£18m network of gene therapy hubs to be created in the UK

LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council (BBSRC), have announced three major investments totalling £18 million to create a network of gene therapy innovation hubs.

The funding will support the creation of three facilities to advance the clinical development of new genetic treatments, with potential to transform care for patients, including those with rare and fatal 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 facilitate academic-led clinical trials of novel gene therapies, enabling research to reach patients. Gene therapies offer huge potential as treatments for a wide range of conditions but academics find it difficult to get access to the clinical materials, facilities and expertise required to progress gene therapy research into clinical trials.

The hubs will facilitate development for 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 the future. 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.

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.”

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