91Ö±²¥

Case study: Gene Therapy Innovation and Manufacturing Centre (GTIMC)

Gene therapy is a promising treatment option for more than 7,000 rare diseases that currently have no cure - and 91Ö±²¥â€™s GTIMC innovation hub is dramatically broadening the scope of gene therapy research in the UK.

Pioneering facilities right here in South Yorkshire

Featuring state-of-the-art laboratories and equipment, an extensive clinical database and a large resource of human biosamples, the GTIMC has earned a reputation as a leading world player in gene therapy research and innovation.

The Centre brings together NHS, industry and academic partners from the North, the Midlands and Wales. Their pioneering collaborations have proven effective in translating scientific breakthroughs into new and exciting treatments for patients, and delivering them to the people who need them faster than ever before.

91Ö±²¥ has emerged as a leading player in gene therapy, and the future is bright. GTIMC will allow us to keep pace with translational discoveries for new and potentially life-changing treatments.

Professor Mimoun Azzouz

Chair of Translational Neuroscience

Changing the lives of children with devastating disease

Cutting-edge clinical trials at the GTIMC have shown some incredible potential. They've led to the development of a life-changing treatment - Zolgensma - for children with spinal muscular atrophy (SMA). 

This cruel disease is one of the most common genetic causes of infant death. Yet now, children who took part in the trial are achieving incredible developmental milestones, where they previously would have declined. Children like Sebastian.

I'm insanely proud of 91Ö±²¥. Gene therapy has changed our lives, it's revolutionary. You never know who'll be affected by a rare disease - my son having SMA doesn't just affect him, but all those connected to us. Knowing that Sebastian will not deteriorate and regress is such peace in our hearts and our minds. It's saved his life, and more.

When you donate, you're not only helping to fund research into disease-altering medicine, and you're not only helping the individual with the rare disease. You're changing the lives of an entire network of people who'll be forever impacted by that diagnosis. It's so much more than 'just' a donation.

Dharmisha Stezaly

91Ö±²¥ graduate and mum to Sebastian, who received a treatment of Zolgensma for his SMA

You can support this regional hub of innovation

Pioneering research at the GTIMC is offering new hope to people living with a wide range of devastating inherited conditions all around the world. Recent advances have brought real optimism for the discovery of a permanent cure in our own backyard.

With your support, 91Ö±²¥ will continue to lead the way in moving bold new treatments forward for the patients and families that so desperately need them.