PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd, Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

Protagonist Therapeutics, Inc., a biopharmaceutical company, develops peptide-based drugs for hematology and blood disorders, and inflammatory and immunomodulatory diseases. It is developing Rusfertide (PTG-300), an injectable hepcidin mimetic that completed phase 2 clinical trials for the treatment of polycythemia vera and other blood disorders; and JNJ-2113, an orally delivered investigational drug to block biological pathways that completed phase 2b clinical trials for the treatment of moderate-to-severe plaque psoriasis; and PN-943, an orally delivered, gut-restricted alpha 4 beta 7 specific integrin antagonist completed a phase 2 clinical trials in patients with moderate to severe ulcerative colitis. The company has a license and collaboration agreement with Takeda to commercialize rusfertide; and JNJ Innovative Medicines to co-develop Interleukin-23 receptor antagonist compound JNJ-2113. Protagonist Therapeutics, Inc. was incorporated in 2006 and is headquartered in Newark, 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.

PureTech Health plc, engages in the development and commercialization of biotechnology and pharmaceutical solutions in the United States. The company is developing LYT-100, which is under Phase 2 stage, to treat idiopathic pulmonary fibrosis (IPF); and LYT-200, a IgG4 monoclonal antibody, currently under Phase 1/2 stage, targeting galectin-9 for treating solid tumors and hematological malignancies. It also develops SPT-300, an oral drug of allopregnanolone for the treatment of anxious depression; SPT-320, a novel prodrug of agomelatine for the treatment of generalized anxiety disorder; and SPT-348, a prodrug of a non-hallucinogenic neuroplastogen for the treatment of mood and other neuropsychiatric disorders. In addition, the company is developing hydrogels to enable the oral administration of peptide therapeutics in preclinical development. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.

Ventyx Biosciences, Inc., a clinical-stage biopharmaceutical company, develops small molecule product candidates to address a range of inflammatory diseases. The company's lead clinical product candidate is VTX958, a selective allosteric tyrosine kinase type 2 inhibitor for psoriasis, psoriatic arthritis, and Crohn's disease. It is also developing VTX002, a sphingosine 1 phosphate receptor modulator that is in Phase II clinical trials for the treatment of ulcerative colitis; and VTX2735, a peripheral-targeted NOD-like receptor protein 3 inflammasome inhibitor to treat patients with cryopyrin-associated periodic syndrome. In addition, the company develops VTX3232, a CNS-penetrant NLRP3 inhibitor. Ventyx Biosciences, Inc. was incorporated in 2018 and is based 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.

VYNE Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing proprietary and therapeutics for the treatment of immuno-inflammatory conditions. The company's lead product is VYN201, a locally administered pan- bromodomain and extra-terminal (BET) inhibitor soft drug to address diseases involving multiple, diverse inflammatory cell signaling pathways with low systemic exposure. It also develops VYN202, a BD2-selective oral small molecule BET inhibitor for the treatment of immuno-inflammatory indications. The company was formerly known as Menlo Therapeutics Inc. and changed its name to VYNE Therapeutics Inc. in September 2020. VYNE Therapeutics Inc. was founded in 2003 and is based in Bridgewater, New Jersey.

Palisade Bio, Inc., a clinical-stage biopharmaceutical company, focuses on focuses on developing therapeutics that protect the integrity of the intestinal barrier in the United States. The company's lead therapeutic candidate is PALI-2108, a prodrug PDE4 inhibitor, currently under pre-clinical development as a therapeutic for patients living with inflammatory bowel diseases, including ulcerative colitis and Crohn's disease (CD), as well as develops PALI-1908, an oral, selective PDE4 inhibitor prodrug that is locally bioactivated in the terminal ileum of CD patients, currently in the research stage. It has a research collaboration and license agreement with Giiant Pharma, Inc. for the development, manufacture, and commercialization of its compounds; a license agreement with the Regents of the University of California; a co-development and distribution agreement with Newsoara Biopharma Co., Ltd; and a transformative strategic collaboration with Strand Life Sciences for advancing precision medicine for ulcerative colitis therapy. The company is based in Carlsbad, California.