Princess Royal opens pioneering Gene Therapy Innovation and Manufacturing Centre

Her Royal Highness, The Princess Royal, officially opened the Gene Therapy Innovation and Manufacturing Centre (GTIMC) at the University of Sheffield on October 2, 2024. The centre is focused on advancing gene therapy research and providing new treatment options for patients with rare and inherited diseases.

The GTIMC brings together researchers, clinicians, and industry partners to facilitate the translation of gene therapy research into clinical trials. These therapies offer treatment potential for conditions such as motor neuron diseases, cystic fibrosis, and haemophilia, by replacing or manipulating faulty genes.

During the visit, The Princess Royal met with patients who stand to benefit from the centre’s work and praised its ability to transform healthcare.

Professor Mimoun Azzouz, Director of the GTIMC, highlighted the impact of gene therapies, “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.”

The University of Sheffield is internationally recognised for its research in neurodegenerative diseases. The university has a database of over 2,000 patients and a resource of human biosamples, positioning it as a leader in gene therapy research.

The launch event included testimony from Dharmisha Stezaly, whose son received gene therapy for Spinal Muscular Atrophy. Dharmisha said, “The gene therapy Zolgensma has changed our lives in an immeasurable way.”

The GTIMC facility aims to accelerate gene therapy development and build career pathways for scientists in the region. It has already created 27 highly skilled jobs, helping to position Sheffield and Yorkshire as future hubs for gene therapy.

Professor Ashley Blom, Vice President of the Faculty of Health at the University of Sheffield, added, “The GTIMC will unlock development pathways for new treatments for people affected by devastating genetic disorders.”

The centre is one of three Innovation Hubs for Gene Therapies in the UK, part of an £18 million network funded by LifeArc and the Medical Research Council (MRC). The hubs operate as a coordinated network, sharing skills and resources to support gene therapy research.

Stéphane Maikovsky, Deputy CEO at LifeArc, emphasised the significance of the network, “Rare diseases affect approximately 3.5 million people in the UK, and approximately 70 percent have a genetic cause. LifeArc is excited about the potential for gene therapy to transform treatment pathways.”

Dr Louise Jones, Director at the MRC, further supported the UK’s investment in gene therapy, stating, “The GTIMC and Innovation Hubs will support academics in developing new genetic treatments for patients.”