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Blueprint Medicines Corp is a biopharmaceutical company. It is focused on improving the lives of patients with diseases driven by abnormal kinase activation. The company has developed a small molecule drug pipeline in cancer and a rare genetic disease. Its drug candidates includes AYVAKIT for the treatment of systemic mastocytosis, BLU-808 a potential treatment for mast cell disorders, including chronic urticaria, sleep disruption and other related diseases. It also has other drugs in its pipeline such as BLU-956, BLU-222 for treatment of breast cancer.
Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and Biogen brought Spinraza to market in 2016 as a treatment for spinal muscular atrophy, and Biogen launched ALS drug Qalsody in 2023. Ionis brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019). Ionis and AstraZeneca launched polyneuropathy drug Wainua in 2024. Ionis received FDA approval in 2024 for Tryngolza for a rare high-triglyceride syndrome, marking its first independent launch.