Gene therapy innovation centre will lead to life-changing treatments

A new gene therapy innovation centre at the University of Sheffield is on track to advance scientific discoveries into treatment options for millions of patients with life-threatening diseases.

Announced on 18 March 2021, the Gene Therapy Innovation and Manufacturing Centre (GTIMC) is one of three new pioneering hubs and will be the first in the north of England.

GTIMC is part of a new £18 million network funded by LifeArc and the Medical Research Council (MRC), and is supported by the Biotechnology and Biological Sciences Research Council (BBSRC).

Gene therapy could potentially offer treatment for over 7,000 rare diseases that are currently without a cure. The aim of gene therapy is to treat these diseases by engineering another gene to replace, silence or manipulate the faulty one.

While the UK has an exceptional genetics research base, finding access to clinical materials, facilities and expertise needed to progress gene therapy research into clinical trials, poses a challenge to scientists.

Professor Mimoun Azzouz, Chair of Translational Neuroscience at the University of Sheffield, and director of the GTIMC, said: “The Gene Therapy Innovation and Manufacturing Centre will tackle major challenges in gene therapy development for some of the most devastating diseases.

“Gene therapies are pioneering medical advances that have the potential to offer much-needed, novel, effective treatments for many rare and incurable diseases that cannot be treated by conventional drug compounds.

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

Professor Dame Pam Shaw, Director of the NIHR Sheffield Biomedical Research Centre and co-applicant on the GTIMC application added: “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.

“The support given to this initiative will greatly accelerate the translational potential of genetic therapies in the UK and bring benefits in key areas of unmet medical need.”

The GTIMC will provide vital translational and regulatory support, as well as extensive training and skills programmes in order to enable upskilling and address the shortage of skills in Good Manufacturing Practice (GMP) manufacturing.

Moreover, it will connect academic institutions, NHS trusts, non-profit and industry partners across the north of England, Midlands and Wales, facilitating academic-led clinical trials of novel gene therapies.

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