Scientists test One-Shot gene therapy to silence root cause of Muscle-Wasting disease
Disease control
Recruiting now
This early-stage study is testing a new, one-time gene therapy called EPI-321 in adults with FSHD, a genetic muscle-wasting disease. The main goal is to find out if the treatment is safe and what dose is best, while also looking for early signs that it might be working. About 12 …
Phase: PHASE1, PHASE2 • Sponsor: Epicrispr Biotechnologies, Inc. • Aim: Disease control
Last updated Mar 10, 2026 12:53 UTC