LifeArc, MRC and BBSRC create £18m network of gene therapy hubs to advance promising research into new treatments for patients

LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC), today announce three major investments totalling £18m to create a national network of cutting-edge “Gene Therapy Innovation Hubs”.

The £18m funding will support the creation of three dedicated facilities to advance the clinical development of new genetic treatments, with potential to transform care for millions of patients including those with rare and life-threatening genetic diseases. Hubs will be at King’s College London, NHS Blood and Transplant in Bristol and the University of Sheffield.

These Innovation Hubs will enable academic-led clinical trials of novel gene therapies to take place, helping the most innovative research to reach patients. Gene therapies offer huge potential as treatments for a wide range of conditions and the UK has a world-class genetics research base – however, to date, academics have found it difficult to get access to the clinical materials, facilities and expertise required to progress gene therapy research into clinical trials.

The Hubs will unlock development pathways for these new treatments by offering access to GMP (good manufacturing practice) facilities for clinical trial materials, alongside essential translational support and regulatory advice. The Hubs will operate as a coordinated network, sharing technical skills and resources to enable innovative gene therapy research.

Dr Melanie Lee, CEO of LifeArc, said: “Recent innovations in gene therapies hold enormous potential for treating conditions such as rare diseases, but often promising ideas – particularly in academia – are not making it through to patients. Through our collaboration, we aim to meet the need for researchers to have access to the essential facilities and translational advice to progress promising research.”

Professor Fiona Watt, MRC’s Executive Chair said: “Support for innovative advanced therapies has been a long-standing priority for MRC and so we are delighted to announce this unique partnership with LifeArc. The new network of Innovation Hubs for gene therapies will build on the UK’s great strengths in this area, providing targeted investment in vital infrastructure to accelerate academic research programmes down the path to patient benefit, supporting the delivery of a new wave of genetic medicines.”

The Innovation Hubs will manufacture commonly used vectors including both lentivirus and adeno-associated virus (AAV) that are needed for genetic therapy trials, while positioning the UK for significant bioprocessing innovation work with the potential to radically increase yields and reduce productivity barriers in future years. The network will also design and share commercially ready platforms, using common cell-lines, plasmids and reagents to reduce costs, facilitate simplified licensing agreements and streamline regulatory reviews. A key aim is to smooth the transition between small-scale supply for early clinical trials through to larger-scale manufacture for patient trials, and beyond.

Dr Lee Beniston, BBSRC’s Associate Director for Industry Partnerships & Collaborative R&D, noted: “Gene therapies have outstanding clinical potential, but their development is critically dependent on the manufacture of the underpinning viral vector delivery technology. Over a number of years, BBSRC has made significant investments to help support bioprocess research and development; we are therefore delighted to be investing in this network of Hubs which will harness the UK’s excellence in bioprocess innovation to tackle key challenges in viral vector manufacturing.”

The creation and ongoing operation of the Hub network will be overseen by a cross-network Coordination Committee to promote sharing of knowledge and capabilities, engage with the academic community and foster interactions with commercial organisations to facilitate the onward the development of new genetic medicines.

About the centres

The University of Sheffield Gene Therapy Innovation Manufacturing Centre (GTIMC)

Sheffield’s GTIMC, led by Professor Mimoun Azzouz, builds on a strong history of translational gene therapy research at the University of Sheffield and partners. The centre includes a new state-of-the-art modular GMP manufacturing facility, located at the University of Sheffield’s Innovation District, that will support gene therapy projects emerging from UK universities.

The facility will utilise highly efficient processes to generate clinical grade AAVs and provide all the necessary quality assurance, regulatory certification and governance. The facility will provide clinical grade material for human trials at Advanced Therapies Treatment Centres and NHS trusts within the GTIMC and the national network. The GTIMC will deliver translational and regulatory support alongside an extensive training and skills programme to enable upskilling and address shortage of skills in GMP manufacturing.

NHS Blood and Transplant Gene Therapy Hub

The NHSBT Gene Therapy Hub will be hosted within a new, state of the art, 1,000m2 facility for the production of gene therapies under GMP, funded jointly by NHSBT and the Department of Health and Social Care and under construction at the NHSBT Filton Blood Centre (Bristol).

Due to be operational by the end of 2021, the new Hub will support early phase academic-led gene therapy trials and facilitate the provision of cost-effective viral vectors and plasmid DNA to stimulate the UK’s gene therapy sector.

The Hub, led by Dr Jon Smythe and Dr Paul Lloyd-Evans, will provide viral vector manufacturing, training and support services for academic-led groups seeking Adeno Associated Viral (AAV), Lentiviral (LV) vectors and plasmid DNA at GMP and research-grade qualities. It will also support academic-led teams in the translation of their research to the clinic and work with the other Hubs to develop, optimise and deliver a comprehensive training package to generate a highly skilled workforce, serving both the academic and commercial gene therapy communities.

The King’s College London/Royal Free/UCL Gene Therapy Hub

The King’s College London/Royal Free/UCL Hub, , led by Professor Robin Ali, will provide a comprehensive capability for clinical grade viral vector manufacturing. 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. In partnership with the network of Innovation Hubs, our vision is to ensure that the UK capitalises on its outstanding academic medical research to deliver novel gene therapies to patients, providing transformative treatments for currently intractable conditions, and generating a vibrant economic landscape.

Background notes

Gene therapies have potential to deliver treatments for a wide range of conditions, including around 7000 rare diseases (the majority of which are caused by one faulty gene). Gene therapy aims to treat – or even cure – these conditions, by engineering another gene to replace, silence or manipulate the faulty one. The therapeutic gene is frequently introduced via a ‘viral vector’, a virus particle modified to remove all unwanted or harmful properties.

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