Breakthrough gene therapy transforms teenager's life

A major milestone in gene therapy has been reached at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (UCL GOSH ICH), where the first-ever clinical trial for patients with p47 Chronic Granulomatous Disease (CGD) is underway.

The rare genetic immunodeficiency affects about one in a million people and leaves patients vulnerable to severe infection and inflammation. Until now, the only treatment option was a bone marrow transplant – a route often limited by the difficulty of finding a suitable donor.

This new therapy has already transformed the life of Remi, the first patient treated, who is now preparing to begin university following a year of restored health and independence.

The trial also marks a bench-to-bedside first: it is the first gene therapy to be researched, manufactured, and delivered entirely under one roof at the Zayed Centre for Research into Rare Diseases in Children.

At AlbionVC, we are proud to back innovation that translates cutting-edge science into life-changing treatments – UCL Technology Fund, managed by us in collaboration with UCL Business, has funded the development of the viral vector, its preclinical testing, and the ongoing clinical trial. This achievement demonstrates the importance of long-term investment in translational research – helping ensure that life-changing therapies developed in the lab can reach patients who need them most.

The Times has more here.

Full press release here.