Sarepta reports microdystrophin expression data for fourth patient in DMD gene therapy trial

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said AAVrh74.MHCK7.micro-Dystrophin led to mean sarcolemma-localized transduced microdystrophin expression, as measured by immunohistochemistry, of 81.2% and a mean fiber intensity of 96% compared with normal controls in four Duchenne muscular dystrophy (DMD) patients ages four to seven in a Phase I/IIa trial. Data were presented at the World Muscle Society meeting in Mendoza, Argentina.

The open-label, U.S. trial is designed to enroll six patients ages three months to three years and six patients ages four to seven years to receive 2x10¹⁴ vg/kg IV AAVrh74.MHCK7.micro-Dystrophin...