Gene therapy is becoming a promising solution for retinal degenerative diseases, as the retina offers an excellent setting for studying and treating eye conditions. Importantly, it was the first tissue to receive approved gene therapy for genetic disorders in the United States. To date, only one retinitis pigmentosa treatment regimen, LUXTURNA (developed by Spark Therapeutics), has been approved in the United States.
LUXTURNA, the only FDA-approved gene therapy for retinitis pigmentosa treatment, also known as voretigene neparvovec-rzyl, is a one-time gene therapy designed for patients with biallelic RPE65 mutation-associated retinal dystrophy. It received ap...
