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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 partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, 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.
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.