Experimental gene 'Silencer' therapy enters human testing for rare muscle disease
Disease control
Ongoing
This early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows a…
Phase: PHASE1, PHASE2 • Sponsor: Epicrispr Biotechnologies, Inc. • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC