Princess Royal Opens Gene Therapy Innovation Centre

Her Royal Highness, The Princess Royal, officially opened the Gene Therapy Innovation and Manufacturing Centre (GTIMC) at the University of Sheffield today (2 October 2024). The state-of-the-art facility marks a significant milestone in accelerating the development of life-changing treatments for patients with rare and inherited diseases.

The GTIMC brings together leading researchers, clinicians, and industry partners to accelerate the translation of groundbreaking gene therapy research into clinical trials. By replacing, silencing, or manipulating faulty genes, these therapies offer hope for patients with conditions such as adult and childhood forms of motor neuron diseases, cystic fibrosis and haemophilia.

During her visit, The Princess Royal met with patients who will directly benefit from the innovative work being conducted at the GTIMC. The Princess Royal praised the centre's potential to revolutionise healthcare and improve the lives of countless individuals.

Professor Mimoun Azzouz, Director of the GTIMC, said: "Gene therapy is a promising treatment option for more than 7,000 rare and inherited diseases that are currently without a cure. Our goal is to provide patients with access to cutting-edge treatments that were previously unimaginable.

"We're delighted that Her Royal Highness, The Princess Royal, has been able to meet some of our researchers and patients to learn more about the life changing work happening here at the University of Sheffield.

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

The University of Sheffield is internationally renowned for its world-class neurodegenerative research. With state-of-the-art laboratories and equipment, a clinical database of over 2,000 patients and a large resource of human biosamples and brain-bank material generously donated by patients, Sheffield has earned its place as one of the leading players in gene therapy research and innovation.

Dharmisha Stezaly's son Sebastian, aged three, has Spinal Muscular Atrophy - a childhood form of motor neuron disease. Sebastian received a gene therapy called AAV9-SMN (Zolgensma) which is currently the only disease altering medication available for Spinal Muscular Atrophy.

"The gene therapy Zolgensma has changed our lives in an immeasurable way," said Dharmisha.

"At the time of administration, we didn't know what the future held. But Sebastian has gone from strength to strength and is such a joy - I am incredibly proud and privileged to be his parent.

"I look forward to a day, where newborn screening for Spinal Muscular Atrophy is nationwide, allowing faster access to this gene therapy, which can be considered curative when administered early enough."

The new GTIMC facility will not only accelerate development of 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 centre has already brought 27 highly skilled jobs to the region.

Professor Ashley Blom, Vice President and Head of the Faculty of Health at the University of Sheffield, said: "At the University of Sheffield we focus our research on finding real-world solutions to some of the biggest global challenges. The GTIMC will unlock development pathways for new treatments for people affected by devastating genetic disorders.

"By addressing the current shortfall in gene therapy production capacity within the UK, the GTIMC will play a pivotal role in accelerating the translation of research into clinical applications. It will also deliver extensive training and skills development for the next generation of scientists and researchers in this emerging field of medicine.

"This is fantastic news for the South Yorkshire region and the North of England and we look forward to working collaboratively to share technical skills and resources across the coordinated network."

The GTIMC, located close to the University's Advanced Manufacturing Research Centre (AMRC), is one of three cutting-edge hubs in the UK dedicated to advancing the clinical development of new genetic treatments. It is part of an £18 million network funded by LifeArc and the Medical Research Council (MRC), with support from the Biotechnology Sciences Research Council (BBSRC). The University of Sheffield is also grateful for over £4.5 million donated to support the GTIMC, with particular thanks to the Law Family Charitable Foundation, the Wolfson Foundation, Garfield Weston Foundation and the Gordon Bramah Charitable Settlement.

The Innovation Hubs for Gene Therapies, located at the University of Sheffield, NHS Blood and Transplant in Bristol, and King's College London (in partnership with University College London and the Royal Free NHS Foundation Trust), operate as a coordinated network, sharing technical skills and resources to enable innovative gene therapy research.

Stéphane Maikovsky, Deputy CEO and Chief Financial Officer at LifeArc, said: "Rare diseases affect approximately 3.5 million people in the UK, and approximately 70 percent of the conditions have a genetic cause. LifeArc is, therefore, excited about the potential for gene therapy to transform treatment pathways for people with rare diseases. We are proud to have launched the Innovation Hub network, including the latest hub in Sheffield, which will play a vital role in speeding up the development of new gene therapies and improving the lives of millions of patients."

Dr Louise Jones, Director of Investigator-Led Themes at the Medical Research Council (MRC) said: "MRC's longstanding and continued investment in gene therapies, and other advanced therapies that directly target and treat diseased cells and organs, underscores our commitment to ensuring advanced therapies research remains a significant UK strength. With the GTIMC and the network of Innovation Hubs for Gene Therapies, academics across the UK will be supported in the development, scale-up and delivery of new and improved genetic treatments to patients."

Notes

Wolfson Foundation:

The Wolfson Foundation is an independent charity with a focus on research and education. Its aim is to support civil society by investing in excellent projects in science, health, heritage, humanities and the arts.

Since it was established in 1955, some £1 billion (£2 billion in real terms) has been awarded to more than 14,000 projects throughout the UK, all on the basis of expert review.

Garfield Weston Foundation

Established in 1958, the Garfield Weston Foundation is a family-founded grant-maker that gives money to support a wide variety of charities across the UK. The Foundation's funding comes from an endowment of shares in the Weston family business - a successful model that still exists today. The Weston family have a consistent aim. The more successful the family businesses, the more money the Foundation can donate.

Each year the Foundation gives away its income and donations have continued to grow. Since it was established it has donated over £1.5 billion, of which over half has been given away in the past ten years. In the most recent financial year the Foundation gave away over £100 million to nearly 1,800 charities across the UK.