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, including Exon 17 mutations, and targets PDGFRa, including the D842V mutation. These mutations activate receptor tyrosine kinases that are drivers of cancer and proliferative disorders, including gastrointestinal stromal tumors (GIST), and systemic mastocytosis (SM). Its drug candidate BLU-554 targets FGFR4, a kinase that is activated in a defined subset of patients with hepatocellular carcinoma (HCC), the common type of liver cancer. It is engaged in developing BLU-667, a drug candidate that targets RET, a receptor tyrosine kinase that is activated by mutations or translocations.