Sheffield’s pioneering gene therapy centre provides hope to millions

Over 300 million people worldwide live with a genetic disease, and there are over 7,000 rare diseases with no cure. 

Genetic diseases are caused by changes in our DNA and can affect both children and adults. These changes can happen in various ways, such as a mutation in a gene or an abnormality in a chromosome. 

Most of these conditions are life-threatening or have a significant impact on a person’s quality of life, with treatment options often failing to stop their progression.

The new Gene Therapy Innovation and Manufacturing Centre (GTIMC) at the University of Sheffield is set to accelerate the development of cutting-edge gene therapies and clinical trials, offering hope for patients with conditions such as motor neuron disease, cystic fibrosis, and haemophilia.

Professor Mimoun Azzouz, Chair of Translational Neuroscience, and Director of the GTIMC is at the forefront of a new generation of medicine set to save lives and rewrite futures.

As a leading international expert in gene therapy, with a track record of translational research productivity, Professor Azzouz holds many prestigious awards that acknowledge his pioneering research, including the ERC Advanced Investigator (2011), ERC Proof-of-Concept (2017) and IMI award (2020). 

“Sheffield, already internationally renowned for its world-class rare disease research, has emerged as one of the leading players in gene therapy. We were the first site in the UK to deliver a genetic therapy trial for motor neuron disease (MND) in collaboration with the pharmaceutical company Biogen. Research by scientists at the University of Sheffield found that gene silencing could slow down the damage caused by a mutation in the SOD1 gene which is responsible for two per cent of MND cases,” explains Professor Azzouz.

“It is hoped that the new GTIMC will provide patients with access to cutting-edge treatments that were previously unimaginable” he adds.

The impact of gene therapy research at Sheffield

Professor Azzouz, a member of the Neuroscience Institute at the University of Sheffield, is committed to using his scientific knowledge to help patients suffering from some of the most devastating diseases in medicine.

He played a pivotal role in the development of a pioneering gene therapy treatment for babies with Spinal Muscular Atrophy (SMA) type 1 – demonstrating the impact of Sheffield’s research, and its capability to transform thousands of lives across the world.

As a neuroscientist and gene therapy expert, I am particularly proud of the translational medical research innovated here in Sheffield. Gene therapy is arguably the most exciting area of therapeutics for rare diseases due to recent progress in the field and the promising possibilities.

Professor Mimoun Azzouz

Chair of Translational Neuroscience and Director of the GTIMC at the University of Sheffield

“Our Sheffield researchers and clinicians were part of a team that demonstrated the use of advanced stem cell therapy to treat multiple sclerosis. To do this they used stem cells from the patient's own bone marrow to effectively reboot their immune system, halting its attack on the nervous system. The trial has produced promising results, with some patients having regained their ability to walk, run and even ride a bike.”

A new centre for Gene Therapy Innovation and Manufacturing

The GTIMC builds, and expands, on Sheffield’s existing expertise and ecosystem by bringing together academic institutions, NHS trusts, non-profit and industry partners across the north of England, Midlands, and Wales. 

It has been formed as part of the Innovation Hubs for Gene Therapies, a new UK initiative designed to advance the clinical development of new genetic treatments by producing clinical-grade vectors, alongside translational support, and regulatory advice. 

“Our leadership and track record in the field led to the University of Sheffield being chosen to house one of three new pioneering gene therapy hubs in an £18 million network funded by LifeArc and the Medical Research Council (MRC), with the support from the Biotechnology Services Research Council (BBSRC),” explains Professor Azzouz.

“The establishment of the innovation hubs will improve the UK capacity for producing gene therapy treatments - this means we will have the scale required to conduct vital Phase 1 and Phase 2 clinical trials which are needed to test the safety and effectiveness of treatments in patients.”

“The new GTIMC facility will not only drive new gene therapies and clinical trials, but it will build career pathways for people in the region and start to develop Sheffield and Yorkshire as a hub for gene therapy in the future. The economic benefits could be considerable, given the potential to catalyse the creation of new start-up companies - similar to the near-explosion of activity seen around the ‘golden triangle’ of London-Cambridge-Oxford. The recently established Sheffield gene therapy start-up companies such as BlackfinBio and Crucible Therapeutics are a result of the excellent ecosystem enabling attraction of investments to the region.”

"The Sheffield Gene Therapy Hub is a game changer that will place the region at the heart of the next generation of medicine, as well as providing hope for the millions living with genetic disease" he adds.

The state-of-the-art centre, which was officially opened by Her Royal Highness, The Princess Royal in October 2024, will allow researchers and clinicians at Sheffield to make a greater impact on the lives of many patients with genetic diseases and their families.

Written by Anna Blagg, Research Marketing and Communications Content Coordinator