FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss

FDA approved a new gene therapy to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna (voretigene neparvovec-rzyl) is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene. Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.

Source: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589467.htm

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