GlobeNewswire·Jul 28, 2026, 7:30 AM ET

Dyne Therapeutics Announces U.S. FDA Clearance of Investigational New Drug (IND) Application for DYNE-302 in Facioscapulohumeral Muscular Dystrophy (FSHD)

DYN

Dyne Therapeutics received FDA clearance for an IND application to initiate a Phase 1 clinical trial for DYNE-302 in facioscapulohumeral muscular dystrophy (FSHD).
DYNE-302 is the third program to enter clinical development utilizing Dyne's FORCE™ platform, following programs for DMD (DYNE-251) & DM1 (DYNE-101).
The investigational therapy consists of a TfR1-targeting Fab conjugated to an siRNA payload designed to suppress DUX4 expression & the downstream DUX4 transcriptome.
Preclinical models demonstrated robust DUX4 knockdown, significant reversal of muscle fiber damage, & functional improvement in a severe FSHD model.
The Phase 1 trial will be a randomized, placebo-controlled, double-blind, multiple ascending dose study in ambulatory adults with FSHD.
The primary endpoint is safety & tolerability, with secondary assessments of pharmacokinetics, pharmacodynamics, muscle DUX4 transcriptome changes, & plasma KHDC1L levels.
The first cohort includes 9 participants receiving three intravenous doses every four weeks, randomized 2:1 to receive either 1.5 mg/kg DYNE-302 or placebo.
Participants completing the placebo-controlled period may enter an open-label long-term extension to receive DYNE-302 for up to 96 additional weeks.
Dyne intends to pursue a traditional regulatory approval pathway in the U.S. for DYNE-302.
FSHD is a rare, progressive genetic disease affecting an estimated 15,000 to 40,000 individuals in the U.S. & 20,000 to 50,000 in Europe, with no currently approved therapies.