World first gene therapy treats Bardet-Biedl Syndrome (BBS)
An eleven-year-old girl from London has become the first NHS patient to receive a revolutionary new treatment for Bardet-Biedl Syndrome (BBS), a progressive form of severe blindness. The world-first gene therapy was developed and manufactured by MeiraGTx, a clinical-stage genetic medicines company, and was delivered by surgeons at St Helier Hospital in Surrey via keyhole surgery by injecting healthy copies of the gene into the patient’s eye. For more information, click here.
Bardet-Biedl Syndrome UK, the only registered charity supporting people with BBS, their families, and carers in the UK, has described the development as 'An important milestone for the Bardet-Biedl syndrome community', but warned that 'this treatment is specifically designed for retinal disease caused by mutations in the BBS10 gene. It does not treat the other features of Bardet-Biedl syndrome, nor is it suitable for retinal disease caused by other BBS genes'. You can their full statement, and find out more about the support provided by BBS UK on their website.
Posted on the 28th of July 2026