Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene; and ELEVIDYS, an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with duchenne with a confirmed mutation in the duchenne gene. The company is also developing SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration and license agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Genevant Sciences; University of Florida; Dyno Therapeutics; Hansa Biopharma; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.

Denali Therapeutics Inc., a biopharmaceutical company, develops a portfolio of product candidates engineered to cross the blood-brain barrier for neurodegenerative diseases and lysosomal storage diseases in the United States. The company's transport vehicle (TV)-enabled programs include DNL310 ETV, an IDS enzyme replacement therapy program for MPS II; TAK-594/DNL593 which is in Phase 1/II for frontotemporal dementia-granulin; DNL126 program for MPS IIIA; and DNL622 for MPS I, as well as other preclinical programs that target amyloid beta and HER2. Its brain-penetrant small molecule programs comprise BIIB122/DNL151 LRRK2 inhibitor program for Parkinson's disease; SAR443820/DNL788 RIPK1 inhibitor program for CNS disease; DNL343 eIF2B Activator program for amyotrophic lateral sclerosis; and SAR443122/DNL758 RIPK1 inhibitor program for peripheral inflammatory diseases. It also provides early stage program include TAK-594/DNL593 program for FTD-GRN; DNL126 program for MPS IIIA, a Sanfilippo Syndrome A; DNL622 for MPS I which is Hurler syndrome; Antibody Transport Vehicle Amyloid beta program; Oligonucleotide Transport Vehicle platform, a novel class of biotherapeutics to address the root cause of diseases through modulation of gene expression; and other TV-enabled discovery programs. The company has collaboration agreements with Biogen MA Inc. and Biogen International GmbH; Genzyme Corporation; Takeda Pharmaceutical Company Limited; F-star Gamma Limited, F-star Biotechnologische Forschungs-und Entwicklungsges m.b.H, and F-star Biotechnology Limited; and Genentech, Inc. The company was formerly known as SPR Pharma Inc. and changed its name to Denali Therapeutics Inc. in March 2015. Denali Therapeutics Inc. was incorporated in 2013 and is headquartered in South San Francisco, California.

Edgewise Therapeutics, Inc., a biopharmaceutical company, discovers, develops, and commercializes therapies for the treatment of muscle disorders. Its lead product candidate, EDG-5506, an orally administered small molecule that is in Phase II clinical trials, designed to address the root cause of dystrophinopathies including Duchenne muscular dystrophy and Becker muscular dystrophy. The company develops EDG-7500, a small molecule for the treatment of hypertrophic cardiomyopathy and other severe cardiac disorders that is in Phase I clinical trials. In addition, it develops a pipeline of precision medicine product candidates that target key muscle proteins and modulators to address genetically defined muscle disorders. Edgewise Therapeutics, Inc. was incorporated in 2017 and is headquartered in Boulder, Colorado.

Dyne Therapeutics, Inc., a clinical-stage muscle disease company, operates as a biotechnology company that focuses on advancing therapeutics for genetically driven muscle diseases in the United States. It is developing a portfolio of muscle disease therapeutics, including programs in myotonic dystrophy type 1; duchenne muscular dystrophy; and facioscapulohumeral dystrophy, as well as rare skeletal muscle, and cardiac and metabolic muscle diseases using its FORCE platform that delivers disease-modifying therapeutics. Dyne Therapeutics, Inc. was incorporated in 2017 and is headquartered in Waltham, Massachusetts.

Voyager Therapeutics, Inc., a biotechnology company, focuses on the treatment of gene therapy and neurology diseases. The company's lead clinical candidate is VY-TAU01, an anti-tau antibody program for the treatment of alzheimer's disease. Its product pipeline includes superoxide dismutase 1 silencing gene therapy, which is in preclinical trial for the treatment of amyotrophic lateral sclerosis; tau silencing gene therapy, which is in preclinical trial for the treatment of alzheimer's disease; and vectorized anti-amyloid antibody, a gene therapy targeting anti-amyloid for the treatment of alzheimer's disease and is in preclinical trial. In addition, the company develops VY-FXN01, which is in preclinical trial to treat friedreich's ataxia; and GBA1 gene replacement to treat parkinson's disease and is in preclinical trial. Further, it provides research program for the treatment of Huntington's disease. The company has collaboration and license agreements with Alexion; AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc; and Sangamo Therapeutics, Inc. Voyager Therapeutics, Inc. was incorporated in 2013 and is headquartered in Lexington, Massachusetts.

Solid Biosciences Inc., a life science company, develops therapies for neuromuscular and cardiac diseases in the United States. The company's lead product candidate is SGT-003, a gene transfer candidate for the treatment of Duchenne muscular dystrophy; and SGT-501 to treat Catecholaminergic polymorphic ventricular tachycardia. It is also developing AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy; SGT-601 to treat a rare cardiac disease characterized by mutations in the gene that codes for cardiac troponin T protein, which helps coordinate contraction of the heart muscle; SGT-701 for treatment of rare inherited disease characterized by mutations in the RBM20 gene, a cardiac splicing factor that regulates alternative splicing, and codes for RNA binding motif protein 20; and AVB-202-TT to treat Friedreich's ataxia, as well as other drugs for the treatment of cardiac and other diseases. In addition, the company is involved in developing platform technologies, including capsid libraries and dual gene expression, a technology for packaging multiple transgenes into one capsid. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne; and a license agreement with the University of Washington, University of Missouri, and University of Florida. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.

PepGen Inc., a clinical-stage biotechnology company, focuses on the development of oligonucleotide therapeutics for use in the treatment of severe neuromuscular and neurologic diseases. Its lead product candidate is PGN-EDO51, an EDO peptide that is in Phase 2 clinical trial to treat Duchenne muscular dystrophy (DMD) patients. The company is also developing PGN-EDODM1, an EDO peptide-conjugated PMO, which is in Phase 1 clinical trial for the treatment of myotonic dystrophy type 1, as well as EDO therapeutic candidates, such as PGN-EDO53, PGN-EDO45, and PGN-EDO44 for the treatment of DMD. PepGen Inc. was founded in 2018 and is based in Boston, Massachusetts.

Entero Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of targeted and orally delivered therapies for the treatment of patients with gastrointestinal diseases. The company's pipeline candidates include latiglutenase, a targeted oral biotherapeutic for celiac disease designed to breakdown gluten into non-immunogenic peptides, currently under Phase 2 studies; and capeserod, a selective 5-HT4 receptor partial agonist under Phase 1 studies for the treatment of gastroparesis. It is also involved in the development of adrulipase, a recombinant lipase enzyme designed to enable the digestion of fats and other nutrients, currently under Phase 2 studies for the treatment of exocrine pancreatic insufficiency in patients with cystic fibrosis and chronic pancreatitis; and niclosamide, an oral small molecule with anti-inflammatory inhibitor therapy, currently under Phase 2 studies for the treatment of ulcerative proctitis and ulcerative proctosigmoiditis, as well as immune checkpoint inhibitor-associated colitis and diarrhea in metastatic cancer patients. The company was formerly known as First Wave BioPharma, Inc. and changed its name to Entero Therapeutics, Inc. in May 2024. Entero Therapeutics, Inc. was incorporated in 2014 and is based in Boca Raton, Florida.