Sarepta leads off Duchenne MD gene therapy study with a ‘home run’ on efficacy — but can they deliver in 2 years?
Sarepta $SRPT has leaped out into the front of the race to develop a once-and-done gene therapy for Duchenne muscular dystrophy.
With their R&D day in full swing Tuesday, company execs and the senior researcher on their groundbreaking human gene therapy study posted a first look at the promising results seen in three young patients who received treatments in a small study with no control which will involve a total of 12 boys. The data are very early, but the immediate response seen in the first few months has inspired the company to believe that it is on the right track to get a new therapy to these patients in as little as two years — provided all the stars align in its favor.
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