Develops RNA-targeted therapeutics for rare and severe diseases, focusing on antisense technology to modulate gene expression.
Ionis Pharmaceuticals, Inc. is a pioneering biopharmaceutical company based in Carlsbad, California, dedicated to the discovery and development of RNA-targeted therapeutics. Renowned for its innovative approach, Ionis Pharmaceuticals has brought groundbreaking treatments to market, including SPINRAZA, designed for spinal muscular atrophy (SMA) across pediatric and adult patient populations. The company also offers TEGSEDI, an injectable therapy addressing polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis, and WAYLIVRA for familial chylomicronemia syndrome and familial partial lipodystrophy.
In its robust pipeline, Ionis Pharmaceuticals is advancing several promising therapies through clinical trials. Notable candidates include Eplontersen, a monthly subcutaneous injection for all types of TTR amyloidosis; Olezarsen targeting severe hypertriglyceridemia; and Donidalorsen, developed for hereditary angioedema. The company is also focused on advancing treatments such as ION363 for amyotrophic lateral sclerosis, Pelacarsen for cardiovascular disease, and Tofersen to inhibit superoxide dismutase 1 production, among others. Ionis Pharmaceuticals collaborates strategically with leading biopharmaceutical companies like Biogen Inc. and has established significant partnerships with AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc.
Founded in 1989, Ionis Pharmaceuticals continues to push the boundaries of RNA-targeted therapeutic innovation. With a steadfast commitment to addressing a wide range of medical needs, from metabolic diseases to infectious diseases, renal diseases, ophthalmic conditions, and cancer, Ionis Pharmaceuticals remains at the forefront of advancing precision medicine solutions globaly.