BioMarin Pharmaceutical Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. It has license and collaboration agreements with Sarepta Therapeutics; Ares Trading S.A.; Catalyst Pharmaceutical Partners, Inc.; and CAMP4 Therapeutics Corporation. The company was incorporated in 1996 and is headquartered in San Rafael, California.

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.

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Japan, Europe, and internationally. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc.; Solid Biosciences Inc.; Regeneron; Abeona; and Daiichi Sankyo Co., Ltd. The company was incorporated in 2010 and is headquartered in Novato, California.

89bio, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapies for the treatment of liver and cardio-metabolic diseases. Its lead product candidate is pegozafermin, a glycoPEGylated analog of fibroblast growth factor 21 for the treatment of nonalcoholic steatohepatitis; and for the treatment of severe hypertriglyceridemia. The company was incorporated in 2018 and is headquartered in San Francisco, California.

Pliant Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes novel therapies for the treatment of fibrosis and related diseases in the United States. The company's lead candidate is bexotegrast, an oral, small-molecule, dual selective inhibitor of avß6 and avß1 integrins, which is in phase 2b trials for idiopathic pulmonary fibrosis and in phase 2a trial for primary sclerosing cholangitis. It also develops PLN-1474, an oral, small-molecule selective inhibitor of avß1 for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis; PLN-101095, a dual inhibitor of integrins avß8 and avß1 for the treatment of solid tumors; and PLN-101325 for treatment of muscular dystrophies. Pliant Therapeutics, Inc. was incorporated in 2015 and is based in South San Francisco, California.

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.

Aligos Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of novel therapeutics to address unmet medical needs in viral and liver diseases. Its drug candidate, ALG-055009, a small molecule THR-ß agonist that is in the Phase 2a clinical trial for the treatment of non-alcoholic steatohepatitis (NASH). The company also develops ALG-000184, a capsid assembly modulator, which is completed Phase 1b clinical trial to treat chronic hepatitis B (CHB); and ALG-125755, a siRNA drug candidate, which is in Phase I clinical trial for the treatment of CHB. In addition, it develops ALG-097558, which is in Phase 2 clinical trial for the treatment of coronavirus. The company has entered into license and research collaboration agreement with Merck to discover, research, optimize, and develop oligonucleotides directed against a NASH; license agreement with Emory University to provide hepatitis B virus capsid assembly modulator technology; license agreement with Luxna Biotech Co., Ltd. to develop and commercialize products containing oligonucleotides targeting hepatitis B virus genome; and research, licensing, and commercialization agreement with Katholieke Universiteit Leuven to develop coronavirus protease inhibitors. It also has a clinical collaboration with Xiamen Amoytop Biotech Co., Ltd. Aligos Therapeutics, Inc. was incorporated in 2018 and is headquartered in South San Francisco, California.

NGM Biopharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of novel therapeutics to treat liver and metabolic diseases, retinal diseases, and cancer. The company develops NGM707, an immunoglobulin-like transcript 2/ immunoglobulin-like transcript 4 dual antagonist monoclonal antibody that is in Phase I/II clinical trials for the treatment of patients with advanced metastatic solid tumors; and Aldafermin, an engineered analog of human hormone fibroblast growth factor 19, which is in Phase IIb clinical trials for the treatment of primary sclerosing cholangitis and non-alcoholic steatohepatitis. It also develops NGM120, an GFRAL antagonist antibody, which is in Phase II clinical trial to treat hyperemesis gravidarum; NGM831 and NGM438 which is in phase I/II clinical trial for the treatment of advanced solid tumors; and NGM621, an monoclonal antibody in Phase II clinical trials for the treatment of geographic atrophy. The company has research collaboration, product development, and license agreements with Merck Sharp & Dohme Corp.; and a collaboration agreement with Merck to focus primarily on the identification, research and development of collaboration compounds directed to targets in the fields of ophthalmology and cardiovascular or metabolic, or CVM, disease, including heart failure. NGM Biopharmaceuticals, Inc. was incorporated in 2007 and is headquartered in South San Francisco, California.