United Therapeutics Corporation, a biotechnology company, engages in the development and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening diseases in the United States and internationally. The company offers Tyvaso DPI, an inhaled dry powder via pre-filled and single-use cartridges; Tyvaso, an inhaled solution via ultrasonic nebulizer; Remodulin (treprostinil) injection to treat patients with pulmonary arterial hypertension (PAH) to diminish symptoms associated with exercise; Orenitram, a tablet dosage form of treprostinil, to delay disease progression and improve exercise capacity in PAH patients; and Adcirca, an oral PDE-5 inhibitor to enhance the exercise ability in PAH patients. It also markets and sells Unituxin (dinutuximab) injection, a monoclonal antibody for treating high-risk neuroblastoma; and Remunity Pump, which contains a pump and separate controller for Remodulin. In addition, the company engages in developing RemoPro and Ralinepag for the treatment of PAH; Aurora-GT, a gene therapy product to rebuild the blood vessels in the lungs; and Nebulized Tyvaso, for the treatment of idiopathic pulmonary fibrosis, as well as xenografts, which are development-stage organ products. It has licensing and collaboration agreements with DEKA Research & Development Corp. to develop a semi-disposable system for the subcutaneous delivery of treprostinil; MannKind Corporation to develop and license treprostinil inhalation powder and the Dreamboat device; and Arena Pharmaceuticals, Inc. to develop Ralinepag. The company was incorporated in 1996 and is headquartered in Silver Spring, Maryland.

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.

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.

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.

Unity Biotechnology, Inc., a biotechnology company, engages in the research and development of therapeutics to slow, halt, or reverse diseases of aging. The company's lead drug candidate includes UBX1325, which is Phase 2 clinical trial for the treatment of age-related diseases of the eye, including diabetic macular edema, age-related macular degeneration, and diabetic retinopathy. It also develops UBX1967 for the treatment of ophthalmologic diseases; UBX2048, a Tie2/VEGF bispecific; UBX2050, a human anti-Tie2 agonist monoclonal antibody for the treatment of eye diseases; and UBX2089, a a-Klotho hormone drug candidate for multiple neurology indications. The company was formerly known as Forge, Inc. and changed its name to Unity Biotechnology, Inc. in January 2015. Unity Biotechnology, Inc. was incorporated in 2009 and is headquartered in South San Francisco, California.

Cyclerion Therapeutics, Inc., a biopharmaceutical company, develops treatments for serious diseases. It is developing Olinciguat, an orally administered vascular soluble guanylate cyclase (sGC) stimulator, which is in Phase 2 clinical trial to out-license for cardiovascular diseases; and Praliciguat, a systemic sGC stimulator that is licensed to Akebia Therapeutics, Inc. for the treatment of rare kidney disease. The company was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.

Vincerx Pharma, Inc., a clinical-stage biopharmaceutical company, researches and develops therapies to address unmet medical needs for the treatment of cancer in the United States. The company is developing enitociclib, a cyclin-dependent kinase-9 inhibitor that is in Phase 1 clinical trials for treating patients with relapsed and refractory leukemia and lymphoid malignancies, as well as solid tumors and non-Hodgkin lymphoma; and VIP236, a small molecule drug conjugate that is in Phase 1 clinical trials to treat advanced or metastatic solid tumors. Its product candidates also include VIP943 in Phase 1 clinical trials for the treatment of relapsed/refractory acute myeloid leukemia, myelodysplastic syndrome, and B-cell acute lymphoblastic leukemia; and VIP924 in preclinical studies for the treatment of B-cell malignancies. Vincerx Pharma, Inc. was founded in 2019 and is based in Palo Alto, California.

Oncternal Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on the development of oncology therapies for cancers with critical unmet medical needs. The company's clinical pipeline includes zilovertamab, a humanized monoclonal antibody that binds to receptor-tyrosine kinase-like Orphan Receptor 1 (ROR1); and ONCT-216, a small molecule inhibiting the biological activity of ETS-family transcription factor oncoproteins, which is in Phase 1/2 clinical trial. It is also developing ONCT-808, a chimeric antigen receptor T-cells (CAR-T), which is in Phase 1/2 clinical trial for the treatment of hematologic malignancies and solid tumors, as well as targets ROR1; and ONCT-534, a dual-action androgen receptor inhibitor product candidate in preclinical development for the treatment of castration-resistant prostate and other androgen receptor-driven cancers. The company has license agreements with the Regents of the University of California; Georgetown University; The University of Texas MD Anderson Cancer Center; Shanghai Pharmaceutical (USA) Inc.; and University of Tennessee Research Foundation. Oncternal Therapeutics, Inc. is headquartered in San Diego, California.