SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin am.

Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an antisense injection for the treatment of polyneuropathy caused by hereditary transthyretin amyloidosis in adults; and WAYLIVRA, an antisense medicine for treatment for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of ATTR; Olezarsen for patients with FCS and severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Tofersen to inhibit the production of superoxide dismutase 1; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Bepirovirsen to inhibit the production of viral proteins associated with hepatitis B virus. In addition, the company develops Cimdelirsen to inhibit the production of growth hormone receptor; IONIS-FB-LRx to inhibit the production of complement factor B and the alternative complement pathway; and ION224 to reduce the production of diacylglycerol acyltransferase 2. It has a strategic collaboration with Biogen for the treatment of neurological disorders; and collaboration and license agreement with Metagenomi, Inc, AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis, Roche, Swedish Orphan Biovitrum AB, and PTC Therapeutics. Ionis Pharmaceuticals, Inc. was incorporated in 1989 and is based in Carlsbad, California.