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.

Crinetics Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate is paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist, which is in Phase 3 trial for the treatment of acromegaly; and Phase 2 trial for treating carcinoid syndrome associated with neuroendocrine tumors. It is also developing CRN04894, an investigational oral nonpeptide product candidate to antagonize the adrenocorticotrophic hormone (ACTH) receptor that has completed a Phase 1 study for the treatment of diseases caused by excess ACTH, including congenital adrenal hyperplasia and Cushing's disease. In addition, the company is developing antagonists of the parathyroid hormone (PTH) receptor for the treatment of primary hyperparathyroidism and humoral hypercalcemia of malignancy, and other diseases of excess PTH; identified investigational orally available somatostatin receptor type 3 targeted nonpeptide agonists for the treatment of autosomal dominant polycystic kidney disease; and developing thyroid-stimulating hormone receptor antagonists for the treatment of graves' disease and thyroid eye disease, as well as Oral GLP-1 and GIP nonpeptides for the treatment of diabetes and obesity. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

Travere Therapeutics, Inc., a biopharmaceutical company, identifies, develops, and delivers therapies to people living with rare kidney and metabolic diseases. Its products include FILSPARI (sparsentan), a once-daily, oral medication designed to target two critical pathways in the disease progression of IgA Nephropathy (endothelin 1 and angiotensin-II); and Thiola and Thiola EC (tiopronin tablets) for the treatment of cystinuria, a rare genetic cystine transport disorder that causes high cystine levels in the urine and the formation of recurring kidney stones. The company's clinical-stage programs consist of Sparsentan, a novel investigational product candidate, which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis in the U.S. and Europe; and Pegtibatinase (TVT-058), a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria. It has a cooperative research and development agreement with National Institutes of Health's National Center for Advancing Translational Sciences and Alagille Syndrome Alliance for the identification of potential small molecule therapeutics for Alagille syndrome. The company was formerly known as Retrophin, Inc. and changed its name to Travere Therapeutics, Inc. in November 2020. Travere Therapeutics, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

Corvus Pharmaceuticals, Inc., a clinical stage biopharmaceutical company, focuses on the development and commercialization of immune modulator product candidates to treat solid cancers, T cell lymphomas, autoimmune, allergic, and infectious diseases. Its lead product candidate is soquelitinib (CPI-818), a selective covalent inhibitor of interleukin 2 inducible T cell kinase (ITK), which is in a multi-center Phase 1/1b clinical trial for the treatment of peripheral T cell lymphoma, solid tumors, and atopic dermatitis. The company is also developing ciforadenant (CPI-444), an oral small molecule antagonist of the A2A receptor that is in Phase 2 clinical trial for the treatment of metastatic renal cell cancer; and mupadolimab (CPI-006), a humanized monoclonal antibody, which is in Phase 1b clinical trial for the treatment of non-small cell lung cancer and head and neck cancer. In addition, it is developing CPI-182, an antibody designed to block inflammation and myeloid suppression that is in investigational new drug application-enabling studies, as well as CPI-935, an adenosine A2B receptor antagonist to prevent fibrosis. Corvus Pharmaceuticals, Inc. has a license afreemnt with Monash University to research, develop, and commercialize certain antibodies directed to CXCR2 for the treatment of human diseases; and Vernalis (R&D) Limited to develop, manufacture, and commercialize products containing certain adenosine receptor antagonists, including ciforadenant, as well as strategic collaboration with Angel Pharmaceuticals Co. Ltd. for the development and commercialization of mupadolimab. Corvus Pharmaceuticals, Inc. was incorporated in 2014 and is based in Burlingame, California.

Contineum Therapeutics, Inc., a clinical stage biopharmaceutical company, focuses on discovering and developing novel oral small molecule therapies for neuroscience, inflammation, and immunology indications with high unmet need. Its lead asset is PIPE-791, a novel, brain penetrant, small molecule inhibitor of the lysophosphatidic acid 1 receptor (LPA1R) for the treatment of idiopathic pulmonary fibrosis and progressive multiple sclerosis (MS). The company also develops PIPE-307, a novel, small molecule selective inhibitor of the muscarinic type 1 M1 receptor to treat depression and relapse remitting MS; and CTX-343, a peripherally-restricted LPA1R antagonist. Contineum Therapeutics, Inc. was formerly known as Pipeline Therapeutics, Inc. and changed its name to Contineum Therapeutics, Inc. in November 2023. The company was incorporated in 2009 and is headquartered in San Diego, California.

Connect Biopharma Holdings Limited, a clinical-stage biopharmaceutical company, engages in the development of therapies for the treatment of T cell-driven inflammatory diseases. The company is building a pipeline of small molecules and antibodies using functional T cell assays to screen and discover potent product candidates against validated immune targets. Its lead product candidate is rademikibart (formerly CBP-201), an antibody designed to target interleukin-4 receptor alpha, which is a validated target for the treatment of inflammatory diseases such as atopic dermatitis and asthma, currently under Phase 3 studies; and icanbelimod (formerly CBP-307), an oral small molecule Sphingosine 1-Phosphate Receptor 1 modulator, currently under Phase 2 clinical for the treatment of ulcerative colitis and Crohn's disease. The company was founded in 2012 and is based in San Diego, California.

CalciMedica, Inc., a clinical-stage biopharmaceutical company, focuses on developing therapeutics for illnesses caused by inflammatory and immunologic processes and direct cellular damage. The company's lead product candidate comprises Auxora, an intravenous formulated small molecule calcium release-activated (CRAC) channel inhibitors, which is in phase 2 clinical trials for the treatment of acute pancreatitis, asparaginase induced pancreatic toxicity, and acute kidney injury, as well as severe COVID-19 pneumonia. It also develops CM6336, an oral CRAC channel inhibitors for chronic inflammatory indications; and Auxora, for the treatment of acute ulcerative colitis and allergic asthma. The company is based in La Jolla, California.

Spruce Biosciences, Inc., a biopharmaceutical company, focuses on developing and commercializing novel therapies for rare endocrine disorders. The company engages in developing tildacerfont, a non-steroidal therapy to enhance disease control and reduce steroid burden for patients suffering from congenital adrenal hyperplasia (CAH), which is in Phase 2b clinical trial; and to evaluate glucocorticoid reduction in adult patients with classic CAH that is Phase 2b clinical trial. It is also developing tildacerfont for the treatment of pediatric classic congenital adrenal hyperplasia in children that is in Phase 2 clinical trial; and for females with polycystic ovary syndrome, which is in Phase 2 clinical trial. Spruce Biosciences, Inc. has a license agreement with Eli Lilly and Company to research, develop, and commercialize compounds for various pharmaceutical uses; and collaboration and license agreement with Kaken Pharmaceutical Co. Ltd. to develop, manufacture, and commercialize tildacerfont for the treatment of CAH in Japan. The company was incorporated in 2014 and is headquartered in South San Francisco, California.