Product Development

Possible factors behind Sarepta’s DMD miss

And what it means, if anything, for its next gene therapies

An assessment of the factor behind Sarepta’s DMD miss and what the result means, if anything, for the company’s next gene therapies.

Sarepta believes the functional endpoint miss in a Phase II trial of its Duchenne muscular dystrophy gene therapy candidate is due to a randomization issue, but investors may be reading the negative result through to its broader gene therapy platform. 

Sarepta Therapeutics Inc. (NASDAQ:SRPT) fell 50% to $84.45 in after-hours trading on