Blueprint Medicines Corporation is a biopharmaceutical company. The Company focuses on patients with genomically defined diseases driven by abnormal kinase activation. It focuses on crafting drug candidates that provide clinical responses to patients without adequate treatment options. It has developed a small molecule drug pipeline in cancer and a genetic disease. Its drug candidate, BLU-285, targets KIT Exon 17 mutants and PDGFRa D842V, which are abnormally active receptor tyrosine kinases for patients with systemic mastocytosis, a myeloproliferative disorder of the mast cells, and defined subsets of patients with gastrointestinal stromal tumor. Its drug candidate, BLU-554, targets FGFR4, a kinase that is aberrantly activated in a defined subset of patients with hepatocellular carcinoma.