Sarepta Therapeutics

Overview

Gene therapy company where Dan McGrath heads engineering and technical services, applying decades of tech transfer expertise to advanced modality manufacturing. Sarepta Therapeutics employs Dan McGrath as head of engineering and technical services, where he has applied more than three decades of tech transfer and infrastructure development experience to successfully bring advanced modality therapies to patients. (Segment 1 (00:00-05:00)) Sarepta Therapeutics developed eteplirsen, a naked PMO for DMD exon 51 skipping, one of the first ASO drugs approved around 2016, delivered by IV infusion without a targeting moiety. Sarepta blazed the trail for PMO-based exon skipping in DMD with eteplirsen, though its naked PMO approach may only modestly slow disease decline versus the deeper tissue distribution and functional improvement demonstrated by Dyne's FORCE-conjugated approach. (Segment 2 (05:00-10:00))