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Ionis Pharmaceuticals, Inc. is a U.S.-based biotechnology company specializing in the discovery and development of RNA-targeted therapeutic agents. The company's commercialized portfolio features SPINRAZA, a treatment for spinal muscular atrophy (SMA) in both pediatric and adult patients. It also offers TEGSEDI, an injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, and WAYLIVRA, approved for familial chylomicronemia syndrome and familial partial lipodystrophy. Beyond its marketed therapies, Ionis is actively progressing numerous drug candidates through pivotal Phase 3 clinical trials. These late-stage programs include Eplontersen, a monthly self-administered subcutaneous injection targeting all types of TTR amyloidosis; Olezarsen, designed for patients with severe hypertriglyceridemia (SHTG); Donidalorsen, for hereditary angioedema; ION363, aimed at amyotrophic lateral sclerosis; Pelacarsen, for individuals with established cardiovascular disease and elevated lipoprotein(a); and Tofersen, developed to inhibit superoxide dismutase 1 production. Furthermore, Ionis is exploring treatments across a broader spectrum of therapeutic areas, such as metabolic, infectious, renal, ophthalmic diseases, and cancer. The company maintains a significant strategic collaboration with Biogen Inc. and holds various collaboration and licensing agreements with other prominent pharmaceutical partners, including AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc. Founded in 1989, Ionis Pharmaceuticals, Inc. operates from its headquarters in Carlsbad, California.
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