Reversing Blindness with New FDA Approved Retinal Gene Therapy

In December 2017, the FDA approved the first gene therapy to treat a genetic disease causing blindness called Luxturna (voretigene neparvovec-rzyl). Initially developed by University of Pennsylvania and Children's Hospital of Philadelphia (CHOP) followed by Spark Therapeutics, Luxturna has been approved for the treatment of patients with inherited retinal disease caused by a mutation in both copies of the RPE65 gene. Mutations in this gene are responsible for early onset blindness from Leber congenital amaurosis, some forms of retinitis pigmentosa, and other conditions. A single injection in each eye has shown to be enough to improve the lost vision for at least 3 years. We are talking to the doctor couple, Dr. Jean Bennett and Dr. Albert M. Maguire, whose work for more than 25 years on congenital blindness led to this ground breaking FDA approval. Join us to learn about the novel therapy, details on treatment procedure and recovery.