FDA Approves First Gene Therapy for Inherited 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 challe