News & Updates

Interim data demonstrated a favorable safety profile and early evidence of disease modification, including statistically significant increases in lean muscle volume and biomarker changes consistent with DUX4 suppression Additional clinical data expected to be presented at the World Muscle Society Annual Congress in September 2026 SAN FRANCISCO–(BUSINESS WIRE)–Epicrispr Biotechnologies, a clinical-stage company pioneering epigenetic therapies, …

Single-Dose EPI-321 Demonstrated a Positive Safety Profile and Increased Lean Muscle Volume Across the First Three Evaluable Patients at Six MonthsEPI-321 is the First Investigational Therapy Designed to Silence DUX4, the Genetic Driver of FSHD SAN FRANCISCO, CA – June 26, 2026 – Epicrispr Biotechnologies, a clinical-stage company pioneering epigenetic therapies, today announced new interim data …

GAITHERSBURG, Md.–(BUSINESS WIRE)–Mirecule, Inc., a leader in Antibody RNA Conjugate (ARC) therapeutics targeting rare neuromuscular diseases, today announced an expansion of its strategic collaboration with Sanofi. This collaboration focuses on delivering transformative treatments for patients with Facioscapulohumeral Muscular Dystrophy (FSHD). Building upon the companies’ successful partnership in developing ARC therapies, the expanded agreement will facilitate …

Company to present at J.P. Morgan Healthcare Conference on Tuesday, Jan 13, 2026 SAN FRANCISCO–(BUSINESS WIRE)–Epicrispr, a clinical-stage company pioneering gene-modulating therapies, today reported early clinical data from its ongoing first-in-human, open-label study evaluating EPI-321, an investigational epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD). Epicrispr holds the first and only open epigenetic editing Investigational New …

− First patient with facioscapulohumeral muscular dystrophy (FSHD) dosed with Restem-L umbilical lining modified progenitor cells (UMPCs) − The Phase 1/2a study will evaluate the safety and preliminary efficacy of Restem-L in patients with FSHD − Clinical study is funded by SOLVE FSHD MIAMI and VANCOUVER, British Columbia, Dec. 08, 2025 (GLOBE NEWSWIRE) — RESTEM …

Altay Therapeutics is pleased to announce that the FDA has granted Orphan Drug Designation to DX5057, the first and only oral small molecule DUX4 inhibitor in development for facioscapulohumeral muscular dystrophy (FSHD). FSHD affects ~40,000 people in the U.S. and leads to progressive muscle weakness, disability and loss of independence, yet no therapies are currently …

– EPI-321 is the first investigational therapy designed to silence DUX4 expression via epigenetic modulation – FDA has granted Fast Track, Rare Pediatric Disease, and Orphan Drug Designation for EPI-321 in FSHD SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Epicrispr Biotechnologies, a clinical-stage company pioneering gene-modulating therapies, today announced that the first patient has been dosed in its …

Cambridge, Mass., May 12, 2025 – Epicrispr Biotechnologies, a biotechnology company focused on developing curative therapies, today announced it has been named a Finalist Team in XPRIZE Healthspan FSHD Bonus Prize, earning a $250,000 Milestone Award for its work addressing facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy globally. Epicrispr was one of eight global …