LUXTURNA (voretigene neparvovec-rzyl) is a groundbreaking gene therapy product designed for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, a rare genetic condition that leads to vision loss and may result in blindness. This innovative treatment works by delivering a normal copy of the RPE65 gene directly into retinal cells, enabling them to produce the necessary protein that converts light to an electrical signal in the retina to restore patient's vision or prevent further deterioration. Administered directly into the eye through a surgical procedure, LUXTURNA is the first gene therapy approved by the FDA for a genetic disease and represents a significant advancement in the field of genetic medicine, offering hope to patients with this previously untreatable condition.