RTTNews | May 18, 2021 09:29AM EDT

09:29 Tuesday, May 18, 2021 (RTTNews.com) - 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.

Read the original article on RTTNews ( https://www.rttnews.com/3195747/sarepta-s-gene-therapy-for-treatment-of-duchenne-muscular-dystrophy-shows-robust-expression.aspx)

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