Gene Therapy Researchers Awarded Breakthrough Prize for Inherited Blindness Treatment
April 22, 2026
Three researchers from Penn Medicine and Children’s Hospital of Philadelphia received the
2026 Breakthrough Prize in Life Sciences for developing the first FDA-approved gene therapy for an inherited condition. The therapy,
voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics), improves sight in people with Leber congenital amaurosis (LCA), a rare form of retinal blindness caused by mutations in the
RPE65 gene. According to Penn Medicine, their contributions helped catalyze more than 140 gene therapy clinical trials targeting retinal diseases.
