Dyne Therapeutics Receives FDA Clearance to Begin Phase 1 Trial of DYNE-302 for FSHD
Wednesday, July 29, 2026
Dyne Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to begin a Phase 1 clinical trial of DYNE-302 for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
DYNE-302 is an investigational therapy developed using the company's FORCE™ platform, which is also used in its clinical programmes for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).
FSHD is a rare inherited muscle disease with no approved treatments. The disease is caused by abnormal activation of the DUX4 gene in skeletal muscle, leading to progressive muscle damage, reduced physical function and significant impact on patients' daily lives. DYNE-302 is designed to deliver a small interfering RNA (siRNA) to skeletal muscle using a transferrin receptor 1 (TfR1)-targeting antibody fragment. The therapy aims to reduce DUX4 expression and limit disease progression.
Preclinical studies showed that DYNE-302 achieved strong suppression of the DUX4 transcriptome in skeletal muscle, reduced muscle fibre damage and improved muscle function in severe disease models. The findings suggest the therapy may have the potential to reverse existing muscle damage by targeting DUX4 messenger RNA.
The Phase 1 study will be a randomised, placebo-controlled, double-blind, multiple ascending dose trial involving ambulatory adults with FSHD. The primary objective will be to assess the safety and tolerability of DYNE-302. The study will also evaluate pharmacokinetics, pharmacodynamics, changes in the DUX4 transcriptome in muscle tissue and plasma KHDC1L levels, a biomarker identified by the company for FSHD.
The first study cohort will include nine participants, who will receive three intravenous doses administered every four weeks. Participants will be randomised in a 2:1 ratio to receive either DYNE-302 at a dose of 1.5 mg/kg or placebo. Subject to the results from the initial cohort, the company plans to investigate higher doses and less frequent dosing schedules.
Participants completing the placebo-controlled phase may continue into an open-label extension study and receive DYNE-302 treatment for up to an additional 96 weeks.
Dyne Therapeutics plans to seek traditional regulatory approval in the United States if the clinical programme is successful. DYNE-302 remains an investigational therapy and has not yet been approved for use.
Source: globenewswire.com
