Ionis Pharmaceuticals, Inc., a commercial-stage biotechnology company, provides RNA-targeted medicines in the United States. The company offers TRYNGOLZA reduces triglyceride levels in adults with familial chylomicronemia syndrome (FCS) and acute pancreatitis; WAINUA for the treatment of the polyneuropathy of hereditary transthyretin-medicated amyloidosis (ATTRv-PN) in adults; and SPINRAZA for pediatric and adult patients with spinal muscular atrophy (SMA). It also provides QALSODY for the treatment of Amyotrophic Lateral Sclerosis (ALS); TEGSEDI for the treatment of ATTRv-PN in adults; and WAYLIVRA for treatment for FCS and familial partial lipodystrophy. It also develops products under Phase 3 clinical trials, such as Olezarsen for patients with hypertriglyceridemia (SHTG) and cardiovascular disease (CVD); Donidalorsen for patients with hereditary angioedema; and Zilganerse, a potential treatment for people with genetically confirmed Alexander disease, as well as ION582 that has completed Phase 2 clinical trial for the potential treatment of AS, a rare genetic neurological disease.
