The Pluripotent Stem Cells and Engineered Cell (PSEC) Hub
Lead Research Organisation:
University of Cambridge
Department Name: Clinical Neurosciences
Abstract
The ability to repair the injured or diseased human body with human pluripotent stem cell (hPSC) derived products is emerging as a realistic therapeutic option. However this brings with it a number of major challenges;
1) Can this be done safely?
2) Can this be done usefully and competitively? i.e. it the treatment as good or better than what is available; and
3) Can we do this at the scale needed to treat all patients that might require this type of therapy?
In this new hub we will work on these issues around two particular therapeutic agents. The first involves making dopamine nerve cells to replace those lost in the brain of patients with Parkinson's disease (PD). The second involves making a specific component of blood that is vital for clotting- namely platelets generated from hPSC-derived megakaryocytes. These two cell products and the diseases linked to their use are not seen as the primary output of this grant, but rather will serve to contextualise our work, which is primarily designed to build a platform by which any hPSC-derived therapy can be brought to the clinic. We have three major aims:
The first relates to acquired genetic changes seen in cells grown in the laboratory and what this means for the safety of our hPSCs/cell products. Every cell has DNA, which contains all our genes, and between cells, there will be natural variations along with random errors and mistakes. Many of these changes are of no consequence, while others may be potentially harmful. The question we need to answer is can we reliably detect the bad genetic variants in the hPSCs and the cells we produce from them, while also recognising those genetic changes that are unimportant and can be safely ignored. This is obviously not an easy question to answer, but what we will do, is look at the changes in the genes in a number of different hPSCs/products and then compare what we find with databases that contain information on the links such genetic changes have to known human diseases and cancers. This will allow us to develop guidelines, which will then hopefully be adopted by the relevant regulatory agencies and applied to any hPSC-derived therapy going to clinic.
Our second aim is to address the major issues related to how we can manufacture our hPSC-derived products in the numbers and quality required for clinical use in large numbers of patients. Currently, many of the protocols we have developed in the lab, work well, but the reagents we use are not of the grade needed for use in patients- so called GMP grade. Thus we will seek to develop the necessary clinically compatible protocols and then work out how we can scale up and scale out the manufacturing of the cells we are interested in developing so that ultimately all relevant patients could benefit from these types of treatment. For some diseases, this will be quite straight forward, as we only need relatively few cells to treat patients- e.g. 500,000 to a million cells for a single patient with PD. In other conditions, we will need millions and billions of cells, such as platelets, and this creates huge challenges for manufacturing. We will therefore work on ways to do this, such that we can reliably and reproducibly do this at the level needed for clinical use.
Our final major aim is to develop new techniques to make our cell therapies work better when transplanted. This will involve two main approaches; (i) Techniques to allow us to make the cells we want more efficiently, i.e. so we can manufacture large numbers of cells from our hPSCs as will be needed to treat patients and; (ii) Silencing critical molecules/proteins in the cells that trigger immune rejection, i.e. so that when the cells are grafted into patients their immune system will react to them less vigorously. This in turn will mean that we can use less aggressive immunosuppressive regimes to stop the grafts being rejected and by so doing reduce any side effects from these drugs.
1) Can this be done safely?
2) Can this be done usefully and competitively? i.e. it the treatment as good or better than what is available; and
3) Can we do this at the scale needed to treat all patients that might require this type of therapy?
In this new hub we will work on these issues around two particular therapeutic agents. The first involves making dopamine nerve cells to replace those lost in the brain of patients with Parkinson's disease (PD). The second involves making a specific component of blood that is vital for clotting- namely platelets generated from hPSC-derived megakaryocytes. These two cell products and the diseases linked to their use are not seen as the primary output of this grant, but rather will serve to contextualise our work, which is primarily designed to build a platform by which any hPSC-derived therapy can be brought to the clinic. We have three major aims:
The first relates to acquired genetic changes seen in cells grown in the laboratory and what this means for the safety of our hPSCs/cell products. Every cell has DNA, which contains all our genes, and between cells, there will be natural variations along with random errors and mistakes. Many of these changes are of no consequence, while others may be potentially harmful. The question we need to answer is can we reliably detect the bad genetic variants in the hPSCs and the cells we produce from them, while also recognising those genetic changes that are unimportant and can be safely ignored. This is obviously not an easy question to answer, but what we will do, is look at the changes in the genes in a number of different hPSCs/products and then compare what we find with databases that contain information on the links such genetic changes have to known human diseases and cancers. This will allow us to develop guidelines, which will then hopefully be adopted by the relevant regulatory agencies and applied to any hPSC-derived therapy going to clinic.
Our second aim is to address the major issues related to how we can manufacture our hPSC-derived products in the numbers and quality required for clinical use in large numbers of patients. Currently, many of the protocols we have developed in the lab, work well, but the reagents we use are not of the grade needed for use in patients- so called GMP grade. Thus we will seek to develop the necessary clinically compatible protocols and then work out how we can scale up and scale out the manufacturing of the cells we are interested in developing so that ultimately all relevant patients could benefit from these types of treatment. For some diseases, this will be quite straight forward, as we only need relatively few cells to treat patients- e.g. 500,000 to a million cells for a single patient with PD. In other conditions, we will need millions and billions of cells, such as platelets, and this creates huge challenges for manufacturing. We will therefore work on ways to do this, such that we can reliably and reproducibly do this at the level needed for clinical use.
Our final major aim is to develop new techniques to make our cell therapies work better when transplanted. This will involve two main approaches; (i) Techniques to allow us to make the cells we want more efficiently, i.e. so we can manufacture large numbers of cells from our hPSCs as will be needed to treat patients and; (ii) Silencing critical molecules/proteins in the cells that trigger immune rejection, i.e. so that when the cells are grafted into patients their immune system will react to them less vigorously. This in turn will mean that we can use less aggressive immunosuppressive regimes to stop the grafts being rejected and by so doing reduce any side effects from these drugs.
Technical Summary
We will deliver a platform of technologies and expertise that will enable new human pluripotent stem cell (hPSC) based therapies to more readily enter the clinic. This will be done around 2 major therapeutic areas - hPSC derived dopaminergic neurons and megakaryocytes. These exemplars will contextualise the overarching aim of the hub which is to further understand and develop the next generation of genetic and reprogramming tools needed to allow ANY hPSC-based product to progress to clinic. This will be done through 3 major research programmes: THEME 1 - CELL CHARACTERISATION AND STABILITY: We will assess the phenotypic consequences of specific genetic variants using our clinical exemplars, while also developing sensitive high-throughput detection methods for genetically variant cells. We will the use this information to optimise culture conditions to suppress the appearance of concerning genetic variant, while also producing lists of non-consequential genetic variants that we detect which we will then feed into international guidelines for adoption by relevant regulatory agencies. THEME 2 - IDENTIFYING AND MODELLING KEY DETERMINANTS OF MANUFACTURING OUTCOMES FOR HPSC PRODUCTS AND PROVIDING A REGULATORY ROADMAP: We will address issues to do with manufacturing cells in the quantity and the quality required, for clinical adoption using dynamic process models as well as novel protocols and reagents. This will include a pathway for regulatory integration, to facilitate clinical application and first in human studies. THEME 3 - UNDERSTANDING ROUTES TO DIFFERENTIATION: We will develop a new generation of genetically modified hPSCs with improved differentiation capabilities including purity, cell yield and reduced dependence on expensive media and/or complex cytokine cocktails as well as reducing their immunogenicity such that they can more easily be employed in the clinic without the need for major immunosuppressive anti-rejection therapies and their associated risks.
Planned Impact
The impact of our work has already been discussed in part in the section on "Academic Beneficiaries". In essence the work that will be done in this hub has the potential to enhance and transform the UK landscape around hPSC therapies and regenerative medicine in the broadest possible sense.
The hub will generate outputs that will define the significant acquired genetic changes seen in hPSCs and their products and what this means. This will then be used by regulatory agencies to develop guidelines that are likely to be adopted worldwide. This will impact on all hPSC based therapies being developed for clinical use anywhere in the world where there exists an overseeing national agency - e,g, MHRA; EMA, FDA and so on. Furthermore we will also start exploring not just the acquired genetic variation that comes with growing and differentiating these cells, but how this can be better controlled, especially during any manufacturing process and storage of the derived cell products. Finally we will start to explore this at the level of the epigenome which is likely to represent the next level of regulatory scrutiny as more of these cells move into the clinical space.
The second major impact will be in the undertaking of new clinical trials with hPSC derived products and how this can best be done using the emerging genetic engineering and differentiation protocols that have been developed in recent years. The ability to do this in the research lab is well established, but how this can then be done using clinical grade cells, that need to be made reproducibly in a GMP facility, is still unknown. We will address this issue which will impact on any group wishing to adopt similar strategies with their cell products. This work in turn will impact on the future treatment of patients with a whole variety of diseases as well how this can best be delivered in the context of the NHS, and the wider medical market, at an affordable price.
The third major impact will be in terms of the UK regenerative medicine landscape and investment both from funding agencies supporting hPSC based product development by academics as well as biotech companies and big pharma, both nationally and internationally. The investment that has already been made by the UK in this area is impressive (National Stem Cell Bank; Cell and Gene Therapy Catapult etc), but the work we propose will make the UK the optimal location for developing and trialling hPSC derived products. Indeed to date, we have already worked with other groups outside the UK who do not have the national framework and initiatives that we do around hPSC therapies, and thus the investment in the regenerative medicine market is likely to become a major growth area in the UK economy going forward driven in part by the work we are doing through this hub. This will become an increasingly important asset in the UK post Brexit.
Finally our work will impact on many other groups. This will include the greater scientific community and especially those scientists working with hPSCs in the future and in particular how they can optimally culture the cells and the way in which they can be best manipulated genetically for therapeutic use. The patients for whom these therapies are being developed will clearly directly benefit from this work which could have a major impact on how these diseases will routinely be treated in the future. This will lead to a wider discussion at the societal level as to how we see such therapies from an ethical and therapeutic perspective, as well as what we are prepared to pay for them and how all these issues can best be discussed and presented.
In summary the programme of work we propose has the ability to significantly change and impact on the UK such that it will be seen as a global centre for hPSC based regenerative medicine, leading the world in how this can best be done in terms of minimising risk while optimising their speed of development for clinical adoption.
The hub will generate outputs that will define the significant acquired genetic changes seen in hPSCs and their products and what this means. This will then be used by regulatory agencies to develop guidelines that are likely to be adopted worldwide. This will impact on all hPSC based therapies being developed for clinical use anywhere in the world where there exists an overseeing national agency - e,g, MHRA; EMA, FDA and so on. Furthermore we will also start exploring not just the acquired genetic variation that comes with growing and differentiating these cells, but how this can be better controlled, especially during any manufacturing process and storage of the derived cell products. Finally we will start to explore this at the level of the epigenome which is likely to represent the next level of regulatory scrutiny as more of these cells move into the clinical space.
The second major impact will be in the undertaking of new clinical trials with hPSC derived products and how this can best be done using the emerging genetic engineering and differentiation protocols that have been developed in recent years. The ability to do this in the research lab is well established, but how this can then be done using clinical grade cells, that need to be made reproducibly in a GMP facility, is still unknown. We will address this issue which will impact on any group wishing to adopt similar strategies with their cell products. This work in turn will impact on the future treatment of patients with a whole variety of diseases as well how this can best be delivered in the context of the NHS, and the wider medical market, at an affordable price.
The third major impact will be in terms of the UK regenerative medicine landscape and investment both from funding agencies supporting hPSC based product development by academics as well as biotech companies and big pharma, both nationally and internationally. The investment that has already been made by the UK in this area is impressive (National Stem Cell Bank; Cell and Gene Therapy Catapult etc), but the work we propose will make the UK the optimal location for developing and trialling hPSC derived products. Indeed to date, we have already worked with other groups outside the UK who do not have the national framework and initiatives that we do around hPSC therapies, and thus the investment in the regenerative medicine market is likely to become a major growth area in the UK economy going forward driven in part by the work we are doing through this hub. This will become an increasingly important asset in the UK post Brexit.
Finally our work will impact on many other groups. This will include the greater scientific community and especially those scientists working with hPSCs in the future and in particular how they can optimally culture the cells and the way in which they can be best manipulated genetically for therapeutic use. The patients for whom these therapies are being developed will clearly directly benefit from this work which could have a major impact on how these diseases will routinely be treated in the future. This will lead to a wider discussion at the societal level as to how we see such therapies from an ethical and therapeutic perspective, as well as what we are prepared to pay for them and how all these issues can best be discussed and presented.
In summary the programme of work we propose has the ability to significantly change and impact on the UK such that it will be seen as a global centre for hPSC based regenerative medicine, leading the world in how this can best be done in terms of minimising risk while optimising their speed of development for clinical adoption.
Organisations
- University of Cambridge (Lead Research Organisation)
- University College London (Collaboration)
- Cell Therapy Catapult (Collaboration)
- National Health Service (Collaboration)
- NHS National Services Scotland (NSS) (Collaboration)
- The Wellcome Trust Sanger Institute (Collaboration)
- UNIVERSITY OF CAMBRIDGE (Collaboration)
- Genentech, Inc (Collaboration)
- AstraZeneca (United Kingdom) (Collaboration)
- Lund University (Collaboration)
- Advanced Bioprocess Services Ltd (Collaboration)
- National Institutes of Health (NIH) (Collaboration)
- University of Bristol (Collaboration)
- KING'S COLLEGE LONDON (Collaboration)
Publications
Armstrong JPK
(2020)
A blueprint for translational regenerative medicine.
in Science translational medicine
Zhao Y
(2020)
A fluorescent molecular imaging probe with selectivity for soluble tau aggregated protein.
in Chemical science
Roger Alistair Barker
(2019)
A Professional Standard for Informed Consent for Stem Cell Therapies
in Apollo - University of Cambridge Repository
Pantazis CB
(2022)
A reference human induced pluripotent stem cell line for large-scale collaborative studies.
in Cell stem cell
Halliwell J
(2020)
Acquired genetic changes in human pluripotent stem cells: origins and consequences.
in Nature reviews. Molecular cell biology
Rostovskaya M
(2022)
Amniogenesis occurs in two independent waves in primates.
in Cell stem cell
Roger Alistair Barker
(2020)
Animal models of Parkinson's disease are they useful or not?
in Apollo - University of Cambridge Repository
Zhang J
(2019)
Anti-apoptotic Mutations Desensitize Human Pluripotent Stem Cells to Mitotic Stress and Enable Aneuploid Cell Survival.
in Stem cell reports
Price CJ
(2022)
Assessing Cell Competition in Human Pluripotent Stem Cell (hPSC) Cultures.
in Current protocols
S Skidmore
(2020)
Assessing human embryonic stem cell-derived dopaminergic neuron progenitor transplants using non-invasive imaging techniques
in Apollo - University of Cambridge Repository
Robbins M
(2021)
Biofunctionalised bacterial cellulose scaffold supports the patterning and expansion of human embryonic stem cell-derived dopaminergic progenitor cells.
in Stem cell research & therapy
Barker RA
(2021)
Bringing Advanced Therapy Medicinal Products (ATMPs) for Parkinson's Disease to the Clinic: The Investigator's Perspective.
in Journal of Parkinson's disease
Rostovskaya M
(2022)
Capacitation of Human Naïve Pluripotent Stem Cells.
in Methods in molecular biology (Clifton, N.J.)
Skidmore S
(2023)
Challenges in the clinical advancement of cell therapies for Parkinson's disease.
in Nature biomedical engineering
Baker D
(2022)
Characterizing the Genetic Stability of Human Naïve and Primed Pluripotent Stem Cells.
in Methods in molecular biology (Clifton, N.J.)
Thiecke MJ
(2020)
Cohesin-Dependent and -Independent Mechanisms Mediate Chromosomal Contacts between Promoters and Enhancers.
in Cell reports
Cheung M
(2021)
Current trends in flow cytometry automated data analysis software.
in Cytometry. Part A : the journal of the International Society for Analytical Cytology
Wind M
(2021)
Defining the signalling determinants of a posterior ventral spinal cord identity in human neuromesodermal progenitor derivatives.
in Development (Cambridge, England)
Roger Alistair Barker
(2019)
DESIGNING STEM CELL-BASED DOPAMINE CELL REPLACEMENT TRIALS FOR PARKINSON'S DISEASE
in Apollo - University of Cambridge Repository
Shariatzadeh M
(2020)
Distributed automated manufacturing of pluripotent stem cell products.
in The International journal, advanced manufacturing technology
Halliwell JA
(2020)
DNA Fiber Assay for the Analysis of DNA Replication Progression in Human Pluripotent Stem Cells.
in Current protocols in stem cell biology
Stacey A
(2018)
Experimentally integrated dynamic modelling for intuitive optimisation of cell based processes and manufacture
in Biochemical Engineering Journal
Wu J
(2021)
Fitness selection in human pluripotent stem cells and interspecies chimeras: Implications for human development and regenerative medicine.
in Developmental biology
Kusena JW
(2019)
From protocol to product: ventral midbrain dopaminergic neuron differentiation for the treatment of Parkinson's disease.
in Regenerative medicine
Buttery PC
(2020)
Gene and Cell-Based Therapies for Parkinson's Disease: Where Are We?
in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
Stavish D
(2020)
Generation and trapping of a mesoderm biased state of human pluripotency.
in Nature communications
Price CJ
(2021)
Genetically variant human pluripotent stem cells selectively eliminate wild-type counterparts through YAP-mediated cell competition.
in Developmental cell
Vitillo L
(2020)
GMP-grade neural progenitor derivation and differentiation from clinical-grade human embryonic stem cells
in Stem Cell Research & Therapy
Roger Alistair Barker
(2018)
Human Trials of Stem Cell-Derived Dopamine Neurons for Parkinson's Disease: Dawn of a New Era.
in Apollo - University of Cambridge Repository
Tomás-Daza L
(2023)
Low input capture Hi-C (liCHi-C) identifies promoter-enhancer interactions at high-resolution
in Nature Communications
Thompson O
(2020)
Low rates of mutation in clinical grade human pluripotent stem cells under different culture conditions.
in Nature communications
Rostovskaya M
(2022)
Maintenance of Human Naïve Pluripotent Stem Cells.
in Methods in molecular biology (Clifton, N.J.)
Lawrence M
(2022)
Mapping the biogenesis of forward programmed megakaryocytes from induced pluripotent stem cells.
in Science advances
Halliwell JA
(2021)
Nanopore Sequencing Indicates That Tandem Amplification of Chromosome 20q11.21 in Human Pluripotent Stem Cells Is Driven by Break-Induced Replication.
in Stem cells and development
Ruwani S Wijeyekoon
(2020)
Peripheral innate immune and bacterial signals relate to clinical heterogeneity in Parkinson's disease
in Apollo - University of Cambridge Repository
Wijeyekoon RS
(2020)
Peripheral innate immune and bacterial signals relate to clinical heterogeneity in Parkinson's disease.
in Brain, behavior, and immunity
Jerber J
(2021)
Population-scale single-cell RNA-seq profiling across dopaminergic neuron differentiation
in Nature Genetics
Lawrence M
(2021)
Process analysis of pluripotent stem cell differentiation to megakaryocytes to make platelets applying European GMP
in npj Regenerative Medicine
Von Meyenn F
(2022)
Profiling DNA Methylation in Human Naïve Pluripotent Stem Cells.
in Methods in molecular biology (Clifton, N.J.)
Laing O
(2019)
Rapid PCR Assay for Detecting Common Genetic Variants Arising in Human Pluripotent Stem Cell Cultures.
in Current protocols in stem cell biology
Lawrence M
(2021)
Recent Advances in iPSC-Derived Cell Types
Harris K
(2022)
Reduced expression of dopamine D2 receptors on astrocytes in R6/1 HD mice and HD post-mortem tissue
in Neuroscience Letters
| Description | A director of the ISSCR |
| Geographic Reach | North America |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Description | Guidelines for stem cell line selection |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Contribution to new or improved professional practice |
| Description | ISCI |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Participation in a guidance/advisory committee |
| Description | ISSCR Task Force on Standards in Stem Cell Research |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Contribution to new or Improved professional practice |
| Description | Identification of a reference iPSC cell line |
| Geographic Reach | Multiple continents/international |
| Policy Influence Type | Contribution to new or improved professional practice |
| Impact | The perceived reproducibility crisis has hampered the stem cell field, and the introduction of a deeply-characterised reference line will help improve public attitudes (though these have not been objectively measured yet). Furthermore, the use of a common cell line will likely lead to more effective use of research funding (public service delivery) and speed the process of biological discovery by facilitating data, integration, and reproducibility. |
| 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 | Cell Therapy Manufacturing |
| Amount | £192,717 (GBP) |
| Organisation | Advanced Bioprocess Services Ltd |
| Sector | Private |
| Country | United Kingdom |
| Start | 01/2020 |
| End | 12/2025 |
| Description | Modelling Pluripotent Stem Cell Manufacture - Industrial Studentship |
| Amount | £130,211 (GBP) |
| Organisation | Advanced Bioprocess Services Ltd |
| Sector | Private |
| Country | United Kingdom |
| Start | 04/2019 |
| End | 03/2022 |
| Description | Multidisciplinary approach to design engineered tissue to improve cell transplantation therapy for Parkinson's disease. |
| Amount | £6,000 (GBP) |
| Organisation | UK Regenerative Medicine Platform |
| Sector | Academic/University |
| Country | United Kingdom |
| Start | 04/2019 |
| End | 04/2020 |
| Description | NYSCF - Robertson Stem Cell Investigator Award |
| Amount | $1,500,000 (USD) |
| Funding ID | NYSCF-R-156 |
| Organisation | New York Stem Cell Foundation |
| Sector | Charity/Non Profit |
| Country | United States |
| Start | 01/2020 |
| End | 12/2024 |
| Description | STEM-PD trial |
| Amount | £994,206 (GBP) |
| Organisation | Novo Nordisk |
| Sector | Private |
| Country | Denmark |
| Start | 03/2022 |
| End | 03/2027 |
| Description | SilkPlateket |
| Amount | € 2,500,000 (EUR) |
| Funding ID | 101058349 |
| Organisation | European Commission |
| Department | Horizon 2020 |
| Sector | Public |
| Country | European Union (EU) |
| Start | 10/2022 |
| End | 09/2025 |
| Description | Special initiative |
| Amount | £10,000,000 (GBP) |
| Organisation | Wellcome Trust |
| Sector | Charity/Non Profit |
| Country | United Kingdom |
| Start | 05/2019 |
| End | 04/2024 |
| Description | Targeting shared mechanisms in metabolic and neurodegenerative disease (Ben Barres Early Career Acceleration Award) |
| Amount | $2,500,000 (USD) |
| Funding ID | 191942 |
| Organisation | Chan Zuckerberg Initiative |
| Sector | Private |
| Country | United States |
| Start | 01/2019 |
| End | 12/2023 |
| Description | The Pluripotent Stem Cells and Engineered Cell (PSEC) Hub |
| Amount | £4,095,179 (GBP) |
| Funding ID | MR/R015724/1 |
| Organisation | Medical Research Council (MRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 06/2018 |
| End | 05/2023 |
| Description | The immunogenicity of stem cells derived dopamine cells and its implications for first in human clinical trials (Parkinson's)(JBCF) |
| Amount | £173,732 (GBP) |
| Funding ID | M654 |
| Organisation | Rosetrees Trust |
| Sector | Charity/Non Profit |
| Country | United Kingdom |
| Start | 04/2017 |
| End | 08/2021 |
| Description | Transitioning in vitro platelet production to GMP". |
| Amount | £100,000 (GBP) |
| Funding ID | X527722 |
| Organisation | Defence Science & Technology Laboratory (DSTL) |
| Sector | Public |
| Country | United Kingdom |
| Start | 03/2020 |
| End | 03/2021 |
| Description | UKRMP/Immunology: Immunogenecity test platform - in vitro and in vivo |
| Amount | £907,910 (GBP) |
| Organisation | Medical Research Council (MRC) |
| Sector | Public |
| Country | United Kingdom |
| Start | 03/2019 |
| End | 03/2022 |
| Title | KOLF2.1J: A reference human induced pluripotent stem cell line |
| Description | Using deep genotyping and phenotyping, we worked collaboratively to compare 8 candidate iPSC lines and select one to serve as a reference cell line for the field. A pre-print describing this cell line has been published, and we are working to easily share the cell line and accompanying whole genome sequencing data via a website that is scheduled to launch in March 2022. |
| Type Of Material | Cell line |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | The cell line has been shared with over 200 laboratories, and is forming the basis of several large gene editing studies at the NIH, Jackson Laboratories, and the Wellcome Sanger Institute. |
| URL | https://www.jax.org/jax-mice-and-services/ipsc |
| Title | Live Cell Quantification using Image Analysis. |
| Description | We developed pipelines to enable the rapid quantification of cells based on staining by a live-cell non-toxic membrane dye. For applications where there are many conditions in which cells need to be quantified, this approach significantly reduces labor and increases speed, while delivering comparable performance. |
| Type Of Material | Technology assay or reagent |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | This method allows us to perform a head-to-head comparison of dozens of media conditions in a single experiment, yielding valuable insights into which media conditions best promote genomic stability of stem cells. |
| URL | https://www.protocols.io/view/live-cell-quantification-using-image-analysis-bzgep3te |
| Title | RVG:pff model of PD |
| Description | We have recently created this novel model of Parkinson's disease in wild-type rats. This is generated by intravenous administration of pathogenic alpha synuclein ifbrils, hich is reversibly complexed with a peptide carrier, and that a single injection through the tail vein is sufficient to generate neurodegeneration and alpha synuclein pathology in the rat brain in a site-specific and dose-dependent way. We have submitted our manuscript to J Clin Invest and is currently revising our paper for resubmission to this journal. |
| Type Of Material | Model of mechanisms or symptoms - non-mammalian in vivo |
| Year Produced | 2017 |
| Provided To Others? | No |
| Impact | Rather than generating transgenic animals or injecting pathogenic substances through intracerebral administration, our one-off, intravenous delivery of foreign substances will have a profound impact on the welfare of animals (3Rs: replacement, reduction and refinement). In addition to the delivery of pathogenic alpha synuclein fibrils, we have also demonstrated the flexibility of our novel strategies by transvascularly delivering a range of other substances including green fluorscent protein and bioactive enzymes. |
| Title | pipeline for image-based cell quantification |
| Description | Cell counting at each passage is essential for ensuring the consistency of culturing technique, and for comparing growth rates across culture conditions. We developed a pipeline that allows cell numbers to be rapidly and consistently estimated by imaging cultures stained with a non-toxic membrane-bound dye. |
| Type Of Material | Technology assay or reagent |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | n/a |
| URL | https://www.protocols.io/view/live-cell-quantification-using-image-analysis-bzgep3te.html |
| Title | Data relating to pooled single-cell sequencing of dopaminergic neurons (https://doi.org/10.1038/s41588-021-00801-6) |
| Description | Managed access data from scRNA-seq are accessible in the EGA under the study number EGAS00001002885 (dataset EGAD00001006157). Open access scRNA-seq data are available in the European Nucleotide Archive (ENA) under the study ERP121676. Processed single-cell count data and eQTL and colocalization summary statistics are available on Zenodo at https://zenodo.org/record/4333872. The two iPSC single-cell datasets are available on Zenodo (https://zenodo.org/record/3625024) and GEO (GSE118723). |
| Type Of Material | Database/Collection of data |
| Year Produced | 2021 |
| Provided To Others? | Yes |
| Impact | The methods we helped develop have been expanded upon by The Foundational Data Initiative for Parkinson Disease, as recently described by Bressan and colleagues in Cell Genomics (https://doi.org/10.1016/j.xgen.2023.100261) |
| URL | https://zenodo.org/record/4333872 |
| Title | Single-cell and whole genome sequencing data relating to the KOLF2.1J manuscript (10.1101/2021.12.15.472643) |
| Description | Whole genome sequencing data of 8 human induced pluripotent stem cell lines, generated in the pluripotent state, and single-cell RNA sequencing data generated in the pluripotent state and over 4 distinct differentiation protocols. |
| Type Of Material | Database/Collection of data |
| Year Produced | 2022 |
| Provided To Others? | Yes |
| Impact | The cell line that was selected based on the analysis performed in this study (supporting data in the database above) has been requested by and distributed to over 200 distinct groups already, and promises to improve reproducibility and data sharing in the stem cell field. |
| URL | https://fair.addi.ad-datainitiative.org/#/data/datasets/a_reference_induced_pluripotent_stem_cell_li... |
| Title | Whole-genome sequences of 143 human embryonic stem cell lines that enable rational line selection based on genetic variation |
| Description | Despite their widespread use in research, there has not yet been a systematic genomic analysis of human embryonic stem cell (hESC) lines at a single-nucleotide resolution. We therefore performed whole-genome sequencing (WGS) of 143 hESC lines and annotated their single-nucleotide and structural genetic variants. We found that while a substantial fraction of hESC lines contained large deleterious structural variants, finer-scale structural and single-nucleotide variants (SNVs) that are ascertainable only through WGS analyses were present in hESC genomes and human blood-derived genomes at similar frequencies. Moreover, WGS allowed us to identify SNVs associated with cancer and other diseases that could alter cellular phenotypes and compromise the safety of hESC-derived cellular products transplanted into humans. As a resource to enable reproducible hESC research and safer translation, we provide a user-friendly WGS data portal and a data-driven scheme for cell line maintenance and selection. The dataset on DUOS is: DUOS-000121 |
| Type Of Material | Database/Collection of data |
| Year Produced | 2022 |
| Provided To Others? | Yes |
| Impact | These data have enabled researchers to avoid cell lines that carry genetic abnormalities that would make certain lines less suitable for use, and select those that are best suited based on their genetic characteristics. |
| URL | https://duos.broadinstitute.org/ |
| 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 | Astra Zeneca industrial collaboration for universal cell therapies |
| Organisation | AstraZeneca |
| Country | United Kingdom |
| Sector | Private |
| PI Contribution | This is a network based partnership involving several PIs at the Stem Cell Institute, Cambridge, PSEC and Astra Zeneca R&D (based in Gothenburg) with the avowed aim to look at gene editing techniques to product iPSC cell lines from which universal (immune silent) cell therapies can be produced |
| Collaborator Contribution | Exchange of ideas and strategies |
| Impact | Only just started |
| Start Year | 2020 |
| Description | Austin Smith |
| Organisation | University of Cambridge |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Epigenomics and multi-omics sequencing |
| Collaborator Contribution | Human and mouse pluripotent stem cells |
| Impact | Several collaborative publications. |
| Start Year | 2013 |
| Description | Collaboration with Cell Therapy Catapult |
| Organisation | Cell Therapy Catapult |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | Gap analysis for large scale production of platelets in vitro_we are looking to implement them |
| Collaborator Contribution | Gap analysis for large scale production of platelets in vitro_we are looking to implement them |
| Impact | DSTL grant application |
| Start Year | 2020 |
| Description | Collaboration with Smart Materials Group (UCL) |
| Organisation | University College London |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | This is a collaboration looking at the effect if smart materials on the survival and immunogenecity of DA neurons. Our role in the collaboration is to provide the cells, and to provide intellectual input into the study design. This work was initially part of a small grant (mathematical modelling grant from UKRMP), but has now extended beyond the life of that grant. |
| Collaborator Contribution | Our collaborators (James Phillips and team), are experts in biomaterials. They are designing, carrying out an analysing the effects of smart materials on DA cell survival. |
| Impact | Poster presentation |
| Start Year | 2019 |
| Description | Computational biology - Marioni |
| Organisation | Genentech, Inc |
| Country | United States |
| Sector | Private |
| PI Contribution | We provide in vitro and mouse model systems and data |
| Collaborator Contribution | Our collaborators provide computational expertise and analysis of our data |
| Impact | We have published one manuscript and are working on two others. Disciplines: stem cell biology, neuroscience, animal physiology, computational biology |
| Start Year | 2020 |
| Description | Edited iPSC lines for research on SARS-CoV-2 effect on megakaryocytes and platelets |
| Organisation | University of Bristol |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | We have generated cell lines knock-out for putative SARS-CoV-2 receptors using the gene editing strategy developed with PSEC |
| Collaborator Contribution | The two Bristol groups (Poole and Davidson) provide experimental support with MKs (Poole) and SARS-CoV-2 co-cultures. |
| Impact | Still ongoing |
| Start Year | 2020 |
| Description | HipSci cell lines and computational methods for cell fraction estimation |
| Organisation | The Wellcome Trust Sanger Institute |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | We provide experimental model systems and analysis to identify media conditions to minimise selective pressures of stem cells in culture |
| Collaborator Contribution | We have obtained genetically distinct induced pluripotent stem cells lines, as well as code to estimate cell fractions from pools of genetically distinct cells. |
| Impact | we are currently analysing data, but have no publications yet. Collaboration is multi-disciplianary: cell biology, computational biology, genetics |
| Start Year | 2021 |
| Description | Jenny Nichols mouse and human embryology |
| Organisation | University of Cambridge |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Single cell multi-omics, mutants of epigenetic regulators. |
| Collaborator Contribution | Mouse and human embryos |
| Impact | Several collaborative publications and two joint grants. Multi-disciplinary between epigenetics, multi-omics, embryology, stem cell research. |
| Start Year | 2014 |
| Description | Lund Collaboration |
| Organisation | Lund University |
| Country | Sweden |
| Sector | Academic/University |
| PI Contribution | Supply of fetal and post mortem brain tissue to collaborators in Lund. Collaboration on the stem cell for parkinson's disease project. |
| Collaborator Contribution | Collaboration on the stem cell for parkinson's disease project. |
| Impact | Publications |
| Start Year | 2010 |
| Description | Mathematical Modelling Collaboration with UCL |
| Organisation | University College London |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | This is a brand new collaboration with James Philips and Rebecca Shipley of UCL. We have two collaborative projects which aim to use mathematical modelling approaches to (i) design engineered tissues to improve cell transplantation therapy for Parkinson's disease and (ii) develop novel controlled release biomaterials (eg controlled release immunomodulating agents) for CNS cell therapies. The primary role of the Cambridge research team will be to provide the cellular material, and to perform in vitro immunogenecity assays for the controlled biomaterial project. The collaboration has not yet bought in funds - but we have applied for two UKRMP pump priming grants. |
| Collaborator Contribution | Our UCL partners with provide the mathematical modelling know-how (Rebecca Shipley), and the smart materials expertise. |
| Impact | Nil to date - the collaboration was only formed 1 month ago. |
| Start Year | 2019 |
| Description | NHS National Clinical Entrepreneur programme |
| Organisation | National Health Service |
| Country | United Kingdom |
| Sector | Hospitals |
| PI Contribution | Taking part in this programme has allowed useful networking and a learning of business related matters in term of exploiting academic outcomes |
| Collaborator Contribution | Network and teachings |
| Impact | Network and knowledge of business translation of academic discoveries |
| Start Year | 2021 |
| Description | Open Targets - NeuroID CRISPR screens |
| Organisation | The Wellcome Trust Sanger Institute |
| Country | United Kingdom |
| Sector | Charity/Non Profit |
| PI Contribution | We are assisting with the generation of cellular assays, neuronal differentiation, and biological interpretation of resulting data. |
| Collaborator Contribution | Our collaborators are proving expertise in CRISPR gene editing and bioinformatics. |
| Impact | one manuscript is in the early stages of preparation. We developed methods of making induced astrocyes from hPSCs, and in characterising these found that LRP1 is a receptor for monomeric tau uptake in astrocytes, much as it is in neurons. |
| Start Year | 2020 |
| Description | Production of universal platelets from iPSC at GMP |
| Organisation | NHS National Services Scotland (NSS) |
| Department | Scottish National Blood Transfusion Service |
| Country | United Kingdom |
| Sector | Public |
| PI Contribution | Developing an iPSC universal cell line for the production of platelets at GMP |
| Collaborator Contribution | Process development and gene editing at GMP |
| Impact | Process development and gene editing at GMP |
| Start Year | 2020 |
| Description | UKRMP Collaboration |
| Organisation | King's College London |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | This award enabled us to form a collaboration with Kings College London and enabled us to apply for and be awarded a UKRMP grant (total award 900K, our portion of the award £328,032). Our research team helped develop the idea behind the grant application, and wrote the application. Within this award/new collaboration we will be utilising our humanised mouse model to test the immunogenecity of ES-DA cells. |
| Collaborator Contribution | Our collaborators also helped develop the idea behind the grant application, and helped to write the application. Within the award/new collaboration they will be testing the immunogenecity of iPSC-hepatocytes in vitro and in vivo (using a humanised mouse model). |
| Impact | Nil yet as this grant/collaboration has only been active a few months. |
| Start Year | 2019 |
| Description | iNDI collaboration to select hiPSC lines |
| Organisation | National Institutes of Health (NIH) |
| Department | National Institute of Neurological Disorders and Stroke (NINDS) |
| Country | United States |
| Sector | Public |
| PI Contribution | We are assisting with the analysis of whole genome sequencing data, and are differentiating a panel of human induced pluripotent stem cell (hiPSC) lines to various neuronal lineages in order to select a cell line for future use by a consortium. |
| Collaborator Contribution | Our collaborators have selected and obtained a panel of cell lines, have performed whole genome sequencing, and are also differentiating these cell lines down different lineages for comparision. |
| Impact | We have published one manuscript that has already had a substantial impact on the stem cell field. |
| Start Year | 2019 |
| Title | Collaboration agreement |
| Description | This collaboration agreement covers work to be carried out under the Wellcome Human Developmental Biology Initiative |
| IP Reference | |
| Protection | Protection not required |
| Year Protection Granted | 2019 |
| Licensed | No |
| Impact | None yet |
| Title | Methylome of human blastocysts |
| Description | Collaboration agreement with Karolinska Institute |
| IP Reference | |
| Protection | Protection not required |
| Year Protection Granted | 2017 |
| Licensed | No |
| Impact | Several datasets have been obtained |
| Title | UKRMP2 pluripotent stem cell and engineered cell hub |
| Description | Collaboration agreement with PSEC on human pluripotent stem cells |
| IP Reference | |
| Protection | Protection not required |
| Year Protection Granted | 2018 |
| Licensed | No |
| Impact | Several datasets have been obtained |
| Title | RESTORE |
| Description | Primary haematopoietic progenitors-derived red cells injected into volunteer to show improvement of recovery and survival compared to donor-derived red cells |
| Type | Therapeutic Intervention - Cellular and gene therapies |
| Current Stage Of Development | Initial development |
| Year Development Stage Completed | 2020 |
| Development Status | Under active development/distribution |
| Clinical Trial? | Yes |
| Impact | Inform future large scale production of in vitro derived blood cells |
| URL | https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-002178-38 |
| Title | Code relating to the KOLF2.1J manuscript |
| Description | Code describing the analysis of whole genome and single-cell datasets |
| Type Of Technology | Software |
| Year Produced | 2022 |
| Open Source License? | Yes |
| Impact | n/a |
| Title | cell line fraction estimation from cell pools |
| Description | Pooling genetically distinct cell lines together and culturing them under diverse conditions is a powerful way to understand how the composition of each cell line changes over time. The method takes advantage of the fact that if the whole genome sequences of each cell line in the pool is known, there are many single nucleotide variants (SNVs) that are informative about cell identity. Even if the pool of cells is sequenced at low depth of sequencing coverage (e.g. 1x), enough SNVs can be called to confidently assign cell identity. |
| Type Of Technology | Software |
| Year Produced | 2021 |
| Open Source License? | Yes |
| Impact | n/a |
| Title | code for stem cell genomic data web portal |
| Description | We created a user-friendly online data portal (https://hscgp.broadinstitute.org/hscgp) that enables users with more limited computational expertise to readily search for sequence variants of interest among sequenced hESC lines. For example, a search for TP53 reveals all variants in the gene that were detected in the sequenced cell lines, the names of those cell lines, as well as bioinformatic predictions about the likely consequences of these variants. Search results can be graphically visualized and exported for further analysis in a variety of formats. Specific cell lines can also be interrogated for the presence of variants of interest, and raw sequencing alignments can be visualized via the integrative genomics viewer IGV. |
| Type Of Technology | Software |
| Year Produced | 2022 |
| Open Source License? | Yes |
| Impact | Dozens of interested groups have visited the GitHub repository, and the code is being implemented as part of the JAX website to display WGS data from the KOLF2.1J cell line. |
| URL | https://github.com/broadinstitute/hscgp |
| Title | stem cell genome data portal |
| Description | allows users to generate data portals to easily share and browse whole genome sequencing data |
| Type Of Technology | Webtool/Application |
| Year Produced | 2021 |
| Impact | portal is being adopted by a major institution distributing stem cell lines (Jackson Laboratories and NIH) |
| URL | https://hscgp.broadinstitute.org/hscgp |
| Company Name | SAFI BIOSOLUTIONS UK LIMITED |
| Description | As the cell therapy commercialization partner of a 5-year Department of Defense program to manufacture on-demand blood products, Safi Biosolutions and its collaborators are working to 'crack the code' of Cell Therapy 2.0 challenges of manufacturing at appropriate scale, high consistency of product, and economically viable cost of goods by integrating world-leading expertise in cord blood stem cell expansion, bioprocessing optimization, manufacturing scale-up and cryostorage. Lead development programs for manufactured, on-demand cell therapy products include red blood cells for trauma, tailored red blood cells for specific transfusions (e.g. sickle cell disease) and a neutrophil progenitor cell therapy for the treatment of chemotherapy-induced neutropenia. |
| Year Established | 2021 |
| Impact | Established an economic manufacturing platform for therapeutic blood products |
| Website | https://safi.bio/ |
| Company Name | ADVANCED BIOPROCESS SERVICES LIMITED |
| Description | Bioprocess Development Service Provider for Cell and Gene Therapies |
| Year Established | 2016 |
| Impact | Worked with a range of early stage cell and gene therapy companies to deliver novel, economic, and robust manufacturing processes for pre-clinical models; provided the development services that have directly supported in excess of $20M of private raise. |
| Company Name | CELLADVICE LTD |
| Description | Consultancy company specialising in blood transfusion and cell therapies |
| Year Established | 2019 |
| Impact | Currently providing expert advice to 4 different companies |
| Company Name | REGENERATIVE CELL THERAPY CONSULTING LIMITED |
| Description | Regenerative Cell Therapy Consulting (Regen CTC) is a network of UK academic experts with real world experience of translating human pluripotent stem cell (hPSC)-derived cell therapies to the clinic. Established under the UK Regenerative Medicine Platform in 2018, Regen CTC's members include fundamental scientists, manufacturing engineers, clinicians and expert translational scientists with experience in developing quality management systems, clean room operations and process development of research protocols. Our mission is to use our knowledge and experience to accelerate your cell and gene therapy developmental pipeline, helping you to avoid common pitfalls. In so doing, our translational consultants hope to be able to assist advanced therapy developers to deliver their complex therapies to the clinic so that the patients who need them can begin to see their benefits. |
| Year Established | 2022 |
| Impact | The company only established in September 2022, and now has 2 part time scientific consultants |
| Website | https://www.regenctc.com/ |
| Company Name | XAP THERAPEUTICS LIMITED |
| Description | Start up looking to make new therapeutics based on platelets produced in vitro |
| Year Established | 2017 |
| Impact | None as yet |
| Description | BSGCT AGM - Plenary Lecture |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Professional Practitioners |
| Results and Impact | Invited speaker to discuss development of cell transplantation for Parkinson's disease |
| Year(s) Of Engagement Activity | 2019 |
| Description | British Neurosurgery Trainee Meeting, Robinson College, Cambridge |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Professional Practitioners |
| Results and Impact | A talk entitled: Disorders of the motor system and the current and future treatment of Parkinson's disease |
| Year(s) Of Engagement Activity | 2020 |
| Description | Cambridge Science Festival |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Public/other audiences |
| Results and Impact | A stall from the IMS at the Cambridge Science Festival educated the general public about issues in obesity and metabolic science |
| Year(s) Of Engagement Activity | 2020,2023 |
| Description | Cambridge Science Festival |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Public/other audiences |
| Results and Impact | I volunteered to serve as a representative of the WT-MRC Institute of Metabolic Science at the Cambridge Science Festival, where hundreds to thousands of members of the public learned about issues in metabolism relating to their daily lives. |
| Year(s) Of Engagement Activity | 2016,2017,2019,2023 |
| URL | http://www.sciencefestival.cam.ac.uk/events |
| Description | Cell Symposia: Translation of Stem Cells to the Clinic 2018 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Invited talk on stem cell therapies for PD |
| Year(s) Of Engagement Activity | 2018 |
| Description | Charity Concert All Saints 2018 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Public/other audiences |
| Results and Impact | Charity concert at which I and professional musicians performed as well as children of the local drama school. The concept was supported by the Cambridge Stem Cell Institute and the benefits went to Naitbabies.org, a patient organisation supporting patients affected by neonatal alloimmune thrombocytopenia |
| Year(s) Of Engagement Activity | 2018 |
| Description | Charity concert 2019 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Public/other audiences |
| Results and Impact | Charity concert in Newmarket for the benefit of the East Anglia Air Ambulance_PhD students and post-doc presented some of their stem cell work in the context of regenerative medicine to the general public who attended the concert |
| Year(s) Of Engagement Activity | 2019 |
| Description | Charity concert 2022 |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Public/other audiences |
| Results and Impact | Charity concert for the benefit of MON voice, a charity supporting patients with Myeloproliferative disorders. |
| Year(s) Of Engagement Activity | 2022 |
| Description | Clinical Neuroscience - Departmental Away Day |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Professional Practitioners |
| Results and Impact | A debate entitled - The great debate - stem cells for brain diseases: the only hope of a cure |
| Year(s) Of Engagement Activity | 2019 |
| Description | DSTL: better blood, better outcome workshop |
| 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 | DSTL set up the conference to link academics, medical practitioners and industry with an interest in providing blood products to the front line |
| Year(s) Of Engagement Activity | 2022 |
| Description | Departmental seminar, Edinburgh |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Professional Practitioners |
| Results and Impact | A talk presented at Edinburgh departmental neurology meeting: How disease stratification impacts on the experimental therapeutic landscape in PD |
| Year(s) Of Engagement Activity | 2018 |
| Description | G Force PD |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Other audiences |
| Results and Impact | G FORCE PD is an international collaboration between members of various academic institutes to discuss the process of taking stem cell therapies to clinic in patients with Parkinon's disease |
| Year(s) Of Engagement Activity | 2018 |
| Description | G Force PD 2019 |
| 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 | A talk given at the annual G Force PD meeting and a follow up public lecture on Parkinson's disease and stem cell therapies. |
| Year(s) Of Engagement Activity | 2019 |
| Description | Generation of video to highlight the role of minorities in science |
| 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 | Public/other audiences |
| Results and Impact | A graduate student in my group generated a video with the assistance of the University of Cambridge to raise awareness of the impact of minority researchers. This video has been posted to YouTube and has been viewed more than 1800 times. |
| Year(s) Of Engagement Activity | 2018 |
| URL | https://www.youtube.com/watch?v=f90C-psTPkk&t=21s |
| Description | Genome editing workshop |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Public/other audiences |
| Results and Impact | Workshop about Crispr genome editing at the Cambridge Science Festival |
| Year(s) Of Engagement Activity | 2019 |
| Description | How close are we to delivering a stem cell based therapy in Parkinson's Disease |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Recorded plenary lecture as part of Korean Stem Cell Society AGM |
| Year(s) Of Engagement Activity | 2021 |
| Description | ISSCR - LA - Inductry session |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Talk entitled, developing an investigational IND for Parkinson's disease |
| Year(s) Of Engagement Activity | 2019 |
| Description | ISSCR, Melbourne |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Industry/Business |
| Results and Impact | Invited speaker at the International society for stem cell research, held in Melbourne, Australia. Roger gave 3 talks during the whole event. |
| Year(s) Of Engagement Activity | 2018 |
| Description | Interview on BBC world service |
| 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 | I was interviewed on BBC World Service about my role in helping write an open letter to Mark Zuckerberg about the role of Facebook (Meta) in regulating speech on the internet. The broadcast reaches over a million listeners per week (https://media.info/radio/stations/bbc-world-service/listening-figures). |
| Year(s) Of Engagement Activity | 2020 |
| Description | Interviews with international and national press re RESTORE trial (in vitro derived red cells) |
| 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 | Radio, written press and television interview re the RESTORE first-in-human trial with in vitro derived red cells |
| Year(s) Of Engagement Activity | 2022 |
| Description | Invited Speaker - ISSCR Meeting on Ethics |
| 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 | Discussion around the ethical challenges of taking stem cells to clinical therapy |
| Year(s) Of Engagement Activity | 2019 |
| Description | Invited Speaker - Miltenyi GMBH |
| 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 | A talk given entitled, From clinic to bench to clinic - the basis of the heterogeneity of PD and its therapeutic implications |
| Year(s) Of Engagement Activity | 2019 |
| Description | Invited Speaker - NECTAR 2019 |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | A talk on Transeuro - Lessons Learned |
| Year(s) Of Engagement Activity | 2019 |
| Description | Invited Speaker- Novo Nordisk, Copenhagen |
| 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 | A discussion around stem cell therapies for Parkinson's disease |
| Year(s) Of Engagement Activity | 2019 |
| Description | Lessons learnt from TransEuro for taking a cell based therapy to clinic |
| 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 | Professional Practitioners |
| Results and Impact | EU Networking meeting to discuss the lessons learnt from TransEuro for taking a cell based therapy to clinic, web-based seminar. |
| Year(s) Of Engagement Activity | 2019,2020 |
| Description | Lichfield - Science and Engineering Society |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Public/other audiences |
| Results and Impact | Can we repair the chronically degenerating brain? |
| Year(s) Of Engagement Activity | 2019 |
| Description | Live Webinar - DOPAMINE CELL THERAPIES FOR PARKINSON'S DISEASE- WHY BOTHER? |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Annual general meeting of NCER consortium, Luxembourg |
| Year(s) Of Engagement Activity | 2021 |
| Description | Live Webinar - THE NON DOPAMINERGIC ASPECTS OF PARKINSON'S DISEASE- HOW CAN STEM CELL THERAPIES SPEAK TO THEM? |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Third sector organisations |
| Results and Impact | Live Webinar as part of the NSC-Recontruct workshop in Rome |
| Year(s) Of Engagement Activity | 2021 |
| Description | Live talk - TransEuro- lessons learnt to date |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Live (in person) talk at the NECTAR meeting - 16th International Symposium on Neural Transplantation and Repair (INTR) and 31st ECTAR meeting, Edinburgh. |
| Year(s) Of Engagement Activity | 2021 |
| Description | Live webinar - CAN WE REPAIR THE AGING DEGENERATING HUMAN BRAIN? |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Public/other audiences |
| Results and Impact | Live webinar for CUH BRC PPIE group |
| Year(s) Of Engagement Activity | 2021 |
| Description | Masters in Therapeutics Science students |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Undergraduate students |
| Results and Impact | A talk on, Can we repair the chronically degenerating brain? |
| Year(s) Of Engagement Activity | 2020 |
| Description | Oxford Stem Cell Institute annual meeting |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Postgraduate students |
| Results and Impact | An invited talk on Stem Cells and Parkinson's disease - where are we therapeutically to students and scientists |
| Year(s) Of Engagement Activity | 2018 |
| Description | Parkinson's Disease Open Day |
| Form Of Engagement Activity | Participation in an open day or visit at my research institution |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Patients, carers and/or patient groups |
| Results and Impact | Annual Parkinson's disease open day held at the John Van Geest Centre for Brain Repair, patients and carers are invited to attend and the ongoing research of the Barker Lab is presented. |
| Year(s) Of Engagement Activity | 2019 |
| Description | Participant in Roundtable discussion hosted by Nature magazine |
| 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 | A round-table discussion in front of an audience of over 100 professors, postdocs, editors, and policy makers debated the issues surrounding the safety and genetic stability of human stem cell-based research. The results will be published and disseminated via the web to reach a much larger audience. |
| Year(s) Of Engagement Activity | 2018 |
| Description | Participant in public meeting hosted by ThermoFisher |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | A public discussion of issues relating to stem cell genomic stability helped to inform issues relevant to stem cell basic and translation research. |
| Year(s) Of Engagement Activity | 2018 |
| Description | Patient Day |
| Form Of Engagement Activity | Participation in an open day or visit at my research institution |
| Part Of Official Scheme? | No |
| Geographic Reach | Regional |
| Primary Audience | Patients, carers and/or patient groups |
| Results and Impact | Since drugs used to treat obesity have also shown neuroprotective effects, we worked with Prof. Roger Barker and Prof. Williams-Gray to organize a patient day for individuals affected by Parkinson's disease and their families to inform them about the latest research and ongoing trials. |
| Year(s) Of Engagement Activity | 2022 |
| Description | Press release |
| 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 | We generated press releases and social media interest in our work to reach a local audience at the university of cambridge, and wider international audience of interested professionals and the lay public. To complement these efforts, I also write commentaries on informational websites that are read by both professional and lay audiences (e.g. Alzforum) |
| Year(s) Of Engagement Activity | 2022,2023 |
| Description | Public Engagement Champion Cambridge Stem Cell Institute |
| Form Of Engagement Activity | A formal working group, expert panel or dialogue |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Public/other audiences |
| Results and Impact | I have been nominated as the Public Engagement Champion of the CSCI_in short I am the academic PI supporting the Public Engagement team including chairing regular meetings and shaping strategy and implementation thereof |
| Year(s) Of Engagement Activity | 2019,2020 |
| Description | Stem Cell Symposium |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Other audiences |
| Results and Impact | This debate was for scientists |
| Year(s) Of Engagement Activity | 2018 |
| Description | TAKING A DOPAMINE STEM CELL THERAPY TO PATIENTS WITH PARKINSON'S DISEASE- LESSONS LEARNT (SO FAR!) |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | IT WAS A TALK DESCRIBING THE CHALLENGES OF TAKING A STEM CELL PRODUCT TO A FIRST IN HUMAN TRIAL. |
| Year(s) Of Engagement Activity | 2020 |
| Description | THE CHALLENGES OF TAKING A DOPAMINE CELL BASED THERAPY TO PATIENTS WITH PARKINSON'S DISEASE- IS IT WORTH IT? |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Recorded as part of a Nova programme of talks for MDS meeting delivered to Neurologists |
| Year(s) Of Engagement Activity | 2021 |
| Description | THOR invited speaker |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Professional Practitioners |
| Results and Impact | Invited speaker at the THOR conference (acute trauma and haemorrhage) |
| Year(s) Of Engagement Activity | 2022 |
| Description | Webinar & Q&A Session - WHAT HAS CHANGED IN PARKINSON'S SINCE MY LAST TALK IN 2012? A PERSONAL PERSPECTIVE |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | National |
| Primary Audience | Patients, carers and/or patient groups |
| Results and Impact | Live webinar and Q&A session for patients, families and clinicians for Edinburgh PUK branch annual lecture |
| Year(s) Of Engagement Activity | 2021 |
| Description | Webinar - Cell therapies for Parkinson's Disease |
| Form Of Engagement Activity | A talk or presentation |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Undergraduate students |
| Results and Impact | Webinar to engage and inform university students |
| Year(s) Of Engagement Activity | 2021 |
| Description | Webinar - HOW CAN ONE BEST TEST A STEM CELL BASED DOPAMINE THERAPY FOR PATIENTS WITH PARKINSON'S DISEASE? |
| 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 | Webinar for the German Stem Cell network- 10th International meeting |
| Year(s) Of Engagement Activity | 2021 |
| Description | Webinar - THE CHALLENGES OF TAKING A DOPAMINE CELL BASED THERAPY TO PATIENTS WITH PARKINSON'S DISEASE- IS IT WORTH IT? |
| 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 | Invited lecturer to deliver the annual Druker lecture at Harvard Medical School |
| Year(s) Of Engagement Activity | 2021 |
| Description | Weert College ethics workshop |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | International |
| Primary Audience | Schools |
| Results and Impact | This was a scientific ethics workshop for a school in the Netherlands |
| Year(s) Of Engagement Activity | 2019 |
| Description | development of a Neurodegeneration Community Building initiative |
| 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 | We have developed a community building initiative to bring together groups in Cambridge working in neurodegeneration. We organised nine virtual symposia with over 35 speakers. The program included talks as well as breakout discussion groups about major unresolved topics. Speakers were drawn from academic and industrial institutions from around the world, and the events attracted hundreds of participants. We reserved the lion's share of presenting opportunities for students, postdocs, and newly independent group leaders. |
| Year(s) Of Engagement Activity | 2020,2021,2022 |
| Description | hosted student in the laboratory |
| Form Of Engagement Activity | Participation in an open day or visit at my research institution |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Undergraduate students |
| Results and Impact | We hosted an undergraduate student in the laboratory for 2 days. She had an interest in biology and sought to gain insight into a career in science. Since visiting the lab, she has applied to MPhil and PhD programmes. Later, we hosted an undergraduate for 2 months in the summer. He reported that he would be much more likely to apply for graduate school as a result of the time in the lab. |
| Year(s) Of Engagement Activity | 2019,2022 |
| URL | https://www.exppg.lifesci.cam.ac.uk/ |