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.

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.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. The company was incorporated in 1989 and is based in Carlsbad, 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.

Regulus Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on discovery and development of drugs that targets microRNAs to treat a range of diseases in the United States. Its product candidates include RGLS8429, an anti-miR next generation oligonucleotide targeting miR-17, which is in Phase 1b clinical trial for the treatment of autosomal dominant polycystic kidney disease. The company is also developing a pipeline of preclinical drug products for target organ-selective delivery strategies. Regulus Therapeutics Inc. was incorporated in 2007 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.

MediciNova, Inc., a biopharmaceutical company, focuses on developing novel and small molecule therapeutics for the treatment of serious diseases with unmet medical needs in the United States. It is developing MN-166 (ibudilast), an oral anti-inflammatory and neuroprotective agent for treating neurological and other disorders, such as primary and secondary progressive multiple sclerosis, amyotrophic lateral sclerosis, chemotherapy-induced peripheral neuropathy, degenerative cervical myelopathy, glioblastoma, and substance dependence and addiction, as well as prevention of acute respiratory distress syndrome, and long COVID. The company's product pipeline also includes MN-221 (bedoradrine), a selective beta-2-adrenergic receptor agonist for the treatment of acute exacerbations of asthma; MN-001 (tipelukast), an orally bioavailable small molecule compound to treat fibrotic and other diseases, including nonalcoholic fatty liver disease and idiopathic pulmonary fibrosis; and MN-029 (denibulin), a tubulin binding agent for treating solid tumor cancers. It has license agreements with Kyorin Pharmaceutical Co., Ltd; Angiogene Pharmaceuticals, Ltd.; and Meiji Seika Kaisha, Ltd. The company was incorporated in 2000 and is headquartered in La Jolla, California.