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.

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.

Publications

10 25 50