Luxturna (voretigene neparvovec)

Luxturna (voretigene neparvovec) is an innovative gene therapy designed to treat a rare form of inherited vision loss called Leber congenital amaurosis caused by mutations in the RPE65 gene. By delivering a healthy copy of this gene directly into the retina, Luxturna aims to restore the function of retinal cells, which can improve or stabilize vision in affected individuals. This therapy represents a significant advancement in treating genetic disorders, offering hope to patients with previously limited options. It is administered as a single injection into the eye and has shown promising results in clinical trials.