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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.
Rhythm Pharmaceuticals Inc is a commercial-stage biopharmaceutical company. It is engaged in the development and commercialization of therapies for patients with rare diseases. The company is focused on its melanocortin-4 receptor (MC4R) agonists, including its lead asset, IMCIVREE (setmelanotide), as a precision medicine designed to treat hyperphagia and severe obesity caused by rare MC4R pathway diseases. Geographically the company generates its revenue from the United States and internationally with the majority being generated from the United States.