In a groundbreaking world-first, the Zayed Centre for Research into Rare Disease in Children at Great Ormond Street Hospital (GOSH), London, has successfully delivered a pioneering genetic treatment for chronic granulomatous disease (CGD).
CGD is a rare immune disorder where white blood cells fail to function properly, leaving patients highly vulnerable to bacterial and fungal infections. This severely impacts both health and quality of life.
Now, thanks to this novel therapy—developed, manufactured, and delivered entirely at GOSH—a 19-year-old patient has received long-term immune stability, offering the prospect of a healthier life.
The treatment forms part of a world-first clinical trial for CGD, with plans to extend it to four additional patients.
The Zayed Centre for Research, a collaboration between GOSH, University College London, and GOSH Charity, was established through a £60 million gift from Sheikha Fatima bint Mubarak.