Sarepta gene therapy shows positive effect in rare type of muscular dystrophy

Sarepta Therapeutics (NASDAQ:SRPT) announces positive results from a Phase 1/2 clinical trial evaluating gene therapy SRP-9003 in six patients with limb-girdle muscular dystrophy type 2E (LGMD2E), also called beta-sarcoglycanopathy, a rare type of muscular dystrophy characterized by weakness in the pelvic and shoulder girdle.

A strong dose-dependent increase in transduction and expression was observed in the high-dose cohort (n=3) at day 60 compared to the low-dose cohort (n=3), showing a mean expression of 72.3% of transduced beta-sarcoglycan (beta-SG) (the absence of this protein causes progressive muscle degeneration and shortened life expectancy in LGMD2E sufferers) properly localized to the muscle sarcolemma (tubular sheath that envelops skeletal muscle fibers).

SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for full-length beta-SG.

Development is ongoing.

https://seekingalpha.com/news/3581103-sarepta-gene-therapy-shows-positive-effect-in-rare-type-of-muscular-dystrophy