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Keros Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops and commercializes novel therapeutics for patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta family of proteins in the United States. The company's lead product candidate is KER-050, which is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia in patients with myelodysplastic syndromes, as well as in patients with myelofibrosis. It also develops KER-012, which is in Phase II clinical trial to treat pulmonary arterial hypertension and cardiovascular disorders; and KER-065 that is in Phase I clinical trial for the treatment of obesity and neuromuscular diseases. In addition, the company has collaboration and license agreement with Hansoh (Shanghai) Healthtech Co., Ltd. to develop, manufacture, and commercialize KER-050 and licensed products containing KER-050. Keros Therapeutics, Inc. was incorporated in 2015 and is headquartered in Lexington, Massachusetts.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, acute leukemias, and solid tumor; and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD) and idiopathic pulmonary fibrosis (IPF). The company is also developing Entinostat. It has an agreement with Eddingpharm International Company Limited for licensing, development, and commercialization of Entinostat. Syndax Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Waltham, Massachusetts.

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.

Verve Therapeutics, Inc., a clinical stage genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases in the United States. The company's lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver; and VERVE-102, a product candidate that targets the PCSK9 gene for the treatment of HeFH. It engages in the development of ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics Inc.; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is headquartered in Cambridge, Massachusetts.