FDA slaps a hold on Sarep­ta's Duchenne MD study in the wake of stel­lar ear­ly re­sults

Just days af­ter Sarep­ta $SRPT man­aged to whip up in­tense ex­cite­ment over ear­ly-stage da­ta from its first hu­man study of their gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy, the biotech re­port­ed that the FDA has put the pro­gram on hold.

The com­pa­ny was in­stant­ly ready to of­fer a de­fense for the move, one day af­ter the of­fi­cial no­tice.

The hold was trig­gered by the “pres­ence of a trace amount of DNA frag­ment in re­search-grade third-par­ty sup­plied plas­mid,” Sarep­ta re­port­ed. “Pre­lim­i­nary in-vi­vo test­ing per­formed by the Re­search In­sti­tute in­di­cates that the trace frag­ment does not re­sult in pro­tein ex­pres­sion and is quick­ly cleared.”

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