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Blueprint Medicines Corporation, a precision therapy company, develops medicines for genomically defined cancers and blood disorders in the United States and internationally. The company is developing AYVAKIT for the treatment of systemic mastocytosis (SM) and gastrointestinal stromal tumors; BLU-263, an orally available, potent, and KIT inhibitor for the treatment of indolent SM, and other mast cell disorders. It is also developing GAVRETO for the treatment of RET fusion-positive non-small cell lung cancer, altered thyroid carcinoma, and medullary thyroid carcinoma; BLU-945 for the treatment of epidermal growth factor receptor driven non-small-cell lung carcinoma (NSCLC); and BLU-451 to treat NSCLC in patients with epidermal growth factor receptor gene (EGFR) exon 20 insertion mutations. In addition, the company is developing BLU-782, for the treatment of fibrodysplasia ossificans progressive; BLU- 222 to treat patients with cyclin E aberrant cancers; and BLU-852 for the treatment of advanced cancers. It has collaboration and license agreements with Clementia Pharmaceuticals, Inc.; Proteovant Therapeutics; CStone Pharmaceuticals; Genentech, Inc.; Hoffmann-La Roche Inc.; and Zai Lab (Shanghai) Co., Ltd. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was incorporated in 2008 and is headquartered in Cambridge, Massachusetts.

Galapagos NV, a biotechnology company, develops medicines focusing on oncology and immunology primarily in the United States and Europe. The company's pipeline products comprise GLPG3667 that has completed phase 1b trial; GLPG5101, a CD19 CAR-T product candidate manufactured at point-of-care, currently in Phase1/2 trial in relapsed/refractory non-hodgkin lymphoma; GLPG5201, a CD19 CAR-T product candidates manufactured at point-of-care, currently in phase 1/2 trial in replapsed/refractory chronic lymphocytic leukemia; and GLPG5301, a BCMA CAR-T product candidate manufactured at point-of-care, currently in phase 1/2 in relapsed/refractory multiple myeloma. The company has collaboration agreements with Gilead Sciences, Inc.; and AbbVie S.à r.l. Galapagos NV was incorporated in 1999 and is headquartered in Mechelen, Belgium.

IVERIC bio, Inc., a biopharmaceutical company, focuses on the discovery and development of novel treatments for retinal diseases with unmet medical needs. The company develops Zimura, an inhibitor of complement factor C5 comprising GATHER1, which has completed Phase 2/3 clinical trial; GATHER2 that is in Phase 3 clinical trial for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD); and STAR or OPH2005 that is in Phase 2b clinical trial for the treatment of autosomal recessive Stargardt disease. Its preclinical product candidatures include IC-500, a high temperature requirement A serine peptidase 1 protein inhibitors for the treatment of GA and other age-related retinal diseases. The company also develops OPH2001 that has completed Phase 1/2a clinical trial of various doses of avacincaptad pegol (ACP) administered as a monotherapy for the treatment of GA; OPH2000, which has completed Phase 1/2a clinical trial of various doses of ACP administered in combination with Lucentis for the treatment of wet AMD; OPH2007 that has completed Phase 2a clinical trial for the treatment of wet AMD; and OPH2002, which has completed Phase 2a clinical trial for the treatment of idiopathic polypoidal choroidal vasculopathy. In addition, its minigene programs comprises miniCEP290 program for LCA10; miniABCA4 Program for STGD1; and miniUSH2A Program for usher syndrome type 2A-Related inherited retinal diseases (IRDs). The company was formerly known as Ophthotech Corporation and changed its name to IVERIC bio, Inc. in April 2019. The company was incorporated in 2007 and is based in Parsippany, New Jersey. As of July 11, 2023, IVERIC bio, Inc. operates as a subsidiary of Astellas US Holding, Inc.

Myovant Sciences Ltd. is a clinical-stage biopharmaceutical company, which engages in the development and commercialization of therapies for the treatment of women's health and endocrine diseases. It offers ORGOVYXTM, an oral gonadotropin-releasing hormone (GnRH) receptor antagonist for the treatment of adult patients with advanced prostate cancer, and MYFEMBREE, which is taken for the management of heavy menstrual bleeding associated with uterine fibroids. The company was founded on February 2, 2016 and is headquartered in London, the United Kingdom.