Entrada Therapeutics, Inc.

Dyne Therapeutics, Inc. is a U.S.-based biotechnology firm focused on developing advanced treatments for genetically driven muscle disorders. Utilizing its proprietary FORCE platform, which is engineered to deliver disease-modifying therapeutics, the company is actively engaged in various programs. These initiatives target conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral dystrophy, in addition to other rare skeletal, cardiac, and metabolic muscle diseases. The company was founded in 2017 and maintains its corporate headquarters in Waltham, Massachusetts.

PepGen Inc.PEPG · BiotechBoston, United States

71% matchPublic

PepGen Inc., a clinical-stage biotechnology company, develops oligonucleotide therapeutics for the treatment of severe neuromuscular and neurologic diseases in the United States. The company engages in the development of PGN-EDODM1, an EDO peptide-conjugated PMO, which is in Phase 2 clinical trial for the treatment of myotonic dystrophy type 1; and FREEDOM2-DM1, which is in Phase 2 for the treatment of myotonic dystrophy type 1. It is also involved in FREEDOM-OLE, an open label expansion study. The company was founded in 2018 and is based in Boston, Massachusetts.

Solid Biosciences Inc.SLDB · BiotechCharlestown, United States

70% matchPublic

Solid Biosciences Inc. is an American biotechnology firm dedicated to creating therapeutic solutions for Duchenne Muscular Dystrophy (DMD). Its most advanced experimental drug, SGT-001, is a gene transfer treatment currently in Phase I/II clinical trials, designed to encourage the production of functional dystrophin protein in patients' muscles. The company is also progressing with SGT-003, which represents a next-generation gene transfer therapy intended for DMD. Beyond specific drug candidates, Solid Biosciences is actively developing innovative platform technologies, such as "dual gene expression," a technique that allows for multiple therapeutic genes to be bundled into a single viral vector, and researching new capsid designs. The company maintains a strategic alliance through a collaboration and license agreement with Ultragenyx Pharmaceutical Inc., focusing on the joint development and market introduction of novel gene therapies for Duchenne Muscular Dystrophy. Founded in 2013, Solid Biosciences Inc. conducts its operations from its headquarters in Cambridge, Massachusetts.

Edgewise Therapeutics, Inc. is a biopharmaceutical firm dedicated to developing small-molecule treatments for a range of musculoskeletal disorders. Its leading investigational drug, EDG-5506, is an orally administered compound specifically engineered to tackle the fundamental genetic causes of dystrophinopathies, including Duchenne and Becker muscular dystrophy. This candidate has successfully concluded its Phase 1 clinical trial. Beyond EDG-5506, Edgewise maintains a pipeline of precision medicine candidates designed to modulate key muscle proteins, thereby addressing various genetically defined muscle conditions. Established in 2017, the company is headquartered in Boulder, Colorado.

Dynacure is a biotechnology company developing antisense oligonucleotide therapies for rare neuromuscular diseases. Its lead program, DYN101, targets DNM2-associated centronuclear myopathy for patients with this serious inherited muscle disorder.

Design Therapeutics, Inc. is a biopharmaceutical company in the preclinical phase that focuses on creating treatments for genetic conditions stemming from nucleotide repeat expansions. Its current pipeline targets Friedreich's Ataxia (FA), a severe, progressive, monogenic, and autosomal recessive disease. FA primarily impairs mitochondrial function across various organ systems, resulting in neurological, cardiac, and metabolic complications. Another significant area of development is Myotonic Dystrophy Type-1 (DM1). This is a progressive, dominantly inherited monogenic neuromuscular illness that impacts skeletal muscle, the heart, brain, and other bodily systems. Beyond these, the company is advancing its GeneTAC product candidate platform to address an array of other monogenic conditions caused by nucleotide repeat expansions. These include Fragile X syndrome, various spinocerebellar ataxias, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Huntington's disease, and spinobulbar muscular atrophy. Established in 2017, Design Therapeutics, Inc. maintains its headquarters in Carlsbad, California.