LUXTURNA (voretigene neparvovec) is a novel, adeno-associated virus (AAV) vector-based gene therapy developed by Spark Therapeutics, Inc. It is intended for the treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy, a serious and sight-threatening condition with no approved pharmacologic treatments. This introduction provides a brief overview of the summaries contained within Module 2, which collectively demonstrate a favorable benefit-risk profile for LUXTURNA in the intended population.