University of Sheffield to help lead new UK research into rare disease treatments

A Yorkshire university will be at the forefront in research to advance scientific discoveries into life-changing treatments.

Thursday, 18th March 2021, 6:00 am

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

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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. Photo credit: Getty Images

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 described the step as a “momentous milestone” for revolutionary medical advances not only for Sheffield and South Yorkshire, but also for the North of England and the UK.

He said: "Sheffield has emerged as one of the leading players in the 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."

The University of Sheffield’s Gene Therapy Innovation and Manufacturing Centre (GTIMC). Photo credit: The University of Sheffield

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

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 aim is for the new hubs to 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.

Professor Mimoun Azzouz, chairman of Translational Neuroscience at the university. Submitted picture

“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 added: “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.

Pictured, Professor Koen Lamberts, the Vice-Chancellor of the University of Sheffield. He said: "This is fantastic news for the City Region and the North of England." Photo credit: JPIMedia

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

Alongside the national network funding from LifeArc, Sheffield University will also use a record multi-million-pound donation to help support the new hub.

Previously the Law Family Charitable Foundation (LFCF), which was established by Andrew and Zoë Law to support charity schemes with an emphasis on education and health, gave the University of Sheffield £5.85m - the largest single gift from an individual alumnus in its history.

From the record donation £3m will go towards searching for ways of treating rare diseases.

Professor Koen Lamberts, the Vice-Chancellor of the University of Sheffield, said: "The Gene Therapy Innovation and Manufacturing Centre will unlock development pathways for new treatments for people affected by devastating genetic disorders, many of which have no cure."

He added: "This is fantastic news for the City Region and the North of England."

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