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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