In an important milestone, voretigen neparvovec (Luxturna®, Novartis) was officially approved by the Food and Drug Administration (FDA) in the US in 2017 and by the European Medicines Agency (EMA) in Europe in 2018 for the treatment of IRDs caused by a biallelic disease-causing variants in RPE65 [31,32]. The gene discussed is RPE65; the disease is respiratory distress syndrome in premature infants.