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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 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.
Arrowhead Pharmaceuticals Inc is an American biotechnology company. Its main target is the development of medicine to treat diseases with a genetic origin, characterized by the overproduction of one or more proteins. Its medical solutions are aimed at the genes that trigger the diseases. Its portfolio includes drugs targeting cardiovascular, Cardiometabolic, Central Nervous System, Pulmonary, Liver, Muscular, and Complement Mediated Disease. It majority products are liver disease curable.