Production of HLA-null universal platelets from GMP banked human pluripotent stem cell lines
Lead Research Organisation:
University of Cambridge
Department Name: Haematology
Abstract
Each year 280,000 bags of platelets are given to patients in the UK. Platelets promote blood clotting after injury. Cancer therapy or heavy bleeding due to trauma or extensive surgery can reduce the number of platelets in the blood. Patients with a low number of platelets have an increased risk of bleeding (including in the bowels and brain). To prevent bleeding, platelets are given that are derived from blood donations.
Platelets cannot be stored in the fridge. Instead they are kept at room temperature, which means they can only be kept for 7 days. This makes stock management a challenge. Also, as with all donor-derived products (blood, organs etc) there is a small risk of transmission of infections from donors to patients. To minimize this risk, blood donations are screened for infections such as HIV or hepatitis. Finally, some patients develop an immune reaction against the "self" signature of platelets that would result in them destroying platelets from "random" donors. These patients are often women who have had several children or patients who repeatedly receive platelets (such as those undergoing chemotherapy). For these patients we use in England 15,000 bags of platelets per year that are specially matched. These bags are 3 times more expensive than unmatched bags. . The production of matched bags requires donors with the same "self" signature on the surface of their platelets. The "self" signature is called HLA Class I. In only 1/3 of cases a perfect match can be found and for 2/3 the platelets are at best a "near match". When "not perfectly matched" platelets are given they last shorter than "perfectly matched" ones which means they need to be given more often. Also, with not perfectly matched platelets it is more likely that patients will bleed and need to stay in hospital longer.
The option of producing platelets from banks of stem cells in the laboratory would allow for guaranteed supply, remove the risk of infection that comes with donor-derived platelets and would allow for better matching of platelets to patients.
Platelets are produced from megakaryocytes (MKs) in the bone marrow. We have in the last 4 years had a breakthrough in developing an efficient method to produce MKs from stem cells in the laboratory. In addition, we have made a devise that allows us to harvest platelets from those manufactured MKs. Our technology can be adapted to make platelets in such a way that they are safe to give to patients. Finally, we are able to delete the "self" signature from the surface of the platelets by modifying the DNA of the stem cells. These "anonymous" platelets would not be recognized by the immune system and would represent a "perfect" match for patients who cannot receive platelets from "random" donors. Crucially, they would not trigger an immune reaction in anyone.
We plan to use our technology to produce platelets for a first trial in humans in the next 5 years. We will use stem cells that can be used to produce products to give to humans. We call these cells "clinical-grade". We will identify which of these "clinical grade" stem cells are very efficient at producing platelets. Subsequently, we will remove the "self" signature from these clinical-grade stem cells to make them a "universal" match. We have already done this for stem cells that are not "clinical-grade" and have shown that taking away the "self" signature does not impact on the quality of the platelets the stem cells make. We now need to confirm this in "clinical-grade" stem cells before we can start producing "clinical-grade" "universal" platelets for a human trial.
Platelets cannot be stored in the fridge. Instead they are kept at room temperature, which means they can only be kept for 7 days. This makes stock management a challenge. Also, as with all donor-derived products (blood, organs etc) there is a small risk of transmission of infections from donors to patients. To minimize this risk, blood donations are screened for infections such as HIV or hepatitis. Finally, some patients develop an immune reaction against the "self" signature of platelets that would result in them destroying platelets from "random" donors. These patients are often women who have had several children or patients who repeatedly receive platelets (such as those undergoing chemotherapy). For these patients we use in England 15,000 bags of platelets per year that are specially matched. These bags are 3 times more expensive than unmatched bags. . The production of matched bags requires donors with the same "self" signature on the surface of their platelets. The "self" signature is called HLA Class I. In only 1/3 of cases a perfect match can be found and for 2/3 the platelets are at best a "near match". When "not perfectly matched" platelets are given they last shorter than "perfectly matched" ones which means they need to be given more often. Also, with not perfectly matched platelets it is more likely that patients will bleed and need to stay in hospital longer.
The option of producing platelets from banks of stem cells in the laboratory would allow for guaranteed supply, remove the risk of infection that comes with donor-derived platelets and would allow for better matching of platelets to patients.
Platelets are produced from megakaryocytes (MKs) in the bone marrow. We have in the last 4 years had a breakthrough in developing an efficient method to produce MKs from stem cells in the laboratory. In addition, we have made a devise that allows us to harvest platelets from those manufactured MKs. Our technology can be adapted to make platelets in such a way that they are safe to give to patients. Finally, we are able to delete the "self" signature from the surface of the platelets by modifying the DNA of the stem cells. These "anonymous" platelets would not be recognized by the immune system and would represent a "perfect" match for patients who cannot receive platelets from "random" donors. Crucially, they would not trigger an immune reaction in anyone.
We plan to use our technology to produce platelets for a first trial in humans in the next 5 years. We will use stem cells that can be used to produce products to give to humans. We call these cells "clinical-grade". We will identify which of these "clinical grade" stem cells are very efficient at producing platelets. Subsequently, we will remove the "self" signature from these clinical-grade stem cells to make them a "universal" match. We have already done this for stem cells that are not "clinical-grade" and have shown that taking away the "self" signature does not impact on the quality of the platelets the stem cells make. We now need to confirm this in "clinical-grade" stem cells before we can start producing "clinical-grade" "universal" platelets for a human trial.
Technical Summary
WP1. Generation of HLA Class I null lines
Three hPSC lines with the best MK output will be transfected with each of the paired guide RNAs and Cas9 nickase cloned into 2 vectors containing either GFP or dTomato. Double-positive single cells will be sorted and subsequent colonies will be screened for HLA Class I expression by flow cytometry. Pluripotency will be confirmed in standard assays. This method has been validated on research hPSC lines in our lab.
WP2. Determine off-target rate.
The whole genome sequence of each HLA null line will be compared to the parent line to ascertain the off-target rate. This will be supported by the HipSCi initiative.
WP3. FoP MK production and phenotyping.
Using validated batches of lentiviral vectors from the CiC project we will FoP parent and HLA null lines to compare the dynamics of MK differentiation and proliferation, the potential to freeze-thaw the MKs and their ability to form proplatelets. RNA sequencing will be used to confirm whole genome expression levels are the same in null MKs compared to HLA expressing ones.
WP4. Platelet output and functionality.
We will seed mature FoP MKs from the parent and null lines in our bioreactor to compare the efficiency of platelet production. Platelet functionality will be assessed in a range of assays including adhesion and spreading, aggregation, thrombus formation in collagen-coated flow chambers and ultrastructure analysis by electron microscopy.
WP5. In vivo platelet survival studies.
NSG mice will be injected with 40-240x10^6 (equivalent to a therapeutic dose of platelets [3x10^11] in adult patients) in vitro-produced "null" or "wild-type" platelets or donor-derived platelets. The survival in circulation will be monitored by flow cytometry Subsequently, the IgG fraction of patient plasma samples containing polyclonal HLA antibodies will be injected into NSG mice and platelet survival studies repeated to demonstrate a survival advantage of "null" over "wild-type" platelets.
Three hPSC lines with the best MK output will be transfected with each of the paired guide RNAs and Cas9 nickase cloned into 2 vectors containing either GFP or dTomato. Double-positive single cells will be sorted and subsequent colonies will be screened for HLA Class I expression by flow cytometry. Pluripotency will be confirmed in standard assays. This method has been validated on research hPSC lines in our lab.
WP2. Determine off-target rate.
The whole genome sequence of each HLA null line will be compared to the parent line to ascertain the off-target rate. This will be supported by the HipSCi initiative.
WP3. FoP MK production and phenotyping.
Using validated batches of lentiviral vectors from the CiC project we will FoP parent and HLA null lines to compare the dynamics of MK differentiation and proliferation, the potential to freeze-thaw the MKs and their ability to form proplatelets. RNA sequencing will be used to confirm whole genome expression levels are the same in null MKs compared to HLA expressing ones.
WP4. Platelet output and functionality.
We will seed mature FoP MKs from the parent and null lines in our bioreactor to compare the efficiency of platelet production. Platelet functionality will be assessed in a range of assays including adhesion and spreading, aggregation, thrombus formation in collagen-coated flow chambers and ultrastructure analysis by electron microscopy.
WP5. In vivo platelet survival studies.
NSG mice will be injected with 40-240x10^6 (equivalent to a therapeutic dose of platelets [3x10^11] in adult patients) in vitro-produced "null" or "wild-type" platelets or donor-derived platelets. The survival in circulation will be monitored by flow cytometry Subsequently, the IgG fraction of patient plasma samples containing polyclonal HLA antibodies will be injected into NSG mice and platelet survival studies repeated to demonstrate a survival advantage of "null" over "wild-type" platelets.
Planned Impact
The impact of the proposed research project will be on 3 main groups: 1. NHS and patients, 2. Academic beneficiaries and 3. Commercial partners and UK economy.
1. NHS and patients.
The potential advantages of in vitro- over donor-derived platelets are numerous: no risk of transmission of donor-acquired infections, a continuous supply and in particular, better matching. The R&D directorate of the NHSBT (the likely future end user/distributor of in vitro-produced blood cells in England) holds an annual stakeholder meeting that comprises patient and religious groups, industry representatives and blood product prescribers. The programme of research proposed in this application has received strong support at the 2013 and 2015 meetings. This clearly indicates that stakeholders see benefit in in vitro-produced blood products. A market research and customer survey commissioned by CGT identified improved safety, supply and matching as the main drivers for the potential purchase of in vitro-derived platelets by health providers. This engagement with both patients and end users will continue during the lifetime of this grant and will be key to the future promotion of the product and commercialization. The NHSBT will provide practical and financial support for these meetings and events.
The creation of a "universal" platelet product will have a major impact on the provision of platelets to refractory alloimmunised patients who face longer hospital stays (associated with higher costs to the NHS), an increased risk of bleeding and decreased survival. It will also benefit patients who are likely to need repeated prophylactic platelet transfusions such as patients with malignancies or haematological disorders who receive about 50% of all platelet transfusions.
2. Academic beneficiaries.
The main impact of the scientific strategy used in the current proposal is likely to be in the field of regenerative medicine. A nationalnetwork of key hubs has been created through the UK Regenerative Medicine Platform initiative. Dr Ghevaert has an ongoing collaboration with two key hubs in this field, namely, the Biochemistry of the Niche Hub (Prof S Forbes, Edinburgh) and the Pluripotent Stem Cell Hub (Profs P Andrews and A Smith, Sheffield/Cambridge). Through contact with these hubs, dissemination of the knowledge gained within this project can be carried out effectively and we would therefore envisage that this project will have an impact beyond the production of platelets in vitro.
3. Commercial partners and UK economy.
A robust manufacturing protocol for the generation of platelets from hPSCs has a global market of $300m. Our long-term intention is to translate the 3 key technologies of FoP MKs, bioreactor with 3D functionalized scaffolds and HLA null lines into a manufacturing process for human use. Our product is expected to obtain a large share of the market due to advantages in providing consistent cell supply from a validated, pathogen-free source and the generation of "universal" cells. Based on figures from other hPSC regenerative medicine licensing deals, the ROI is expected to be at least 10-fold. The novel platelet production process will therefore have wider benefits, such as providing job opportunities and revenue to the UK economy.
1. NHS and patients.
The potential advantages of in vitro- over donor-derived platelets are numerous: no risk of transmission of donor-acquired infections, a continuous supply and in particular, better matching. The R&D directorate of the NHSBT (the likely future end user/distributor of in vitro-produced blood cells in England) holds an annual stakeholder meeting that comprises patient and religious groups, industry representatives and blood product prescribers. The programme of research proposed in this application has received strong support at the 2013 and 2015 meetings. This clearly indicates that stakeholders see benefit in in vitro-produced blood products. A market research and customer survey commissioned by CGT identified improved safety, supply and matching as the main drivers for the potential purchase of in vitro-derived platelets by health providers. This engagement with both patients and end users will continue during the lifetime of this grant and will be key to the future promotion of the product and commercialization. The NHSBT will provide practical and financial support for these meetings and events.
The creation of a "universal" platelet product will have a major impact on the provision of platelets to refractory alloimmunised patients who face longer hospital stays (associated with higher costs to the NHS), an increased risk of bleeding and decreased survival. It will also benefit patients who are likely to need repeated prophylactic platelet transfusions such as patients with malignancies or haematological disorders who receive about 50% of all platelet transfusions.
2. Academic beneficiaries.
The main impact of the scientific strategy used in the current proposal is likely to be in the field of regenerative medicine. A nationalnetwork of key hubs has been created through the UK Regenerative Medicine Platform initiative. Dr Ghevaert has an ongoing collaboration with two key hubs in this field, namely, the Biochemistry of the Niche Hub (Prof S Forbes, Edinburgh) and the Pluripotent Stem Cell Hub (Profs P Andrews and A Smith, Sheffield/Cambridge). Through contact with these hubs, dissemination of the knowledge gained within this project can be carried out effectively and we would therefore envisage that this project will have an impact beyond the production of platelets in vitro.
3. Commercial partners and UK economy.
A robust manufacturing protocol for the generation of platelets from hPSCs has a global market of $300m. Our long-term intention is to translate the 3 key technologies of FoP MKs, bioreactor with 3D functionalized scaffolds and HLA null lines into a manufacturing process for human use. Our product is expected to obtain a large share of the market due to advantages in providing consistent cell supply from a validated, pathogen-free source and the generation of "universal" cells. Based on figures from other hPSC regenerative medicine licensing deals, the ROI is expected to be at least 10-fold. The novel platelet production process will therefore have wider benefits, such as providing job opportunities and revenue to the UK economy.
Organisations
- University of Cambridge (Lead Research Organisation)
- LOUGHBOROUGH UNIVERSITY (Collaboration)
- National Institute of Health and Medical Research (INSERM) (Collaboration)
- Cell Therapy Catapult (Collaboration)
- Bloodcenter of Wisconsin (Collaboration)
- University of Pavia (Collaboration)
- National Health Service (Collaboration)
- NHS National Services Scotland (NSS) (Collaboration)
- Platelet BioGenesis, Inc (Collaboration)
- University Observatory Munich (Collaboration)
- AstraZeneca (Collaboration)
- Leiden University Medical Center (Collaboration)
- Cardiff University (Collaboration)
- UNIVERSITY OF BIRMINGHAM (Collaboration)
- University of Leuven (Collaboration)
- UNIVERSITY OF SYDNEY (Collaboration)
People |
ORCID iD |
Cedric Ghevaert (Principal Investigator) |
Publications
Crosby A
(2018)
Hematopoietic stem cell transplantation alters susceptibility to pulmonary hypertension in Bmpr2-deficient mice.
in Pulmonary circulation
Description | Biogenesis and bioengineering of human platelets |
Amount | £1,200,000 (GBP) |
Funding ID | 219472/Z/19/Z |
Organisation | Wellcome Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 04/2020 |
End | 04/2025 |
Description | EU Horizon 2020 FET |
Amount | € 4,800,000 (EUR) |
Organisation | European Commission |
Department | Horizon 2020 |
Sector | Public |
Country | European Union (EU) |
Start | 11/2017 |
End | 10/2021 |
Description | Generating platelets in vitro for the clinic: optimisation and added clinical efficacy |
Amount | £474,809 (GBP) |
Funding ID | MR/V005413/1 |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 09/2020 |
End | 09/2024 |
Description | SilkPlateket |
Amount | € 2,500,000 (EUR) |
Funding ID | 101058349 |
Organisation | European Commission |
Department | Horizon 2020 |
Sector | Public |
Country | European Union (EU) |
Start | 09/2022 |
End | 09/2025 |
Description | Supercharging platelets as a novel therapy for promoting the repair of cardiac tissue post myocardial infarction. |
Amount | £950,000 (GBP) |
Funding ID | FS/19/54/34889D |
Organisation | British Heart Foundation (BHF) |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 04/2022 |
End | 04/2025 |
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 | UK Regenerativ Medicine Platform |
Amount | £3,700,000 (GBP) |
Organisation | UK Regenerative Medicine Platform |
Sector | Academic/University |
Country | United Kingdom |
Start | 03/2018 |
End | 02/2021 |
Description | Universal cells to overcome HLA barriers in regenerative medicine |
Amount | £836,002 (GBP) |
Funding ID | MR/S02090X/1 |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 12/2018 |
End | 11/2022 |
Title | Production in vitro of megakaryocytic by a forward programming method |
Description | This methods which is now published in a book chapter allows potential users to produce megakaryocytes in their own laboratory from pluripotent stem cell lines. It also allows them to use our own inducible cell lines which does not rely on lentiviruses to forward programme. Complementary to this method is a methods movie that illustrate the text published in the book. |
Type Of Material | Cell line |
Year Produced | 2018 |
Provided To Others? | Yes |
Impact | We have now >15 collaborative groups that are using the technology published in 2016 in Nature Communications |
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 | 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 Platelet Biogenesis |
Organisation | Platelet BioGenesis, Inc |
Country | United States |
Sector | Private |
PI Contribution | Platelet Biogenesis is a company based in Boston which has licensed the MK forward programming technology. We have now a sponsored research agreement that allows us to pursue this work further with the company itself |
Collaborator Contribution | They have contributed to cell line research in our laboratory and are testing our cell lines in the bioreactor in Boston. |
Impact | Successful further funding for Platelet Biogenesis which has allowed them to underwrite a sponsored research agreement with my research group. |
Start Year | 2017 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | Bloodcenter of Wisconsin |
Country | United States |
Sector | Hospitals |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | Cardiff University |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | Leiden University Medical Center |
Country | Netherlands |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | Loughborough University |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | National Institute of Health and Medical Research (INSERM) |
Country | France |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | University Observatory Munich |
Country | Germany |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | University of Birmingham |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | University of Leuven |
Country | Belgium |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaboration wth multiple academic groups who are now using our forward programming technology for the production of MKs |
Organisation | University of Sydney |
Country | Australia |
Sector | Academic/University |
PI Contribution | In short we have facilitated the transfer of the forward programming technology to various laboratories that use it for disease modelling and basic research |
Collaborator Contribution | We are benefitting from our technology being potentially used for publication |
Impact | None so far |
Start Year | 2015 |
Description | Collaborations with Paris and Pavia for the production of platelets in vitro (part of EU grant) |
Organisation | National Institute of Health and Medical Research (INSERM) |
Country | France |
Sector | Academic/University |
PI Contribution | The team in Pavia and Paris are using the pluripotent stem cell lines generated in the Ghevaert group to do disease modelling or platelet production in 3D bioreactors. The latter can be functionalised with recombinant proteins that have been identified and generated in my lab, that can promote platelet release. |
Collaborator Contribution | The team in Pavia is providing the 3D silk-based bioreactors. The team in Paris gives us access to patients cell lines for disease modelling. |
Impact | Successful EU grant application |
Start Year | 2015 |
Description | Collaborations with Paris and Pavia for the production of platelets in vitro (part of EU grant) |
Organisation | University of Pavia |
Country | Italy |
Sector | Academic/University |
PI Contribution | The team in Pavia and Paris are using the pluripotent stem cell lines generated in the Ghevaert group to do disease modelling or platelet production in 3D bioreactors. The latter can be functionalised with recombinant proteins that have been identified and generated in my lab, that can promote platelet release. |
Collaborator Contribution | The team in Pavia is providing the 3D silk-based bioreactors. The team in Paris gives us access to patients cell lines for disease modelling. |
Impact | Successful EU grant application |
Start Year | 2015 |
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 | 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 |
Title | Insulator to prevent silencing of inducible system to make MKs from iPSCs |
Description | We have shown improvement to a doxycycline inducible system driving MK differentiation from iPSCs by adding insulators to the original construct |
IP Reference | GB 2312699.8. |
Protection | Patent / Patent application |
Year Protection Granted | 2023 |
Licensed | Commercial In Confidence |
Impact | Much more efficient maturation and somatic cells from iPSCS through forward programming approach. |
Company Name | Xap Therapeutics |
Description | Xap Therapeutics develops a biotechnology platform aiming to assist with the design of cell therapeutics. |
Year Established | 2017 |
Impact | None as yet |
Website | http://www.xaptherapeutics.com |
Company Name | Celladvice Ltd |
Description | |
Year Established | 2019 |
Impact | Currently providing expert advice to 4 different companies |
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 | Charity concert 2023 |
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 support of Action for Lung Fibrosis |
Year(s) Of Engagement Activity | 2023 |
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 | 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 | Panel discussion organised by BARDA (US): State of the Technology Meeting: Blood and Blood Products (Washington, US) |
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 | Panel discussion on the challenges and investment needed to develop in vitro blood cell therapies. |
Year(s) Of Engagement Activity | 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 | 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 | Youtube distributed movie on production of blood cells in vitros |
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 asked the general public and patients groups to send us questions about the production of blood cells in vitro. Our replies are presented in the form of a short movie that is available on Youtube: |
Year(s) Of Engagement Activity | 2018 |
URL | https://www.youtube.com/watch?v=TCKP0dn2uHk&t=7s |