Insmed Incorporated, a biopharmaceutical company, develops and commercializes therapies for patients with serious and rare diseases in the United States, Europe, Japan, and internationally. The company offers ARIKAYCE for the treatment of Mycobacterium avium complex lung disease as part of a combination antibacterial drug regimen for adult patients. It is also developing Brensocatib, an oral reversible inhibitor of dipeptidyl peptidase 1 for the treatment of patients with bronchiectasis and other neutrophil-mediated diseases; and Treprostinil Palmitil Inhalation Powder, an inhaled formulation of a treprostinil prodrug treprostinil palmitil for the treatment of pulmonary hypertension associated with interstitial lung disease and pulmonary arterial hypertension. The company was founded in 1988 and is headquartered in Bridgewater, New Jersey.

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.

Liquidia Corporation, a biopharmaceutical company, develops, manufactures, and commercializes various products for unmet patient needs in the United States. Its lead product candidates include YUTREPIA, an inhaled dry powder formulation of treprostinil for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). The company also offers Remodulin, a treprostinil administered through continuous intravenous and subcutaneous infusion. The company also a license agreement with Pharmosa Biopharm Inc to develop and commercialize L606, an inhaled sustained-release formulation of Treprostinil for the treatment of PAH and PH-ILD. Liquidia Corporation was founded in 2004 and is headquartered in Morrisville, North Carolina.

aTyr Pharma, Inc., a biotherapeutics company, engages in the discovery and development of medicines based on novel immunological pathways in the United States. Its lead therapeutic candidate is efzofitimod, a selective modulator of NRP2 that is in Phase III clinical trial for pulmonary sarcoidosis; and in Phase 1b/2a clinical trial for treatment of other interstitial lung diseases (ILDs), such as chronic hypersensitivity pneumonitis and connective tissue disease related ILD. The company is developing ATYR0101, a fusion protein derived from a domain of aspartyl-tRNA synthetase that is in preclinical development for the treatment of fibrosis; and ATYR0750, a domain of alanyl-tRNA synthetase for the treatment of liver disorders. It has collaboration and license agreement with Kyorin Pharmaceutical Co., Ltd. for the development and commercialization of efzofitimod for ILDs in Japan. aTyr Pharma, Inc. was incorporated in 2005 and is headquartered in San Diego, 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.

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.

Aerovate Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops drugs that enhance the lives of patients with rare cardiopulmonary diseases in the United States. It focuses on advancing AV-101, a dry powder inhaled formulation of imatinib for the treatment of pulmonary arterial hypertension, which is in Phase 2b/Phase 3 trial. The company was incorporated in 2018 and is headquartered in Waltham, Massachusetts.

Lumos Pharma, Inc., a clinical-stage biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of products and therapies for people with rare diseases. Its primary product candidate is LUM-201, an oral growth hormone secretagogue ibutamoren, which is in Phase III clinical trial for the treatment of idiopathic pediatric growth hormone deficiency and other rare endocrine disorders. The company has a clinical collaboration with Massachusetts General Hospital to evaluate oral LUM-201 in nonalcoholic fatty liver disease. Lumos Pharma, Inc. is headquartered in Austin, Texas. As of December 12, 2024, Lumos Pharma, Inc. was taken private.