Gene Therapy for Blindness Approved by FDA in Groundbreaking Moment for DNA-Based Medicine
The Food and Drug Administration approved a groundbreaking gene therapy to treat a rare form of blindness Wednesday. The drug, Luxturna, is intended to treat a genetic form of blindness called retinal blindness that affects about 1,000 to 2,000 people in the United States. The price of the treatment—widely speculated to be $1 million—will not be announced until early January, according to a spokesperson. "It really is a trailblazer for using gene delivery," Stephen Rose tells Newsweek. Rose is the chief scientific officer of the Foundation Fighting Blindness, which funded some of the preliminary research on the therapy. (The treatment, first developed at the Children's Hospital of Philadelphia, has been licensed to Spark Therapeutics.)
"Now there's a map that shows how you do it, what needs to be done, what the FDA and other regulatory groups are going to be looking for," he said. "It provides that proof of concept and proof of principle in humans that it's doable."
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