The mother of a six-year-old girl who had life-changing eye gene therapy says it is "like someone waved a magic wand and restored her sight in the dark". Saffie Sandford, from Stevenage, has the rare inherited condition Leber's Congenital Amaurosis (LCA) which prevents cells in the eye from making a specific protein needed for normal vision.

Providers and payers certainly face some formidable challenges when it comes to gene therapy, but it pales in comparison to what patients go through. “It is a huge commitment to go down the path of gene therapy,

This White House deal will see Regeneron offer all its new medicines at “MFN prices”, while providing Otarmeni for free to eligible patients.

Molecular biologist Jean Bennett and ophthalmologist Albert Maguire were awarded the $3 million Breakthrough Prize in Life Sciences alongside physician Katherine High for developi

By Dennis Thompson HealthDay ReporterTHURSDAY, April 23, 2026 (HealthDay News) — A new gene therapy to treat inherited deafness produces a lasting cure, a new international study has found. The treatment,

Ocugen's gene-agnostic therapy strategy targets broad retinal patient groups with one-time treatments, setting up major 2026-27 catalyst events.