Ionis Pharmaceuticals, Inc.

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.

業界

Biotechnology

セクター

Healthcare

本社

US

最高経営責任者（CEO）

Brett Monia

上場以来

1991年5月17日

従業員

1,480

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.

業界

Biotechnology

セクター

Healthcare

本社

US

最高経営責任者（CEO）

Brett Monia

上場以来

1991年5月17日

従業員

1,480