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 injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It is involved in developing neurology products that include Tominersen for Huntington's diseases; Tofersen for amyotrophic lateral sclerosis (ALS); IONIS-MAPTRx for Alzheimer's diseases; IONIS-C9Rx for genetic from ALS; ION859 for Parkinson's disease; and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing severe and rare disease products, such as AKCEA-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; IONIS-PKK-LRx for hereditary angioedema; IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity; IONIS-ENAC-2.5Rx for cystic fibrosis; and ION357 for retinitis pigmentosa. In addition, its cardio metabolic and renal drugs include AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx for cardiovascular diseases; AKCEA-ANGPTL3-LRx for cardio metabolic disorders; IONIS-GCGRRx for type 2 diabetes; IONIS-AGT-LRX for the treatment of resistant hyper tension; IONIS-FXI-LRx for clotting disorders; IONIS-AZ4-2.5-LRx for cardiovascular diseases; and ION839 for nonalcoholic steatohepatitis. Further, the company develops products for oncology that include IONIS-AR-2.5Rx and danvatirsen for cancers; IONIS-HBVRx and IONIS-HBV-LRx for hepatitis B virus infection; and IONIS-FB-LRx for compliment meditated disease. It has a collaboration agreement with AstraZeneca; Biogen Inc.; and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.