Blueprint Medicines Corporation, a biopharmaceutical company, develops drugs of small molecule kinase inhibitors that target genomic drivers in various cancers and rare genetic diseases. Its lead drug candidates include BLU-285, which is in Phase I clinical trials that targets KIT Exon 17 mutant proteins and PDGFRa D842V, abnormally active receptor tyrosine kinases for patients with systemic mastocytosis, a disorder of the mast cells, and defined subsets of patients with gastrointestinal stromal tumor; and BLU-554, which is in Phase I clinical trials an orally available, potent, selective, and irreversible inhibitor of the kinase FGFR4 that is activated in a defined subset of patients with hepatocellular carcinoma. It is also developing BLU-667, a drug candidate that targets RET, a receptor tyrosine kinase that is abnormally activated by mutations or translocations; and RET resistant mutants that would arise from treatment with first generation therapies, as well as candidates as inhibitors of neurotrophic tyrosine receptor kinase (NTRK) and predicted NTRK resistant mutants. In addition, the company is developing treatments that target cancer and rare genetic diseases. The company has agreements with Alexion Pharma Holding to research, develop, and commercialize drug candidates for an undisclosed activated kinase target, which is the cause of a rare genetic disease; and F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. to discover, develop, and commercialize small molecule therapeutics targeting kinases. The company was formerly known as Hoyle Pharmaceuticals, Inc. and changed its name to Blueprint Medicines Corporation in June 2011. Blueprint Medicines Corporation was founded in 2008 and is headquartered in Cambridge, Massachusetts.
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