NHS England is set to announce on Friday an expansion of its commissioned gene therapy programme for inherited retinal dystrophies, building directly on the high-profile case of Saffie Sandford, the six-year-old Stevenage girl whose restored sight dominated UK health headlines on Thursday. The announcement is expected to outline a pathway for additional treatment centres beyond Great Ormond Street Hospital, where Saffie received her pioneering procedure.
The treatment, targeting mutations in the RPGR gene which causes X-linked retinitis pigmentosa — one of the most common inherited causes of childhood blindness — had previously been available only through a highly restricted NHS Highly Specialised Services route. Health officials indicated Thursday that the therapy's successful real-world outcomes had provided sufficient clinical evidence to trigger a formal review of commissioning criteria.
NHS England's National Specialised Commissioning team is expected to publish updated guidance indicating that eligible patients aged four and above with confirmed RPGR mutations will be assessed at designated centres, with Manchester Royal Eye Hospital and Moorfields Eye Hospital in London identified as likely additional sites. The move would bring the number of children who could access the treatment each year from a handful to potentially several dozen across England.
The developer of the therapy, MeiraGTx, whose partnership with Janssen Pharmaceuticals underpins the treatment's commercial pathway, is anticipated to issue a statement welcoming the commissioning expansion and confirming ongoing manufacturing scale-up at its London facility to meet anticipated increased demand. Shares in the company had already responded positively on Thursday to the media coverage surrounding the Sandford case.
Charities including Fight for Sight and Retina UK are expected to host joint media briefings on Friday urging the devolved health authorities in Scotland, Wales, and Northern Ireland to follow England's lead and adopt equivalent commissioning decisions without delay. Campaigners argue that children outside England face an inequitable postcode lottery for a treatment now proven to deliver transformative outcomes.