Dyne Therapeutics, Inc.

The company is a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. Its proprietary FORCE platform is designed to leverage the transferrin receptor 1, or TfR1, to deliver targeted therapeutics to muscle tissue and the central nervous system, or CNS. The FORCE platform utilizes an antigen-binding fragment antibody, or Fab, targeting TfR1 conjugated to a payload designed to target the genetic basis of the disease. The FORCE platform allows for the deployment of different classes of payloads, such as oligonucleotides and enzymes, with specific mechanisms of action. The company currently focuses on neuromuscular diseases with high unmet need, with etiologic targets and clear translational potential. Using its FORCE platform, the company is assembling a broad portfolio of product candidates, including those being developed for Duchenne muscular dystrophy, or DMD, myotonic dystrophy type 1, or DM1, facioscapulohumeral dystrophy, or FSHD, and Pompe disease. The company plans to expand its portfolio through development efforts focused on diseases involving the CNS, rare skeletal muscle diseases, and cardiac and metabolic muscle diseases. Product candidates have been identified for DMD (amenable to skipping exons 51, 53, 45, 44, and 55), DM1, FSHD, and Pompe programs, which are in varying stages of preclinical and clinical development. The product candidate zeleciment rostudirsen, or z-rostudirsen (also known as DYNE-251), is currently being evaluated in the DELIVER trial, a global Phase 1/2 clinical trial in patients with DMD amenable to exon 51 skipping. The company plans to submit a biologics license application, or BLA, to the U.S. Food and Drug Administration, or FDA, for U.S. Accelerated Approval of z-rostudirsen for DMD patients who have mutations amenable to exon 51 skipping in the second quarter of 2026 based on dystrophin as a surrogate endpoint.