Founded in 1989 and headquartered in Carlsbad, California, Ionis Pharmaceuticals, Inc. is a biopharmaceutical company specializing in the discovery and development of RNA-targeted therapies within the United States. Its commercial portfolio includes three key medications: SPINRAZA, prescribed for spinal muscular atrophy (SMA) in both children and adults; TEGSEDI, an injectable treatment targeting polyneuropathy associated with hereditary transthyretin-mediated amyloidosis in adult patients; and WAYLIVRA, which addresses familial chylomicronemia syndrome and familial partial lipodystrophy. Ionis also maintains a robust pipeline, with several drug candidates currently in Phase 3 clinical trials. These include Eplontersen, a monthly self-administered subcutaneous injection designed for all forms of TTR amyloidosis; Olezarsen, intended for individuals with severe hypertriglyceridemia (SHTG); Donidalorsen, for patients suffering from hereditary angioedema; ION363, aimed at amyotrophic lateral sclerosis (ALS); Pelacarsen, developed for those with established cardiovascular disease and elevated lipoprotein(a); and Tofersen, which works by inhibiting the production of superoxide dismutase 1. Beyond these late-stage assets, the company is actively researching treatments for metabolic, infectious, renal, and ophthalmic diseases, as well as various types of cancer. The firm engages in significant collaborations, notably a strategic partnership with Biogen Inc. Additionally, it holds licensing and collaboration agreements with AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc.
