Dyne Therapeutics
Dyne Therapeutics works on 3 rare diseases tracked on Trial Friend, including Duchenne Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy, with 7 recruiting clinical trials.
Dyne Therapeutics develops targeted muscle therapies using its FORCE platform, which conjugates antisense oligonucleotides to anti-transferrin receptor 1 (TfR1) antibody fragments for direct muscle and CNS delivery. The lead program DYNE-101 (zeleciment basivarsen) is in Phase 1/2 (ACHIEVE trial) for myotonic dystrophy type 1, with FDA Breakthrough Therapy and Fast Track designations. DYNE-251 targets Duchenne muscular dystrophy using exon-skipping.
Focus areas at Dyne Therapeutics
As a rare disease specialist, Dyne Therapeutics has active clinical trial programs and drug development efforts across 3 rare diseases, including Duchenne Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Dyne Therapeutics, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Dyne Therapeutics is headquartered in Waltham, United States, founded in 2017, publicly traded under the ticker symbol DYN. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Dyne Therapeutics Drug Pipeline
Dyne Therapeutics has 7 active clinical trials across 3 development stages, with 7 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Dyne Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Dyne Therapeutics Clinical Trials (7)
Active and recruiting clinical trials sponsored by Dyne Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
FDA-Approved Drugs
Dyne Therapeutics Trial Locations
Dyne Therapeutics clinical trials are running at 126 sites in 16 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (3)
Diseases targeted by Dyne Therapeutics's clinical trial and drug development programs
Duchenne muscular dystrophy is an X-linked genetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood. The defective dystrophin protein normally protects muscl...
Facioscapulohumeral Muscular Dystrophy (FSHD) is an inherited muscular dystrophy characterized by progressive weakness of the facial, shoulder, and upper arm muscles. The condition results from abnorm...
Myotonic Dystrophy (MD) is the most common muscular dystrophy in adults, characterized by progressive muscle weakness and myotonia—the inability of muscles to relax after contraction. The condition re...
Patient Resources
Organizations and resources related to Dyne Therapeutics's rare disease focus areas
Frequently Asked Questions About Dyne Therapeutics
Common questions about Dyne Therapeutics's rare disease programs, clinical trials, and treatments.