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.

Avidity Biosciences, Inc., a biopharmaceutical company, engages in the delivery of RNA therapeutics. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat diseases previously untreatable with RNA therapeutics. The company's lead product candidate AOC 1001 for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease that is in phase 1/2 clinical trial. Its other products in pipeline include AOC 1044 for the treatment of duchenne muscular dystrophy, which is under phase 1/2 clinical development trial; and AOC 1020 to treat facioscapulohumeral muscular dystrophy that is in phase 1/2 clinical trial. The company is developing products for rare skeletal muscle and rare cardiac diseases. Avidity Biosciences, Inc. was incorporated in 2012 and is headquartered in San Diego, California.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. It has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies; and Sanofi for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, 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.

Cabaletta Bio, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of engineered T cell therapies for patients with B cell-mediated autoimmune diseases. The company's lead product candidate is CABA-201, a fully human anti-CD19 binder for the treatment of Phase 1/2 clinical trials in dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, lupus nephritis, non-renal systemic lupus erythematosus, systemic sclerosis, and generalized myasthenia gravis. It also develops DSG3-CAART, which is in Phase I/II clinical trial for the treatment of mucosal pemphigus vulgaris; and MuSK-CAART, an investigational cell therapy that is in Phase I/II clinical trial for treating patients with anti- muscle-specific kinase antibody positive myasthenia gravis. It has a collaboration with the University of Pennsylvania and the Children's Hospital of Philadelphia; Nanjing IASO Biotherapeutics Co., Ltd; Oxford Biomedica; and WuXi Advanced Therapies, Inc. The company was formerly known as Tycho Therapeutics, Inc. and changed its name to Cabaletta Bio, Inc. in August 2018. Cabaletta Bio, Inc. was incorporated in 2017 and is headquartered in Philadelphia, Pennsylvania.

BioCardia, Inc., a clinical-stage regenerative medicine company, develops cellular and cell-derived therapeutics for cardiovascular and pulmonary diseases in the United States. Its lead product candidate is CardiAMP, an autologous mononuclear cell therapy system in Phase III clinical trial for the treatment of ischemic heart failure with reduced ejection fraction and refractory angina resulting from chronic myocardial ischemia. The company is also developing an allogeneic cells therapy platform, which is an investigational culture expanded bone marrow derived mesenchymal cell therapy in Phase I/II trial to treat ischemic heart failure and acute respiratory distress syndrome. In addition, it offers the Helix biotherapeutic delivery system for minimally invasive targeted delivery of biologic agents to the heart; and Morph deflectable guides and sheaths. The company has collaboration agreements with CellProthera in the development of ProtheraCytes, which is currently under Phase II trial for the treatment of acute myocardial infarction. BioCardia, Inc. is based in Sunnyvale, California.

Lineage Cell Therapeutics, Inc., a clinical-stage biotechnology company, develops novel cell therapies for unmet medical needs in the United States and internationally. The company develops OpRegen, an allogeneic retinal pigment epithelium cell replacement therapy, which is in Phase 2a clinical trial for the treatment of the dry age-related macular degeneration; OPC1, an allogeneic oligodendrocyte progenitor cell therapy that is in Phase 1/2a multicenter clinical trial for the treatment of cervical spinal cord injuries; and VAC, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells, which is in Phase I clinical trial to treat non-small cell lung cancer. It also offers ANP1, an allogeneic auditory neuron progenitor cell transplant, which is in preclinical development for the treatment of debilitating hearing loss; and PNC1, an allogeneic photoreceptor cell transplant, which is in preclinical development for the treatment of vision loss due to photoreceptor dysfunction or damage. In addition, the company engages in the research and development of therapeutic products for retinal diseases, neurological diseases, and disorders and oncology. Lineage Cell Therapeutics, Inc. has a collaboration with Immunomic Therapeutics, Inc., for the treatment of glioblastoma multiforme. The company was formerly known as BioTime, Inc. and changed its name to Lineage Cell Therapeutics, Inc. in August 2019. Lineage Cell Therapeutics, Inc. was incorporated in 1990 and is headquartered in Carlsbad, California.

Calidi Biotherapeutics, Inc., a clinical stage immuno-oncology company, engages in developing allogeneic stem cell-based platforms to potentiate and deliver oncolytic viruses to cancer patients in the United States. Its product pipeline comprising CLD-101, which is in phase 1b clinical trial for the treatment of high grade glioma; CLD-101, which is in phase 1 clinical trial for the treatment of recurrent high grade glioma; CLD-201, which is in preclinical trial for the treatment of advanced solid tumors, such as triple-negative breast cancer, metastatic/unresectable melanoma, head and neck squamous cell carcinoma, advanced soft tissue sarcoma, and advanced basal cell carcinoma; and CLD-400, which is in preclinical trial for the treatment of lung cancer and metastatic solid tumors. The company offers NeuroNova platform and SuperNova platform. The company is headquartered in San Diego, California.