Ex­pand­ing its pre­clin­i­cal at­tack on Duchenne MD, Sarep­ta grabs a li­cense for Duke's CRISPR tech

Sarep­ta $SRPT an­nounced this morn­ing that it’s team­ing up with Duke Uni­ver­si­ty to ex­plore new CRISPR-based meth­ods in the hunt for an ef­fec­tive Duchenne mus­cu­lar dy­s­tro­phy ther­a­py.

The com­pa­ny says it’s specif­i­cal­ly in­ter­est­ed in DMD, the rare and lethal con­di­tion it aimed to treat with its drug Ex­ondys51. But re­mem­ber, Sarep­ta got a con­tro­ver­sial reg­u­la­to­ry ap­proval for Ex­ondys51 based on da­ta crit­ics say nev­er met a prop­er thresh­old for ef­fi­ca­cy, and which they ar­gue led the FDA to an er­ro­neous con­clu­sion.

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