Gene Therapy for Blindness Appears Initially Effective, Says U.S. FDA
Published:19 Oct.2017    Source:Scientific American
Spark Therapeutics Inc’s experimental gene therapy for a rare inherited form of blindness is effective, according to a preliminary review by the U.S. Food and Drug Administration. The therapy, Luxturna, or voretigene neparvovec, would be the first-ever gene therapy for any inherited disease to be approved in the United States.

The FDA noted there is no available long-term data to show whether the effectiveness of the therapy is maintained after a year, so the clinical benefit beyond that “is unclear.”