BioCentury
ARTICLE | Clinical News

Sarepta's DMD candidate leads to microdystrophin expression of 76.2% in Phase I/IIa

June 22, 2018 6:27 PM UTC

Sarepta Therapeutics Inc. (NASDAQ:SRPT) reported on June 19 preliminary data from a Phase I/IIa trial evaluating AAVrh74.MHCK7.micro-Dystrophin to treat Duchenne muscular dystrophy (DMD).

In three DMD patients in the open-label, U.S. trial, the microdystrophin gene therapy led to mean sarcolemma-localized transduced microdystrophin expression, as measured by the percentage of microdystrophin-positive fibers, of 76.2% and a mean fiber intensity of 74.5% compared with normal controls. In posttreatment biopsies, Sarepta reported mean microdystrophin levels as measured by Western blot of 38.2% of normal, or 53.7% of normal adjusted for fat and fibrotic tissue. The gene therapy also reduced mean serum creatinine kinase levels by over 87% at day 60...

BCIQ Target Profiles

Dystrophin