BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company, discovers, creates, tests, and delivers transformative medicines to treat patients who suffer from genetic diseases and cancers. Its products in development programs include AG10, a next-generation oral small molecule near-complete TTR stabilizer that is in Phase 3 clinical trial for the treatment of TTR amyloidosis, or transthyretin amyloid cardiomyopathy (ATTR-CM); low-dose infigratinib, an oral FGFR1-3 selective tyrosine kinase inhibitor, which is in Phase 3 double-blinded, placebo-controlled pivotal study for the treatment option for children with achondroplasia; and BBP-631, an AAV5 gene transfer product candidate that is in Phase 1/2 clinical trial for the treatment of congenital adrenal hyperplasia, or CAH, driven by 21-hydroxylase deficiency, or 21OHD. The company also develops Encaleret, a small molecule antagonist of the calcium sensing receptor, or CaSR, which is in phase 3 clinical trial for treating autosomal dominant hypocalcemia type 1, or ADH1; and BBP-418, a glycosylation substrate pro-drug that is in Phase 3 clinical trial for treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). In addition, it engages in developing products for mendelian, oncology, and gene therapy diseases. BridgeBio Pharma, Inc. has license and collaboration agreements with the Leland Stanford Junior University; and Leidos Biomedical Research, Inc. The company was founded in 2015 and is headquartered in Palo Alto, California.

Cytokinetics, Incorporated, a late-stage biopharmaceutical company, focuses on discovering, developing, and commercializing muscle activators and inhibitors as potential treatments for debilitating diseases. It develops small molecule drug candidates primarily engineered to impact muscle function and contractility. The company's drug candidates include omecamtiv mecarbil, a novel cardiac myosin activator that is in Phase III clinical trial in patients with heart failure. It also develops CK-136, a novel cardiac troponin activator that is in Phase I clinical trial; CK-586, a small molecule cardiac myosin inhibitor, that is in Phase I clinical trial; and aficamten, a novel cardiac myosin inhibitor, which is in Phase III clinical trial for the treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy. The company has a strategic alliance with Ji Xing Pharmaceuticals Limited; and a collaboration and license agreement for the development and commercialization of aficamten in Japan for the treatment of patients with obstructive and non-obstructive hypertrophic cardiomyopathy. Cytokinetics, Incorporated was incorporated in 1997 and is headquartered in South San Francisco, California.

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.

Capricor Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the development of transformative cell and exosome-based therapeutics for the treatment of duchenne muscular dystrophy (DMD) and other diseases with unmet medical needs. The company's lead candidate, CAP-1002, an allogeneic cardiac-derived cell therapy, which has completed phase 3 clinical trial for the treatment of patients with late-stage Duchenne muscular dystrophy (DMD). It also focused on developing StealthX, an engineered exosome-based vaccine candidate, under phase 1 clinical study for a range of therapeutic applications, including targeted RNA, protein, and small molecule therapeutics to treat or prevent a variety of diseases. In addition, the company develops CAP-2003, under pre-clinical development for the treatment of trauma related injuries and conditions. It has collaboration and distribution agreement with Nippon Shinyaku Co. Ltd., collaboration agreement with National Institute of Allergy and Infectious Diseases, license agreements with the Johns Hopkins University and Cedars-Sinai Medical Center, as well as cell line license agreement with Life Technologies Corporation. The company was founded in 2005 and is headquartered in San Diego, California.

Benitec Biopharma Inc., a clinical-stage biotechnology company, focuses on the development of novel genetic medicines. The company develops DNA-directed RNA interference-based therapeutics for chronic and life-threatening human conditions. It is developing BB-301, an adeno-associated virus based gene therapy agent for treating oculopharyngeal muscular dystrophy. Benitec Biopharma Inc. was incorporated in 1995 and is headquartered in Hayward, California.

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.

Lexeo Therapeutics, Inc. operates as a clinical stage genetic medicine company that focuses on hereditary and acquired diseases. The company develops LX2006, which is an AAVrh10-based gene therapy candidate for the treatment of Friedreich's ataxia (FA) cardiomyopathy; LX2020, an AAVrh10-based gene therapy candidate for the treatment of plakophilin-2 arrhythmogenic cardiomyopathy; LX2021, a gene therapy candidate for the treatment of DSP cardiomyopathy associated with it; and LX2022, a gene therapy candidate for the treatment of hypertrophic cardiomyopathy, or HCM caused by TNNI3 gene. It also develops LX1001, an AAVrh10-based gene therapy candidate for the treatment of APOE4 homozygous; LX1020, a gene therapy candidate for the treatment of APOE4 homozygous; LX1021 for the treatment of APOE4 homozygotes; and LX1004 for the treatment of CLN2 Batten disease. The company was incorporated in 2017 and is based in New York, New York.

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.