty

Abstract

Developments in biomedical innovation today can be seen in areas such as robotics, digital systems or new imaging techniques - and increasingly in areas marked by highly sophisticated forms of medical biology and biotechnology that involve altering 'natural' biological processes. Three key developments form the focus for this project: the arrival of 'gene-editing' whose goal is to understand and remove disease-related mutations, the creation of induced pluripotent stem cells that can be controlled to create different types of tissue for cell therapy, and the emergence of 3D printing of biological material which aims to create novel structures for bodily repair and renewal.

These developments can all be described as 'biomodifying technologies', that is, those that modify living biological tissue in novel and increasingly patient-orientated and customised ways. These are not simply important in a biomedical sense but also in the ways in which they are reshaping the landscape of biomedical innovation in the 21st century. Not only do these technologies challenge existing governance frameworks in terms of standards for safety, quality control, and traceability of biological materials, equally and perhaps more importantly, they raise new questions for health systems and social science inasmuch as these are 'gateway' technologies with wide-ranging applications, significant commercial engagement and high levels of transferability, which open up far-reaching possibilities. We need to understand and anticipate such developments if we are to build an informed and constructively critical social science of biomedical innovation today. More broadly, this contributes towards the ESRC's core priority and delivery plan aim of supporting research that can promote economic growth and development, and to do so in a way that is based on robust, engaged social science that maps and analyses the implications of innovation.

The project will contribute towards STS theory through the development of a conceptual framework for the analysis of 'experimental space', a framework built through the analysis of the spatial characteristics of the science of biomodifying technologies, the processes of valuation at work in these spaces, and the regulatory and governance landscape of the field. The first element focuses on the sites at which biomodifying technologies are developed, the geographical networks through which they are mobilised, and, the knowledge-based terrain they define, through which biomedical scientists build new models, standards, trials and clinical applications. The second element draws on recent social science work on the meaning of value - sometimes called 'valuation studies'. Here we are interested in discovering how values and benefits are defined, by whom, and according to what criteria. Our third element draws on social science work on regulation and governance, in particular, work that explores the tensions between regulation, law, and new biologies. Together these approaches help us to build and analyse datasets that will allow us to understand the current and future impact of the three technologies in the hospital, the clinic, the firm and the wider implications they will have for the health system.

The project will use a mixed methods approach for UK fieldwork combining documentary analysis of various literatures, including the academic and grey literatures, with qualitative semi-structured interviews with a range of key stakeholders in each of the fields being studied. These include scientists working in academic laboratories, representatives of SMEs, patient groups, research agencies, regulators, and senior staff in important service organisations (e.g. biobanks). Secondary data from other European, US and East Asian sources will also be secured.The project will result in data, academic papers and policy reports that will offer the first comprehensive social science analysis of these major developments in biomedicine.

Planned Impact

The project's research findings will benefit the following groups: innovators and industry, policymakers and regulators, healthcare professionals and managers, patients and publics. Details of each group and how they will benefit are provided below:

Impact on innovators and industry - beneficiaries include non-academic researchers, technology developers and firms (Small / Medium Enterprises and large companies) with an interest in developing, translating and commercialising biomodifying technologies and products and services derived from them. Research findings will have impact by i) informing early stage development of new biomodifying technologies by providing insight into the needs and valuation practices of a variety of stakeholders including patients, regulators, and health service professionals ii) helping to shape viable business models for deploying biomodifying products in the healthcare marketplace. The mapping of the experimental and regulatory landscape will also provide valuable guidance on the legal and ethical requirements and responsibilities of service providers. Several project members are members of the UK Association for Studies in Innovation Science and Technology (AsSIST-UK) which has been set up with the goal of supporting social sciences engagement with science and innovation, civil society, government, and industry within the UK and globally, and is particularly well-suited to facilitating impact of the kind envisioned for this project.

Impact on policy and regulation - beneficiaries will include agencies and regulatory actors responsible for the governance of biomodifying technologies and policy makers promoting the social and economic benefits of innovative medical technologies. Research findings will have an impact by i) providing an evidence-base on the social, regulatory and organisational implications of current research trajectories and translational pathways for biomodifying technologies, ii) identifying what kinds of evidence are being required and what types of valuation practices are applicable for different applications of biomodifying technologies, and iii) highlighting significant dimensions of the experimental space for the three cases studies to inform policy and regulatory decision-making for emerging products and services.

Impact on healthcare professionals and managers - Healthcare professionals and NHS managers will benefit from having a robust empirically informed overview of the experimental space of biomodifying technologies that will allow them to identify prospective applications and the translational and regulatory landscape in which these research trajectories are embedded. This is pertinent for the future uptake of innovative biomodifying technology products and services and where healthcare professionals and managers will play a key role in translating biomodifying technologies from the experimental space to clinical adoption. Healthcare professionals and NHS managers will also benefit from the inclusion of NHS representatives in the expert workshops and data generation phases of the project.

Impact on patients and publics - beneficiaries include patients, charities and patient interest organisations, and various publics. Patients and patient organisations will benefit from the opportunity to have their needs and perspectives on the value of different biomodifying technologies, their associated translational pathways, and the types of evidence and valuation at stake solicited as part of the project's empirical data collection and used to inform the project outputs and findings. We have excellent links with the MHRA's Patient Forum which will provide an important vehicle for this. There will also be opportunities to participate in expert workshops and take part in upstream engagement with other key stakeholder groups. Findings will also have an impact by informing and encouraging participation in public debates on innovative biomodifying technologies.
 
Description Key findings (lay summary)

1) Uptake and utility of case study technologies

The uptake and use each of these technologies is significantly shaped by existing structures. For all three technologies, their most immediate application is as a research tool. Each example demonstrates a form of 'added value' to researchers in one of a number of ways: gene editing tools are more precise and controllable than the earlier generation of recombinant DNA tools, and of these, CRISPR-based systems are widely regarded as faster, cheaper and easier to use than Zinc finger, TALENS, or other mega-nucleases. Moreover, CRISPR systems have much greater capacity for adaptation and modification than other systems. Partly this is a result of there being multiple CRISPRs arising from different strains of bacteria, but it also reflects the long-accumulated expertise in manipulating nucleic acid and protein based structures in molecular biology laboratories across the world. In this sense, the rapid uptake and popularity of CRISPR gene editing cannot be seen merely as a result of the tool's inherent properties. Rather its close fit with existing networks of distribution (for example the non-profit repository AddGene that was initially set up to supply plasmids for 'traditional' genetic engineering was quickly able to supply CRISPR-cas9 kits as well) and the existing skill sets, equipment, databases, and analytic tools of many molecular and developmental biology laboratories in both the academic and commercial sectors (their 'ensemble of technologies' in Hackett's [2005] terms] (Martin et al 2020).

Induced pluripotent stem cells still typically require more 'green fingered' manual skills to successfully produce and maintain in culture. As such, their uptake has been more limited to research groups with expertise in stem cells, especially human embryonic stem cells (hESC) which were the established 'gold standard' for pluripotent stem cells. However, iPSC have a number of advantages over hESC in that they are seen as less ethically problematic as their production does not involve the destruction of a human embryo or the donation of human eggs (oocytes). The technology for producing iPSC has been made available in kit form and the licensing terms for non-commercial research use are considered not to be onerous (compared for example, to the more controversially high licensing fees initially charged by WARF for a licence to utilise hESC technology). Human iPSCs also have the same genetic material as the donor and so can be used to create 'patient-specific' cell lines that can be used to model diseases in vitro and also, potentially to screen small molecule drug compounds for toxicity. This could previously be done with human embryonic stem cells, but only through the technically and ethically more complex process of somatic cell nuclear transfer (also known as therapeutic cloning). For those research groups skilled handling hESC, adopting IPS technology has not been difficult and has not required much in the way of new skills or equipment. Uptake here has a somewhat different shape to that of CRISPR; where CRISPR is accessible enough to be used by almost every developmental or molecular biology laboratory in a high income country, production of iPSCs has been restricted to the more limited pool of research groups with skills in hESC or other stem cells, but thereafter the utility of iPSC as patient-specific disease modelling tools has made them very popular with a wider range of researchers studying a range of human disease. In the latter case, these researchers tend to obtain human iPSCs through collaborations with colleagues who have the skills to make and nurture the cells, or from commercial suppliers. At the time of our study, we did not find a large proportion of scientists sourcing IPSC lines from recently established biobanks such as HipSci (MRC/Wellcome Trust) or the European Bank for induced Pluripotent Stem Cells (EBiSC).

Both gene editing and iPSCs have been attractive to researchers because they enable them to increase the capacity of a research group- whether in the academic or commercial sector- to carry out experiments. This may be by doing things faster- for example, many respondents reported that gene editing with CRISPR is much quicker to do than with recombinant DNA or older gene editing tools, something that is also reported in the scientific literature. It may also be through making something technically or logistically more simple, such as creating patient specific stem cells from a simple skin or hair biopsy. These altered capacities can translate directly into rewards -a greater experimental capacity can lead to more papers for academic researchers or faster knowledge production, while for commercial firms, being able to produce new knowledge can be captured by intellectual property, or simply by being able to position themselves as part of new and emerging markets. Moreover with both technologies their popularity as research tools gives rise to immediate commercial growth in the 'picks and shovels' sector- that is supplying tools, reagents, kits, equipment and biological materials to both academic and commercial scientists.

3D bioprinting involves components that are more heterogeneous: the printer itself, a bio-ink (a gel-like holding medium in which living cells are suspended), Computer Assisted Design software to design the three-dimensional construct to be printed, and potentially, specialised biomaterials unto which the bio-ink can be printed. As such, 3D bioprinting needs more multi-disciplinary collaboration, even at the level of basic research, to enable it. It is a much younger sector than genetic modification or stem cell science, and to a larger extent new research groups have had to coalesce around the technology, bringing together people with expertise in cell biology, engineering and materials chemistry. Some groups also have software design skills although this is rarer and others commonly use 'off the shelf' software. Instead of regarding 3D printing as a faster or easier advance on an existing technology, its advantage lies more in adding a previously unavailable capacity- the controlled creation of three-dimensional structures with living cells (in this it is different from, for example existing 3D bioreactors, or bioscaffolds). The capacity for design is similar to what we see with CRISPR, and again the immediate advantage for early adopters is the capacity to do previously unfeasible experiments. As a younger sector most of the commercial activity - especially in the UK -is focused on supplying equipment- bioinks and bioprinters. At the same time, there is still considerable experimentation in the sector with many academic groups designing their own bioprinters, or customising commercial models with their own features such as modified printer nozzles (Bicudo, Faulkner and Li 2020). There is also an 'open source' software movement designing freely available software for designing 3D bioprinted constructs, although to date it is mainly in competition with generic CAD software rather than purpose build 3D bioprinting design programmes, which remain rare. This variability, with many competing forms of bioprinting such as laser guided, inkjet, electrospraying and electrospinning, as well as bespoke machines and tools is characteristic of a young, emerging sector where the technology has yet to stabilise into a dominant form or specific form associated with particular applications.

Translation of case study technologies

Translation- and specifically deciding which of the many potential areas of application of each technology are worth pursuing - is another domain where the identity of our case study technologies as 'upgrades' on previous technologies or inheritors of particular promises and potential uses is significant. Each of these technologies has been situated within existing translational ideals; such as an existing commitment to using cultured human cells to replace damaged tissues or replacing deleterious genes with non-pathogenic variants using gene-altering technology. Feasibility of specific applications is also shaped by existing material and organisational features, themselves often the result of previous experimental approaches: applying any of these technologies to treat a particular disease, or stratified patient population, is much more likely to be seen as achievable if it builds on existing professional skill sets, manufacturing and delivery capacities, reimbursement mechanisms, and facilities, than if it requires new or radically revised capabilities. In this regard, the UK has already made considerable investment in translational infrastructure for cell and gene therapies, but is less attuned to the needs of the younger 3D bioprinting sector.

The development of biomodifying technologies as tools for pharmaceutical R&D is both an important source of mid- term income for the field and an opportunity for SMEs to experiment with innovative manufacturing and scale up technologies without the high level of risk management needed for human clinical applications.
There may be opportunities for productive interactions with regulatory agencies in this space, for example to develop standards of equivalence that would allow data from novel iPSC-derived or 3D cell cultures or gene modified animal models of disease to be included in regulatory submissions in place of current preclinical animal studies, and in time potentially even phase 1 human trial data. Such measures would support the growth of the market for biomodifying technologies and foster the expertise and know-how for future clinical applications.

In terms of what makes a good therapeutic target, there is no single determining factor. Rather the range of 'good targets' are formed through different sets of justifications and considerations, which sometimes overlap but are also sometimes in competition. By way of illustration, iPSC are being developed as a cell replacement therapy for Parkinson's disease (PD) but also as a source of megakaryocytes to produce platelets. The PD application is justified in terms of it being an area of high unmet need, a well characterised disease, needing small number of actual cells (congruent with existing manufacturing capacity) and where protocols and pathways to clinical trials exist as cell therapy has previously been tried with foetal stem cells in the 1990s and 2000s. Platelets, to support blood clotting in people with injuries causing bleeding, is also an area of need, albeit one where there is existing supply through donor blood derived platelets, but here the advantages are framed in terms of platelets having no nuclear DNA, and thus having very limited risk of the cells carrying an undesirable genetic mutation, having a short duration in the body, and thus avoiding concerns about uncertain long term outcomes, and being adaptable to specific niche areas- such as creating platelets that do not trigger an immune response in people who cannot accept unmatched donor platelets. This latter step would also involve genetic modification of the platelet producing megakaryocyte cells during the manufacturing process. Similarly, within gene editing there remain a range of different visions of what constitutes a good target. Blood disorders such as thalassemia and sickle cell disease are one form of good target because the blood cells can be removed from the body, modified in the laboratory or bioprocessing facility and the returned by transfusion. This enables quality control of the modified cells before transfusion, which remains easier than trying to track and monitor gene editing delivered directly to a patient's internal organs such as the liver or lungs.

Overall, the current criteria for assessing good targets can be summarised as:
• Manageable size of cell population needed to treat
• Speed of production and mode of delivery
• Well-characterised disease
• Vector-selection strategy or characterisation of viable delivery mechanism
• Well-defined subpopulation with unmet need
• Product has capacity to produce clear benefit as defined by QALY or other relevant HTA measure
• Stable product that meets regulatory requirements

Current 'good targets' tend to be relatively narrowly defined indications, favouring orphan or rare diseases, or tightly defined subsets of common diseases. These small initial indications can serve as 'niches' where current manufacturing solutions demonstrate sufficient utility to be worthwhile. As 'next generation' manufacturing solutions come online, increased production speed and scale will support development of biomodifying therapies for larger patient populations. However, it will be important to avoid organisational arrangements, procedures or regulations that unintentionally create 'lock-in' to existing techniques. Developers and regulators should therefore consider what evidence requirements might be necessary and appropriate to enable biomodifying therapies, initially approved for tightly defined sub-populations of disease, to be made available on the NHS for patients with other forms of the same condition. Moreover, it is important to consider whether there needs to be a single 'winning' therapeutic modality for any one disease (e.g. advanced macular degeneration) or biomodifying technology (e.g. iPS 'cell therapy' with a transient effect and limited persistence in the body vs iPS regenerative medicine with long term effects and extensive durability in the body) or whether multiple routes to success can be supported by policy resources.
Innovation policies that continue to assume that successful technologies follow a linear path from basic research to application risk failing to support potentially viable products and services that require considerable 'learning by trying'.

Such innovations will sometimes need to go back to an 'earlier' stage of development in order to solve problems and progress. In this regard attempts to create 'institutional readiness' in recently established ATMP clinical centres (ATTCs) in the UK recognise the importance of the organisational changes and learning needed for ATMPs.

Commercialisation

The successful marketing approval and authorisation of CAR-T therapies is an important marker for the application and commercialisation of cell and gene therapies. It not only signals the first serious engagement of 'big pharma' plyers like Novartis in bringing cell and gene therapies to the clinic, CAR-T act as something of a bellwether for cell and gene therapies. There are currently 'next generation' CAR-T products made using gene editing and/or induced pluripotent stem cells in development, so the future prospects for CAR-T are directly relevant to our case study technologies. Much may depend on whether these next generation products can reduce manufacturing complexity and costs, and whether the CAR-T modality can show similar levels of efficacy against solid tumour cancers as it currently does against blood-based cancers.

In this regard, it is important to remember that each of our case study technologies is in competition with earlier, and in some cases newer options. For example gene therapies, suing recombinant DNA technology are finally coming onto the market (such as Luxturna, Zolgensma and so on) after years of development. Companies that have spent years developing gene therapy products are not going to abandon all that sunk capital of time, finance, scale-up, optimisation of manufacturing, and clinical trials juts because gene editing comes along. Rather gene therapy products that were near market will be supported to marketing authorisation and approval, while companies investigate gene editing as a research tool and potentially as an avenue to next generation therapies. Previous state support to overcome the viral vector manufacturing bottleneck was discontinued due to lack of demand. The bottleneck is now considered pressing, but additional commercial manufacturing capacity is starting to appear. Support for market-led initiatives to address this may be more effective than attempting to predict demand.

Similarly, many academic continue to work with hESC alongside their iPSC work, often running experiments in parallel, as hESC remain better characterised are still the 'gold standard' for pluripotency. Commercial developers have been more wary of hESC, especially as they are often seen as a 'non-starter' for the large US market, but nonetheless some hESC therapies are still in development. There is also considerable ongoing work using other types of stem cells- notably adult mesenchymal and haematopoietic stem cells, as well as the newer technology or transdifferentiation, which takes cells from one lineage to another without going through a pluripotent embryo-like state. At present, transdifferentiation is at a very early stage of development and is much less translationally advanced than IPSC. Nonetheless, in the longer term it may prove attractive as one of the persistent worries about HESC and iPS therapies is that a differentiated cell might revert to its pluripotent form in vivo with an associated risk of tumour formation. 3D bioprinting has a less obvious competitors, but it to be successful it will have to outperform previous tissue engineering products.

Here it is important to recognise that the acceleration of work- and publishing outputs- that results from the utility of gene editing, 3D bioprinting and iPSC in the research setting does not translated into a similar speed of translation into practical products. In part this is because the process of application is not an evaluation of the general utility of the technology itself but of the technology as a solution to a particular problem e.g. gene editing for cystic fibrosis or iPSC for macular degeneration. It is not only that the feasibility of applying 'that technology for that problem' must be worked out in practice through experimentation, but even when proof of concept is demonstrated in the laboratory, it much then be scaled-up and the manufacturing optimised for human clinical use- for example in Good Manufacturing Practice accredited facilities. This additional level of requirement is something that we do not witness to the same extent in non-medical areas such as agricultural uses of gene editing. Moreover, scaling up a laboratory-based procedure to an industrial one is not a simple engineering task- it is itself a further round of experimental and uncertain work, that requires time, investment, and above all, trial and error learning. The rate of translation of biomodifying technologies is partly determined by the pace of innovation in supporting technologies such as biomaterials and automation, and in quality management systems (QMS), supply chain management et cetera.

Policy support should extend to promoting and supporting innovation in these areas as well, for example through co-ordinated RCUK funding or collaboration between the High Value Manufacturing Catapult and the Cell and Gene Therapy Catapult.

Regulation and governance considerations affecting case study technologies

Induced pluripotent stem cells fit most readily into existing regulatory categories designed for cell-based therapies, and they are often seen as a replacement for treatments previously being developed with human embryonic stem cells. Gene editing is also commonly regarded as falling within the EU Advanced Therapy Medicinal Products regulations, but there are questions about how well tools such as CRISPR-Cas9 are actually captured by existing legislation designed to regulate recombinant DNA technology (Mourby and Morrison 2020). 3D printing of cells and tissues is the most 'disruptive' of the three technologies in regulatory terms. The potential for localised customisation of bioprinted implants for individual patients raises questions of manufacturing liability that are rarely seen with existing cell therapies or transplants and it has been more appropriate to look to recent regulatory debates around 'point of care' manufacturing at hospital sites.

In this, we have liaised with the MHRA and are hoping to contribute to their ongoing work and planning in this area. This contribution will persist beyond the lifetime of the funded project.

Translation, and commercialisation of each of these technologies is at different stages of maturity, but they all face socio-technical challenges in making treatment deliverable. As an example, setting standards such as the acceptable parameters for critical quality attributes of a cell or gene based medicinal product requires technical expertise, but it also requires negotiation between academic and commercial developers of the therapy and regulators. It is both social and technical, as it requires coming to agreements about what can be measured and known, what it is important to measure and know, and what degrees of risk and of uncertainty are tolerable, depending on the severity of the disease being treated, the availability of alternative interventions, and the degree of anticipated patient benefit. It is important to avoid premature 'lock-in' to design standards: Design standards for components (clinical grade cell lines, bioinks, gene editing constructs, bioreactors, bioprinters) are necessarily less flexible than procedural standards, but as product and process are so closely linked in biomodifying technologies, design standards will need to be revisited and reviewed periodically to ensure they do not inhibit positive improvements in how treatments are stored, administered or otherwise tailored to patient needs.

The UK has historically been a leader in developing models for regulating socially and technically complex technologies such as IVF, and advanced therapies presents an opportunity to extend this exercise of 'soft power' by developing and promoting internationally accepted standards of practice. By way of illustration, at present most companies developing cell therapy produce their own protocols for collecting starting material, storing and thawing frozen product, measuring and reporting outcomes. If cell therapies become more widely used for a range of diseases, multiple different and incompatible requirements for each different product could put an unsustainable logistical burden on the health service.

However, as a centralised provider the NHS could potentially be in a position to develop standardised protocols for collection, storage, thawing and reporting on cell therapies. This in turn would incentives manufacturers to adopt these standards to facilitate access to the NHS as the major healthcare provider in the UK, thereby encouraging broader adoption of the same standards by other providers in other jurisdictions. The international scope of technology development and scientific research means that standard setting is only likely to gain traction if undertaken in collaboration with strategic international partners.

The following policy recommendations for regulation have been identified through the course of this project:

• It is important to consider how the mix of incentives that are currently shaping translational research with biomodifying technologies aligns with NHS England strategic priorities and the mid-term goals of other devolved health authorities.
• Developers and regulators should consider what evidence requirements might be necessary and appropriate to enable biomodifying therapies, initially approved for tightly defined sub-populations of disease, to be made available on the NHS for patients with other forms of the same condition.
• Policymakers should consider how schemes such as Early Access to Medicines (EAMS), the Accelerated Access Review's 'transformative designation' or the orphan drug designation might best respond to the possibility of several competing high cost biomodifying therapies applying to these schemes in relation to the same, or very similar indications.
• Post-Brexit reforms need to decide whether to support one or multiple production strategies: mass production, mass customisation, and redistributed manufacturing need different organisational structures and require different skill sets from the workforce, and face different logistical and regulatory hurdles to viability. Mass production of therapies for certain conditions will remain the most commercially attractive option, while others could benefit from one or more of the forms of customisation presented here.
• Future policy developments, especially in view of the UK's departure from the European Union, need to evaluate whether to support one or several forms of manufacturing within the UK healthcare sector, as each requires different forms of support.
• Procedural and performance standards need to be capable of evolving over time: ATMP pharmacovigilance, whether one-off studies or registries, should incorporate evaluations from patients and clinicians in addition to standard measures of efficacy and reporting of adverse events. The combination of precision medicine and patient-centred medicine supports ongoing evaluation of the outcomes of treatments, which creates a feedback loop into procedural and performance standards for biomodifying therapies. The three UK Advanced Therapy Treatment Centres should identify workflows across companies and hospitals that support this ongoing refinement of the entire 'pathway to the patient'.
• Users, both patients and clinicians have an important role to play in the design, development and evaluation of biomodifying therapies. This is especially pertinent when considering the evaluation of conditionally approved therapies where multiple types of data (observational, registry, patient record, PROMs) could be integrated.

References
Bicudo, E., Faulkner, A. and Li, Phoebe (2020) Patents and the experimental space: social, legal and geographical dimensions of 3D bioprinting. International Review of Law, Computers & Technology, 35:1, 2-23, DOI: 10.1080/13600869.2020.1785066

Martin, P., Morrison, M., Turkmendag,I., Nerlich, B., McMahon, A., De Saille, S. and Bartlett, A. (2020) Genome editing: the dynamics of continuity, convergence, and change in the engineering of life, New Genetics and Society, 39:2,219-242, DOI: 10.1080/14636778.2020.1730166

Mourby, M. and Morrison, M. (2020) Gene therapy regulation: could in-body editing fall through the net? European Journal of Human Genetics, 28:979-981, https://doi.org/10.1038/s41431-020-0607-y
Exploitation Route As we write up and disseminate our findings we are be able to provide a nuanced account of the advantages and limitations of the UK landscape for developing Biomodifying technologies to policy makers, regulatory, academic and companies working in this space, and to identify areas for strategic attention. In addition the report we are working on from our joint forum with patient groups and charities on the value of cell and gene therapies and bioprinted implants which will provide a basis for (further and more focused) dialogue between scientific and technical experts on the one hand and the end users of Biomodifying technologies, the patients, on the other.
Sectors Healthcare,Government, Democracy and Justice,Manufacturing, including Industrial Biotechology,Pharmaceuticals and Medical Biotechnology
URL https://www.regmednet.com/esrc-biomodifying-technologies-policy-briefing-1
 
Description Narrative impact Statement 2022 Throughout the duration of the project, team members have participated in several engagement and policy making events. Professor Andrew Webster and Dr Michael Morrison participated in a two-day workshop on the governance of gene editing for human clinical applications organised by the Working Party on Biotechnology, Nanotechnology and Converging Technologies (BNCT) of the Organisation for Economic Co-operation and Development (OECD) in conjunction with the German Federal Ministry of Education and Research (BMBF) and the Korean legislation Research Institute (KLRI). Professor Webster contributed text to the final text of the policy report produced by the workshop while Dr Morrison provided a recently published paper on regulation of clinical gene-and-cell therapies, on which he was a co-author, to the OECD working group, which was also cited in the final report. Dr Phoebe Li has been interviewed for Al Jazerra on the legal and ethical implications of 3D bioprinting and took part in a scenario workshop on additive biomanufacturing and 3D printing for human application organised by the Rathenau Institute (Netherlands) and hosted at the European Parliament. Professor Faulkner has been interviewed about the regulatory challenges posed by 3D bioprinting by 3Dmednet, a popular website for the academic and industrial bioprinting community. Professor Webster took part in an expert roundtable meeting on gene editing organized by the UK Department for Business, Energy and Industrial Strategy (BEIS). Several team members contributed feedback and/or suggestions to the recent POSTNote on human germline gene editing and a further POSTNote on 3D printing in medicine. Dr Morrison was also invited to speak to an international audience of scientists and researchers about the governance of CRISPR at joint meeting of the International Centre for Genetic Engineering and Biotechnology (ICGEB) and the Joint Research Centre (JRC) of the European Commission and has also contributed a podcast (with Dr Jessica Bell) on data protection issues in induced pluripotent stem cell banking and research for the Global Alliance for iPSC Therapies (GAiT). Dr Morrison and Professor Webster both contributed entries in the 'ethics/policy/regulatory' category to Future Science Group's online glossary of regenerative medicine. Through all of these events, we have engaged with audiences outside our disciplinary area, many of whom are actively working with our case study technologies or are involved in their oversight. In 2020 we held four engagement and dissemination workshops: one for each case study technology and a jointly hosted Patient Forum with the MHRA. Our one-day expert events brought together academic scientists, representatives of commercial firms, regulators, health technology assessment professionals, clinicians and NHS staff. The project team presented several issues that our work has identified as key: making 'good targets' in clinical research, manufacturing and scale up, business models and reimbursement, and governance. On each topic, we received feedback and discussion with a mixed group of experts, all of whom represented primary audiences for our research, and concluded with an open discussion of future directions and outstanding issues. The input from these workshops, served to calibrate key project findings, which were then disseminated through a series of policy briefing documents. There have been three briefings to date, each hosted on an open access basis by the Future Medicine group through the RegMedNet and 3DMedNet websites and mailing lists, which reports apx. 17,000 registered members across academia, industry and healthcare. Each briefing focuses on a specific aspect of the project's initial research questions: Briefing 1# Making 'good targets' for translational research. Published July 2020 Briefing 2# Accelerating innovation: speed and timing in translational research. Published September 2020 Briefing 3# Trends in customization and personalization of advanced therapies. Published January 2021 The following impact data for 2020-2021 was provided by the Future Medicine group: Briefing 1: 312 page views, 2753 PDF views Briefing 2: 267 page views, 2063 PDF views Briefing 3: 254 page views, 400 PDF views. A fourth briefing is planned for later in 2022. We expect that the published briefings will continue to garner attention and serve as a longer-term vehicle to disseminate project findings to key stakeholder groups. We have also produced a series of short articles conveying key ideas and recommendations from the project in an accessible, jargon-free format through the Open Access Government platform. These articles can be summarised as follows: 1) "The promises and challenges of biomodifying technologies for the UK" (published July 2019). Introduces the project and its key aims. 3,450 reported page views by 2021 2) "Targeted policy support for emerging biomedical innovations" (published July 2020). Reports project policy recommendations for translation and commercialisation of biomodifying technologies in the UK. 718 page views reported by 2021. 3) "Prospects for personalised medicine using advanced therapies" (published January 2021). Describes current trends in personalised and customisation of biomodifying technologies and their implications. 265 page views reported by 2021. 4) "Building readiness for innovative health technologies" (published May 2021) introduces an model of 'readiness' for disruptive technologies like 3D bioprinting and iPSC that operates at the level of technologies, institutions and national systems 5) "Japan: A case study of national 'readiness' for regenerative medicine" (published September 2021). With Dr Maki Umemura (Cardiff University Business school). Applies the readiness model to analyse how Japan has adapted its national innovation system for advanced therapies 6) "Australia's medical innovation approach: is it suitable for regenerative medicine?" (published January 2022). With Dr John Gardner (Monash University). Applies the readiness model to analyse Australian policy and infrastructure development for advanced therapies. The MHRA Patient Forum allowed us to engage with a range of representatives of medical charities and support groups as well as some non-affiliated patients who attended. The Forum allowed us to present information about the three technologies to a self-selected audience of non-specialist who wanted to learn more about them, and to raise three specific issues from our own analysis for further discussion: customisation / personalisation of therapies, data collection and ownership for long-term outcome monitoring, and risk and uncertainty associated with novel therapeutics. A report from the workshop has been produced. The initial text of the report was written by PI Morrison and edited for accessibility by expert patient Katharine Keats-Rohan. This text was then subject to multiple rounds of review by the MHRA and the workshop participants to ensure a final, co-produced report that was approved by all parties. The report was published online and open access through Zenodo (DOI: 10.5281/zenodo.5243793). To date Zenodo reports 479 views and 254 downloads of the report. Ultimately, there is no single 'impact' message, but rather all these events have been opportunities to put our understanding of the dynamics of innovation across our case study technologies to use in shaping conversations and trying to ensure that our insights and issues that we have identified as important are raised in these contexts. The future application of each of our case study technologies depends on a complex, multi-faceted array of skills, institutional arrangements, governance mechanisms, technical and scientific achievements, and political, financial and societal factors. Therefore, there is no one group or knowledge set that has sole responsibility; rather it must necessarily be a conversation between different groups and perspectives. Through these engagements, we have tried to be impactful by contributing to that conversation and by ensuring that we reached all of the core groups involved. Part of our utility is that the people working on this project are not committed to one of these perspectives e.g. basic laboratory research, health technology assessment, or working for an SME, but can bridge the institutionally fragmented pathway from discovery research to product approval and assess the bigger picture, while retaining an understanding of the situated needs and perspectives of different stakeholder groups involved. Several further manuscripts from this project are in various stages of development and should see publication over the course of 2022 and 2023. These and forthcoming and planned presentations and policy briefings will further extend and enrich this dialogue.
First Year Of Impact 2018
Sector Healthcare,Manufacturing, including Industrial Biotechology,Pharmaceuticals and Medical Biotechnology,Other
Impact Types Societal,Policy & public services
 
Description Contributed to GAiT podcast on Regulatory considerations pertinent to iPSC Scalable Manufacture and Allogeneic Therapeutic Development
Geographic Reach Multiple continents/international 
Policy Influence Type Influenced training of practitioners or researchers
URL https://soundcloud.com/stephen-sullivan-161592538/regulatory-virtual-workshop-3rd-october-2019/s-im0...
 
Description Dr Phoebe Li acted as a pre-publication peer review of UK IPO report on 3D printing
Geographic Reach National 
Policy Influence Type Contribution to a national consultation/review
URL https://www.gov.uk/government/publications/3d-printing-and-intellectual-property-futures
 
Description Participation in OECD Expert Workshop Gene editing for advanced therapies: governance, policy and society
Geographic Reach Europe 
Policy Influence Type Contribution to a national consultation/review
URL https://www.innovationpolicyplatform.org/system/files/imce/OECD_Gene%20editing%20expert%20meeting_dr...
 
Description Prof Webster invited to participate in BEIS roundtable on genome editing
Geographic Reach National 
Policy Influence Type Contribution to a national consultation/review
URL https://www.york.ac.uk/satsu/news-events/news/genomeeditingresearchlandscape/
 
Description A new agenda for understanding industrialised tissue-based products
Amount £24,745 (GBP)
Funding ID 218807/Z/19/Z 
Organisation Wellcome Trust 
Sector Charity/Non Profit
Country United Kingdom
Start 09/2019 
End 03/2021
 
Description Fondation Brocher workshop
Amount SFr. 6,000 (CHF)
Organisation Brocher Foundation 
Sector Charity/Non Profit
Country Switzerland
Start 07/2017 
End 12/2018
 
Description Responsive mode grant
Amount £340,000 (GBP)
Funding ID RPG-2017-330 
Organisation The Leverhulme Trust 
Sector Charity/Non Profit
Country United Kingdom
Start 09/2018 
End 08/2021
 
Title Biomodifying technologies anonymised interview transcript collection 
Description The anonymised transcripts from qualitative interviews with i) academic scientists working with iPSCs, ii) UK companies working with, or whose business was relevant to the iPSC case study, iii) academic scientists working on 3D bioprinting, iv) UK companies working with, or whose business was relevant to the bioprinting case study have been uploaded to a managed access Biomodifying technologies project repository on the UK data service ReShare platform. Copies of the interview questions for each case study, blank copies of the consent forms, and participant information sheets for the study have also been uploaded. I am still in the process of anonymising the remaining transcripts of interviews with regulators and gene editing scientists and companies. When these are complete the repository will be published and available through the ReShare platform. 
Type Of Material Database/Collection of data 
Year Produced 2021 
Provided To Others? No  
Impact This dataset underpins the main qualitative research of the biomodifying technologies project and will be referenced in several forthcoming publications. 
 
Description Ethics in advanced therapy manufacturing study for Future Targeted Healthcare Manufacturing Hub 
Organisation University of Vienna
Country Austria 
Sector Academic/University 
PI Contribution This collaboration with the FTMH Hub drew on my expertise and knowledge of advanced therapies and the societal issues associated with their manufacture and delivery to contribute to a report that allowed the Hub to address an area where they had limited in house expertise and advice. A substantial part of this expertise derived from my leadership of the ESRC Biomodifying technologies project and subsequent funded work that builds on this project (see Wellcome Trust and Leverhulme Trust funded work in the 'Further funding' section)
Collaborator Contribution The FTMH Hub provided funding from their overall funding (EP/P006485/1) to buy a portion of the research time of the myself and my (non-project) colleagues Dr Burton and Professor Prainsack and to enable us to hire a postdoctoral researcher, Dr Maria Kilaldi, to undertake the bulk of data collection and drafting work of the report.
Impact I worked with Dr Saheli Datta Burton (UCL), Dr Maria Kilaldi (UCL) and Professor Barbara Prainsack (University of Vienna) to produce a report on "Responsible Personalised Medicine: Exploring the Ethical, Legal, Social, Political and Economic Issues of Manufacturing, Distribution, Access and Reimbursement" for the Future Targeted Manufacturing in Healthcare Hub. Dr Burton and myself continue to be involved with the Hub in an advisory capacity.
Start Year 2021
 
Description "Inventing biomedical accuracy: between customisation and personalisation of advanced therapies" British Sociological Association Medical Sociology conference 2021 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Postgraduate students
Results and Impact Project members Morrison and Faulkner recorded a video presentation on the topic "Inventing biomedical accuracy: between customisation and personalisation of advanced therapies" for the British Sociological Association Medical Sociology (BSA MedSoc) conference 202. Videos were made available online for several weeks prior to the start of the event and virtual panel discussions of presentations were held each Friday of September 2021. It is difficult to gauge the number of views of our specific video as this data was not provided by the event organisers but any academic or public sector participants who signed up to MedSoc 21 could access the material at any time that suited for over a month.
Year(s) Of Engagement Activity 2021
URL https://www.britsoc.co.uk/media/25609/medsoc2021_conf_prog.pdf
 
Description 3D Bioprinting workshop 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact We organised a one day workshop at the Said Business School, Oxford for key expert audiences for the findings relating to 3D bioprinting from the Biomodifying technologies project. The audience was deliberately selected to include academic scientists and engineers, working on translational applications of 3D printing, representatives from UK companies working in the bioprinting space, and professionals working on the regulation, funding, and infrastructural support of distributed manufacturing in the UK. This included representatives from the Medical Research Council, Medicines and Healthcare products Regulatory Agency, Innovate UK, the UK, Knowledge Transfer Network, and the UK redistributed manufacturing in healthcare network (RiHN), as well as from hospitals, companies and university research groups. A series of short presentations were delivered by the project team presenting findings and ideas from the project concerning: i) introducing the project, ii) the 'Experimental space' of 3D bioprinting , iii) Global trends in bioprinting: hardware/software, business landscape, patents, iv) Current IP issues in bioprinting - patentability, methods patents, ethical licensing, v) Challenges of the regulatory and liability environment, and vi) a report of preliminary findings from the MHRA patient workshop held by the project on 30th January. Each session was followed by an audience Q&A with a further opportunity for in-depth discussion on all issues in the last part of the day. Georgi Makin from the website 3D med net which reports on developments in 3D bioprinting to a global audience of scientists and clinicians, was also present and has agreed to write up the event as a post for website along with a profile of project member Professor Alex Faulkner.
Year(s) Of Engagement Activity 2020
 
Description Biomodifying Technologies project website 
Form Of Engagement Activity Engagement focused website, blog or social media channel
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact The Biomodifying technologies project website provides a description of the project, what we hope to achieve and who this research is intended to benefit. It provides an online point of reference for anyone interested in learning more about our work or who is considering responding to an invitation to be interviewed as part of our fieldwork. The website also provides information about the project team members, has a 'contact' facility and a page where we can disseminate news and activities from the project.
Year(s) Of Engagement Activity 2017,2018
URL https://www.biomodtech.com/
 
Description Biomodifying technologies Twitter account 
Form Of Engagement Activity Engagement focused website, blog or social media channel
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact The Biomodifying technologies Twitter account is run by Dr Bartlett at the University of York. It serves multiple functions including raising awareness of the project among relevant stakeholder communities, collecting up-to-date information on academic, commercial, regulatory and policy developments in the fields of genome editing, induced pluripotent stem cells, and 3D bioprinting, and serves as one site where actors from each of these research areas who are active in the UK can be identified. The account will also be used to promote project events and outputs (e.g. policy briefs) as these become available during the course of our work.
Year(s) Of Engagement Activity 2018
URL https://twitter.com/BioModTech
 
Description Dr Phoebe Li interviewed by Al Jazeera TV on ehtical and legal challenges of 3D bioprinting 
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 Dr Phoebe Li (Sussex) was interviewed by Al Jazeera's English language channel for a news item about Russian plants to put a 3D bioprinter in space. Dr Li was asked comment on the legal and regulatory implications of bioprinting and of distributed manufacturing of medical devices and implants being carried out in extra-terrestrial- domains- that is space shuttles and orbiting platforms. Dr Li's segment of the item begins at 2m 10s into the YouTube clip. As of March 2019 the clip had over 3000 views in addition to the audience for the original audience.
Year(s) Of Engagement Activity 2018
URL https://www.youtube.com/watch?v=-VxFaB8yR6o
 
Description IngentaConnect profile piece of the project and its aims 
Form Of Engagement Activity A magazine, newsletter or online publication
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Policymakers/politicians
Results and Impact The Impact publication is promoted to 35,000 individuals worldwide within academia, government, research centres, funding agencies and research councils. This specific issue is 80 pages in length, with other articles from researchers working in Social Sciences from across the world featuring alongside the project-specific article.
There are over 24,000 Universities, institutes and companies in Europe and 21,000 in the USA, from both the public and private sector, who are registered for their staff to access content from this resource. The article will be free to read and download, open access, on IngentaConnect and as a result of this, will be available through Google Scholar, EBSCO, Primo Central and World Cat Discovery, while also receiving a Crossref DOI and be deposited in Portico - one of the leading deep repositories.
Year(s) Of Engagement Activity 2019
URL https://www.ingentaconnect.com/content/sil/impact/2019/00002019/00000001/art00021
 
Description Interviewed for article on CRISPR in Chemistry World 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Other audiences
Results and Impact I was quoted by journalist Anthony King for a short article in Chemistry World on a social media analysis of public perceptions of gene editing. I judged this to be a useful contribution because, as my comment indicated, broad social medial analyses risks flattening the entire complexity of discussions by different groups on different aspects of gene editing into a simplistic 'for' or 'against' discourse. I felt that the analysis being discussed was technically skilled but methodologically flawed and did not give a nuanced picture of attitudes to gene editing. This is a small but illustrative example of how being invited to contribute to the wider conversations around our case study technologies can help shape, and hopefully improve, those conversations as part of the wider impact of the project.
Year(s) Of Engagement Activity 2019
URL https://www.chemistryworld.com/news/twitter-reveals-growing-global-public-anxiety-about-crispr-gene-...
 
Description Invited presentation at the ICGEB-JRC Workshop on "Genome Editing applications and beyond" 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Project PI Michael Morrison was selected as an invited speaker to a workshop organised by the International Centre for Genetic Engineering and Biotechnology (ICGEB) and the Joint Research Centre (JRC) of the European Commission. The workshop was titled "Genome editing applications and beyond" and was held at ICGEB headquarters in Trieste, Italy on 19-22 November 2019. The presentation drew on project work on regulation biomodifying technologies and was entitled "Continuity and disruption in the regulation of gene editing" and set out how human clinical applications of gene editing are likely to be overseen under existing European regulations for gene therapy and GMOs, and with what potential implications . The audience was an international gathering of scientists working on gene editing from Europe, Asia, Africa and Southern/Central America and there were several follow-up questions and discussions with me following the presentation.
Year(s) Of Engagement Activity 2019
URL https://www.icgeb.org/icgeb-jrc-workshop-genome-editing-applications-and-beyond
 
Description MHRA Patient Forum on Biomodifying technologies 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Patients, carers and/or patient groups
Results and Impact We co-hosted a one day workshop with the Medicines and Healthcare products Regulatory Agency (MHRA), utilizing their Patient Forum to present work from the BioModifying technologies project to more than 20 patients and patient group members as part of their existing Patient Forum. The day was split into two sections: in the morning session we introduce each case study technology: gene editing, 3D bio-printing and induced pluripotent stem cells. Each short presentation was followed by a Q&A session with the audience. The MHRA also gave a presentation on thier role and functions. After lunch we introduced three aspects of bio-modifying technologies identified by our research as likely to be of interest and impact to patients and carers: risk and uncertainty associated with novel therapies, personalisation or customization of treatment, and data collection and long term surveillance of recipients of novel therapies. Each short presentation was followed by audience responses and more in-depth group discussion of each issue raised. The event was also attended by representatives of the MHRA Innovation Office, Licensing, medical devices, Inspection, Enforcement & Standards, and Communications groups and by representatives from the National Institute for Biological Standards and Control (NIBSC) and the Cell and Gene Therapy Catapult.
Year(s) Of Engagement Activity 2020
 
Description Presentatio at special session on advanced therapies at EMBT 48th annual meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Presentation to European Bone Marrow Transplant Society on legal and ethical issues relating to advanced manufacturing of (stem) cell based therapies for haematological conditions. The event was a multi-stakeholder, virtual panel session and reached clinical staff, engineers, and industry staff attending the meeting. It was a useful opportunity to engage with some of the main stakeholder communities for biomodifying technologies project. The impact of this outreach is difficult to measure because its main aim is to raise awareness of issues that professionals need to take account of in their own work.
Year(s) Of Engagement Activity 2022
URL https://ebmt2022.abstractserver.com/program/#/details/sessions/183
 
Description Presentation on "CRISPR in Context: A critical historical perspective on the promise and utility of gene editing" 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Postgraduate students
Results and Impact Presented at a workshop entitled "We new utopiansgene editing and echoes of future life" hosted by the Research group Biotechnologies, Nature and Society at the Goethe University, Germany. The group was primarily composed of postgraduate (PhD and MA) students as well as other invited speakers from different disciplines, Law, Ethics etc.
Year(s) Of Engagement Activity 2019
URL https://lasst.uni-frankfurt.de/event/we-new-utopians-genome-editing-and-echoes-of-future-life/
 
Description Science and Technology Options Assessment Panel project: additive biomanufacturing 3D printing for medical recovery and human enhancement - Scenario workshop at European Parliament 
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 Dr Phoebe Li attended and participated in a scenario workshop at the European parliament organised by the Rathenau Institute (NL) as part of the STOA (The European Parliament's Science and Technology Options Assessment Panel) project: on "Additive bio-manufacturing, 3D printing for medical recovery and human enhancement". the workshop was held in September 2017. Workshop participants were presented with four scenarios of how 3D bio-printing and medical bio-manufacturing might operate in 2035 and asked to assess and evaluate these scenarios and provide constructive feedback on each. The aim of the workshop is to support the Members of the European Parliament and their Committee to understand potential trends in bio-printing and additive manufacturing for medical purposes in order to anticipate and plan for their policy impact.
Year(s) Of Engagement Activity 2017
 
Description Talk on Infrastructures of valuation in tissue based industries 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Postgraduate students
Results and Impact I presented the talk "Infrastructures of valuation: the social shaping of innovation trajectories in tissue-based industries", co-authored with Dr Edison Bicudo (formerly of the sister 'Governing biomodification' project and now at UCL) at the European Association for the Study of Science and Technology (EASST) conference, in Madrid, Spain (6-9 July 2022). The presentation was part of a session on "Pharmaceutical transformations: advanced therapies, personalized medicine and the remaking of healthcare" . This catalysed several productive conversations and an invitation to be part of future (now ongoing) work o social pharmaceutical innovation models.
Year(s) Of Engagement Activity 2022
URL https://easst2022.org/
 
Description Virtual presentation "Making Clinical and Commercial Value: Insights from the translation of biomodifying technologies" 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presentation to the joint European Association for the Study of Science and Technology (EASST) and the North American Society for the Social Studies of Science (4S) conference virtually hosted by the University of Prague. The presentation was part of the session on session 'Value in biomedicine: value creation of emerging technologies'
Year(s) Of Engagement Activity 2020
 
Description Workshop on Translation and Innovation in Induced Pluripotent Stem Cell Science 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact We organised a one day workshop at the Said Business School, Oxford for key expert audiences for the findings relating to iPSC from the Biomodifying technologies project. The audience was deliberately selected to include representatives from academic and clinical sectors working on translational applications of human iPSC, representatives from UK companies working in the iPSC space, professionals working on the regulation, funding, and infrastructural support of cell therapies in the UK. This included representatives from the National institute for Biological Standards & Controls, Cell and Gene Therapy Catapult, the Medical Research Council, Medicines and Healthcare products Regulatory Agency, the Health Research Authority as well as from a number of UK universities and companies and the Global alliance for iPS Therapies. A series of short presentations were delivered by the project team presenting findings and ideas from the project concerning: i) introducing the project, ii) uptake and dissemination of iPSC technology, iii) making 'good targets' for clinical translation, iv) commercialisation, scale up and manufacturing, v) report on the MHRA patent Forum discussion held by the project on 30 January, and vi) governance strategies and challenges. Each session was followed by an audience Q&A with a further opportunity for in-depth discussion on all issues in the last part of the day.
Year(s) Of Engagement Activity 2020
 
Description Workshop on clinical and translational gene editing in the UK 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact We organised a one day workshop at the Said Business school, Oxford for key expert audiences for the findings relating to human gene editing and gene therapy from the Biomodifying technologies project. The audience was deliberately selected to include academic scientists and clinicians, working on translational applications of gene editing, representatives from UK companies working in the gene editing/ gene therapy space, and professionals working on the regulation, funding, and infrastructural support of human-centred genetic technologies in the UK. This included representatives from the National Institute for health and Care excellence (NICE), the Cell and Gene Therapy Catapult, Genomics England / Dept. of Health and Social Care, and the Parliamentary Office for Science and Technology as well as from hospitals, companies and university research groups. A series of short presentations were delivered by the project team presenting findings and ideas from the project concerning: i) introducing the project, ii) The Rhetorics and realities of Gene Editing, iii) Good Targets and 'Low Hanging Fruit' in clinical translation, iv) a report of preliminary findings from the MHRA patient workshop held by the project on 30th January, v) Finance, Manufacturing, and current Bottlenecks, and vi) Governance of gene editing: Regulation and Reimbursement. Each session was followed by an audience Q&A with a further opportunity for in-depth discussion on all issues in the last part of the day.
Year(s) Of Engagement Activity 2020