RPE65 and respiratory distress syndrome in premature infants: Among these clinical trials, voretigene neparvovec (Luxturna) that targets RPE65-associated LCA was the first FDA-approved gene therapy drug, while other clinical trials of IRDs, including Retinitis Pigmentosa (RP), Choroideremia and X-linked Retinitis Pigmentosa (XLRP), are also under way.