Sarepta's Gene Therapy For Treatment Of Duchenne Muscular Dystrophy Shows Robust Expression

(RTTNews) - Sarepta Therapeutics Inc. (SRPT) said that its investigational gene therapy for the treatment of duchenne muscular dystrophy, SRP-9001, demonstrated robust expression and consistent safety profile using the company's commercial process material.

The results from the first 11 participants enrolled in study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus.

The company noted that the treated patients achieved mean micro-dystrophin expression levels of 55.4% of normal as measured by western blot.

Micro-dystrophin was properly localized to the muscle sarcolemma, with patients achieving mean percentage of dystrophin positive fibers of 70.5% and intensity of micro-dystrophin expression of 116.9% of normal control, as measured by immunofluorescence.

The company noted that the safety profile was consistent with prior studies and there was no new safety signals identified.