King's/Royal Free/UCL Gene Therapy Innovation Hub
Lead Research Organisation:
King's College London
Department Name: Genetics and Molecular Medicine
Abstract
The Kings/Royal Free/UCL Gene Therapy Innovation Hub will manufacture clinical-grade gene therapies for the UK academic and clinical community. This will allow promising treatments for a wide range of rare and common diseases to be tested in patients. The results from these early clinical studies may then support larger-scale trials and ultimately new therapies for patients.
Provision of suitable quality (GMP) gene therapy product is a key limiting factor for progress in this exciting field. Our Hub will address this directly through a major increase in UK capacity. This will cover the major types of gene therapy used, adeno-associated virus (AAV) and lentivirus, as well as gamma-retrovirus. In addition, we will invest in developing new approaches to increase the amount of gene therapy product that can be made at one time (in one batch). This will mean that each trial needs fewer batches, and that applications needing high doses (for example, administration systemically) become possible. It will also reduce costs of manufacture.
We will also work to increase the UK's overall capabilities to manufacture gene therapies, through creation and provision of dedicated training courses, both online and in person, at a variety of levels, to suits varying needs of different groups. In addition to this, we will lend our expertise to other developing Gene Therapy Innovation Hubs, to help them to become operational as quickly as possible.
King's, Royal Free and UCL offer unmatched expertise in the UK in gene therapy manufacturing, and we are ideally-placed to contribute to the UK's success in improving therapies for many poorly-treated diseases, and generating sustainable economic benefit for the country.
Provision of suitable quality (GMP) gene therapy product is a key limiting factor for progress in this exciting field. Our Hub will address this directly through a major increase in UK capacity. This will cover the major types of gene therapy used, adeno-associated virus (AAV) and lentivirus, as well as gamma-retrovirus. In addition, we will invest in developing new approaches to increase the amount of gene therapy product that can be made at one time (in one batch). This will mean that each trial needs fewer batches, and that applications needing high doses (for example, administration systemically) become possible. It will also reduce costs of manufacture.
We will also work to increase the UK's overall capabilities to manufacture gene therapies, through creation and provision of dedicated training courses, both online and in person, at a variety of levels, to suits varying needs of different groups. In addition to this, we will lend our expertise to other developing Gene Therapy Innovation Hubs, to help them to become operational as quickly as possible.
King's, Royal Free and UCL offer unmatched expertise in the UK in gene therapy manufacturing, and we are ideally-placed to contribute to the UK's success in improving therapies for many poorly-treated diseases, and generating sustainable economic benefit for the country.
Technical Summary
The King's/Royal Free/UCL Hub will provide a comprehensive capability for GMP viral vector manufacturing, addressing the primary barrier to academic-led development of gene therapies. This will include both AAV and lentivirus production for early-phase trials, alongside substantial programmes in process innovation, knowledge transfer, and training to address critical skills shortages.
We are uniquely placed to deliver this vision. MRC/LifeArc investment will build on nationally leading expertise in GMP vector production - we have manufactured more lentiviral vectors for clinical trials than any other centre in Europe, and are the only academic group in the UK to have released GMP lentivirus or AAV. We have experience in gene therapy pre-clinical development and clinical trials, alongside a track record in commercialisation (inc. MeiraGTx PLC founded by Ali). We lead the Research England-funded London Advanced Therapies partnership; this, combined with strong links to other centres, places us close to the academic groups developing new gene therapies and driving demand for GMP vectors.
Investment in our Hub will provide a near-immediate enhancement of UK manufacturing capacity, providing time for newer centres to come online. We will make our expertise available to support other centres directly and via the Cell & Gene Therapy Catapult. With our UCL Hub members, we will develop postgraduate training programmes for viral vector manufacturing, focusing on regulatory and technical aspects, and new on-line training modules.
Viral vector manufacturing - particularly for early-phase trials - is limited by yield, scalability of technologies, and analytic standards. KCL and Royal Free Hub members will undertake a substantial programme of process innovation work to address these challenges.
All these activities will link to our Hub's UK-leading early phase clinical trial activities in gene therapy, based at King's, Royal Free and UCL.
We are uniquely placed to deliver this vision. MRC/LifeArc investment will build on nationally leading expertise in GMP vector production - we have manufactured more lentiviral vectors for clinical trials than any other centre in Europe, and are the only academic group in the UK to have released GMP lentivirus or AAV. We have experience in gene therapy pre-clinical development and clinical trials, alongside a track record in commercialisation (inc. MeiraGTx PLC founded by Ali). We lead the Research England-funded London Advanced Therapies partnership; this, combined with strong links to other centres, places us close to the academic groups developing new gene therapies and driving demand for GMP vectors.
Investment in our Hub will provide a near-immediate enhancement of UK manufacturing capacity, providing time for newer centres to come online. We will make our expertise available to support other centres directly and via the Cell & Gene Therapy Catapult. With our UCL Hub members, we will develop postgraduate training programmes for viral vector manufacturing, focusing on regulatory and technical aspects, and new on-line training modules.
Viral vector manufacturing - particularly for early-phase trials - is limited by yield, scalability of technologies, and analytic standards. KCL and Royal Free Hub members will undertake a substantial programme of process innovation work to address these challenges.
All these activities will link to our Hub's UK-leading early phase clinical trial activities in gene therapy, based at King's, Royal Free and UCL.
| Description | University of Sheffield MRC/LifeArc Gene Therapy Innovation Hub |
| Organisation | University of Sheffield |
| Country | United Kingdom |
| Sector | Academic/University |
| PI Contribution | Regular meetings to discuss GT manufacturing |
| Collaborator Contribution | Regular meetings to discuss GT manufacturing |
| Impact | To early for specific outputs |
| Start Year | 2022 |
| Description | 27th Feb 2025 - Visit from 26 students from MSc biopharmaceutical from KCL |
| 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 | Postgraduate students |
| Results and Impact | 27th Feb 2025 - Visit from 26 students from MSc biopharmaceutical from KCL. Talks from multiple staff to give overview of different roles and careers avenues, then given virtual tour, followed by Q&A with senior staff and directors to give overview of working in a GMP facility, how they can follow this as a career path, various working opportunities, group leader reported that students were very engaged and showed an increased interest in entering this field in the future. |
| Year(s) Of Engagement Activity | 2025 |
| Description | 9th December 2024 - KCL Maths School workshop, Stitch & Stem |
| Form Of Engagement Activity | Participation in an activity, workshop or similar |
| Part Of Official Scheme? | No |
| Geographic Reach | Local |
| Primary Audience | Schools |
| Results and Impact | Stitch and stem public engagement event held as part of the KCL Maths School Workshop. A Level stundets were invited to spend an evening embroidering scientific images whilst engaging in conversations with researchers, technicians and students to learn more about the research. The activity is a valuable opportunity for attendees to learn about the images they are recreating, the broader aim of our research and many other topics around the research, such as career pathways, scientific approaches etc. The discussions were ongoing during the event and participants were very engaged and happy to learn about the research. The students were also particularly interested in learning about the various career pathways available to them. |
| Year(s) Of Engagement Activity | 2024 |
| Description | GTVF - MSC student GMP experience day, 22nd Feb 2024 |
| 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 | Postgraduate students |
| Results and Impact | We welcomed KCL MSc Biopharmaceuticals students on the 22nd of February and UCL BIoprocessing and commercialisation students on the 23rd Feb 2024. The days were an opportunity for them to learn more about working in a GMP facility, with a tour of the working facilities, talks by several members of the team working in a range of roles, a Q&A session and networking. The aim was to give them a better understanding of working in a GMP facility and an opportunity to learn more and operations and opportunitues from those working at GTVF. The students were very engaged and interested to learn more about the potential career pathways available to them. |
| Year(s) Of Engagement Activity | 2024 |
| Description | Oct 2024 - New Scientist Live - Stitch and Stem 3 day event |
| 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 | CGTRM hosted a stand for the full 3 days of the New Scientist Live Event in Excel, where we hosted a Stitch and Stem public engagement activity that invites participants to embroider simplified designs of scientific images whilst learning about the research. For inclusivity, we also offer the option to replicate scientific images on black card using neon pens. The event was attended by over 20,000 people and the stand was exceptionally busy for the entire duration. We welcomed primary and secondary aged children, undergraduates, postgraduates, professionals (inc. medics) and members of the general public, who learned about the cells they were either stitching or drawing (we also had the option to contribute to a larger, collaborative embroidery piece). We had many engaging conversations with children and young people about the research (aiming to make the infromation accessible, to breakdown barriers and myth bust), the different career options and routes etc. Attendees were interested and wanted to learn more about our current and past research. We have placed some of our resources on our website at the request of many attendees, including teachers who wanted to replicate the activity in the classroom so the work will continue to be discussed long after the event. We also engaged in conversations with others who participate in other art communication activities and hope this will be the start of new PPE opportunities. Finally, one of the visitors (an A Level Student) has undertaken work experience within our centre, while another will be spending time with a collaborator we were able to put them in touch with. Quotes from attendees: "Stitch and Stem makes biology more fun by learning how to draw cells in simple diagrams with multiple colours" "I learnt about different cell types and that they all fit inside everyone's body. Amazing!" Quote from teacher who attended: "Stitch and Stem was the most incredible workshop - I loved speaking to the scientists and finding out more about their research, and learning about their studies, their jobs, and how things really worked. I had an amazing time and I am so glad I got to take part!" |
| Year(s) Of Engagement Activity | 2024 |
| URL | https://live.newscientist.com/ |