New gene therapy trial hopes to silence FSHD at its source
Disease control
Recruiting now
This early-stage trial tests a single-dose gene therapy called EPI-321 in 12 adults with FSHD type 1, a genetic muscle disease. The therapy aims to turn off the faulty gene that causes muscle damage. Researchers will monitor safety and look for early signs that the treatment is w…
Phase: PHASE1, PHASE2 • Sponsor: Epicrispr Biotechnologies, Inc. • Aim: Disease control
Last updated May 26, 2026 12:07 UTC