The FDA has recently approved Luxturna, a new gene therapy developed by Spark Therapeutics, to treat an inherited form of vision loss that can result in blindness. The most prevalent inherited retinal dystrophies are retinitis pigmentosa (RP), Leber congenital amaurosis (LCA) and Stargardt disease (SD), all of which have in common a mutation of the RPE65 gene. Patients with Leber congenital amaurosis will be the first beneficiaries of retinal gene therapy, with RP and SD to follow after further study.
“I believe gene therapy will become a mainstay in treating,and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, M.D.
More Information: LUXTURNA Website