FDA Approves First Gene Therapy for Genetic Disease

FDA approved Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl) for treating children and adults with the rare, inherited blindness disorder biallelic RPE65 mutation-associated retinal dystrophy. Approval of the one-time adeno-associated virus vector (AAV) delivered gene therapy, which FDA commissioner Scott Gottlieb, M.D., described in a statement as “a milestone that reinforces the potential of this breakthrough approach in treating a wide-range of challenging d