Network of gene therapy hubs ‘offers hope to millions of patients’

Dave Higgens, PA
·3-min read

Three state-of-the-art gene therapy innovation hubs are being set up across England aimed at ensuring cutting-edge innovation is translated into transformative treatments which offer hope to millions with life-threatening diseases.

The planned network of hubs, at King’s College London, NHS Blood and Transplant in Bristol and a new University of Sheffield facility in South Yorkshire, will speed up the development of a new wave of genetic medicines, researchers say.

The charity LifeArc and the Medical Research Council (MRC), who are behind the £18 million move, say it is aimed at helping the most innovative research to reach patients.

They say the UK has a world-class genetics research base but academics have struggled to get access to the practical means to progress research into trials.

They say the new hubs will give academics this support and operate as a co-ordinated network, sharing technical skills and resources.

LifeArc chief executive Melanie Lee 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.”

MRC’s executive chairwoman Professor Fiona Watt said: “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.”

Revolutionary gene therapy aims to treat conditions by engineering another gene to replace, silence or manipulate the faulty one.

The new hubs will manufacture commonly used vectors including both lentivirus and adeno-associated virus (AAV) that are needed for genetic therapy trials.

The network will also facilitate simplified licensing agreements and streamline regulatory reviews.

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One of the hubs will be the University of Sheffield’s Gene Therapy Innovation and Manufacturing Centre (GTIMC), led by Professor Mimoun Azzouz, chairman of Translational Neuroscience at the university.

The new centre, which will be the first in the north of England, is planned for a site in Rotherham, close to the university’s high-profile Advanced Manufacturing Research Centre (AMRC).

Prof Azzouz said: “Sheffield has emerged as one of the leading players in cell and gene therapy and this national network of partners, facilities and training programmes will allow us to keep pace with translational discoveries for new and potentially life-changing treatments.

“This is a momentous milestone for revolutionary medical advances not only for Sheffield and South Yorkshire, but also for the north of England and the UK.”

Professor Dame Pam Shaw, director of the NIHR Sheffield Biomedical Research Centre said: “This exciting development and partnership will speed up the pull through of new gene therapies into early phase clinical trials and offer hope to patients with neurological and other rare diseases that can be addressed in this way.”

NHS England chief scientific officer Professor Dame Sue Hill said: “The NHS is already a global leader in introducing gene therapies for patients and this latest announcement is a welcome addition to the work going on in this area as the NHS continues to work with academia and life sciences organisations to expand the range of treatments available.

“The NHS has led the world in the development of a range of other innovations and therapies including the first-ever treatment for Covid-19 and last year becoming the first country in the world to vaccinate against the virus, outside of clinical trials, with more than 20 million people in England jabbed only 100 days later.”