October 17, 2017 Source: cnbc 276
Spark Therapeutics Inc's experimental gene therapy for a rare form of blindness improves vision and should be approved, advisers to the Food and Drug Administration concluded on Thursday, paving the way for the first U.S. gene therapy for an inherited disease.
The panel voted unanimously in favor of the treatment, Luxturna, which is designed to treat inherited retinal diseases caused by defects in a gene known as RPE65, which tells cells to produce an enzyme critical to normal vision.
The FDA is not obliged to follow the recommendations of its advisers but typically does. (Reporting by Toni Clarke in Washington; Editing by Steve Orlofsky)
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