Gene therapy center aims to cure the incurable

“Gene therapy has huge potential. It has already led to cures for conditions such as spinal muscular atrophy (SMA). In the future, we will be looking at conditions like motor neurone disease, Alzheimer’s and rare cancers,”

April 30, 2021
Jeff Bouley
Gene therapy center aims to cure the incurable

A new dedicated gene therapy hub in Northern England is seeking to fast-track treatments for diseases and conditions which are at present incurable.

The Gene Therapy Innovation and Manufacturing Centre (GTIMC) housed in Sheffield will be one of three cutting-edge hubs in the United Kingdom dedicated to advancing the training, capacity building, and development of clinical development of new genetic treatments.

It is the only such center north of London and will look toward pioneering scientific discoveries into promising treatment options for millions of patients with life threatening diseases.

It is part of an £18-million network created by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology and Biological Sciences Research Council.

Prof. Sherif El-Khamisy led the University of Bradford in the consortium’s successful bid—he also is director of the Institute of Cancer Therapeutics at the university and said the scheme would develop new cures to conditions which are at present considered “incurable,” including inherited disorders.

“Gene therapy has huge potential. It has already led to cures for conditions such as spinal muscular atrophy (SMA). In the future, we will be looking at conditions like motor neurone disease, Alzheimer’s and rare cancers,” noted El-Khamisy. “[There] are disorders which cannot be cured by traditional therapeutics, including SMA and MND—gene therapy is expected to deliver cures for those disorders.”

In fact, gene therapy is a promising treatment option for more than 7,000 rare diseases that currently have no cure. 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 Gene Therapy Innovation and Manufacturing Centre will tackle major challenges in gene therapy development for some of the most devastating diseases,” said Prof. Mimoun Azzouz, director of the GTIMC and chair of the Translational Neuroscience department at the University of Sheffield. “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.”

The state-of-the-art center will bring together academic institutions, National Health Service trusts, and  non-profit and industry partners across the north of England, Midlands, and Wales to help enable clinical trials of novel gene therapies. The GTIMC will deliver essential translational and regulatory support alongside extensive training and skills programs.

“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,” said Prof. Dame Pam Shaw, director of the NIHR Sheffield Biomedical Research Centre and co-applicant on the GTIMC application. “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.”

Added Prof. Fiona Watt, MRC’s executive chair: “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 programs down the path to patient benefit, supporting the delivery of a new wave of genetic medicines.”

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