voretigene neparvovec

Voretigene neparvovec is a gene therapy used to treat a rare inherited eye disease called RPE65-related retinal dystrophy, which can lead to vision loss. It works by delivering a functional copy of the defective gene directly into retinal cells, helping them produce the protein necessary for normal vision. The therapy is administered as a one-time surgical procedure, aiming to improve or stabilize visual function. It offers hope for patients with this specific genetic condition, potentially restoring some sight or preventing further deterioration.