Dyne Therapeutics

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).

Myotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.Show more

The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.Show more

The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD. Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) will have approximately 450 FSHD participants followed for a minimum of 3 years. A subset of MOVE FSHD participants, approximately 200, will participate in the MOVE+ sub-study which includes whole body MRI and reachable workspace, as well as optional muscle biopsy and wearable device (US participants only).Show more

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (208 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.Show more

The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.Show more

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (288 weeks).Show more

Dyne Therapeutics develops targeted muscle therapies using its FORCE platform, which links antisense oligonucleotides to an antibody fragment that binds transferrin receptor 1 (TfR1), a doorway into muscle and nerve cells. Its Duchenne muscular dystrophy drug zeleciment rostudirsen (z-rostudirsen, DYNE-251), an exon 51 skipping therapy given into a vein every 4 weeks, is under FDA review for accelerated approval. The FDA accepted the application with Priority Review in July 2026 and is due to decide by January 21, 2027. The confirmatory Phase 3 FORZETTO trial began in May 2026.

For myotonic dystrophy type 1, zeleciment basivarsen (z-basivarsen, DYNE-101) holds FDA Breakthrough Therapy and Fast Track designations. Enrollment of 71 people in the registrational cohort of the Phase 1/2 ACHIEVE trial finished in June 2026, with topline results planned for the first quarter of 2027, and dosing in the confirmatory Phase 3 HARMONIA trial began in July 2026. In July 2026 the FDA also cleared Dyne to start a Phase 1 trial of DYNE-302 for facioscapulohumeral muscular dystrophy (FSHD).

None as of November 2026. Dyne Therapeutics has no FDA-approved drug yet; the FDA is reviewing its most advanced program.

Most advanced: Zeleciment rostudirsen (under FDA review), Zeleciment basivarsen (Phase 3).

Where to watch: the FDA calendar lists any Dyne Therapeutics application once a decision date is public, and the trials list on this page shows what is recruiting now.