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 form of ALS ION859 for Parkinson's disease ION464 for multiple system atrophy and Parkinson's disease ION541 for amyotrophic lateral sclerosis and IONIS-DNM2-2.5Rx for centronuclear myopathy. The company is also developing products for cardiometabolic disease, such as IONIS-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly IONIS-APOCIII-LRx for familial chylomicronemia syndrome and TG- driven diseases IONIS-APOCIII-LRx for treatment resistant hypertension Pelacarsen for Lp(a)cardiovascular disease risk reduction Vupanorsen for sHTG/CVDRR IONIS-FXI-LRx for clotting disorders ION449 for cardiovascular diseases and IONIS-GCGRRx for diabetics. In addition, it develops IONIS-ENAC-2.5Rx for cystic fibrosis and chronic obstructive pulmonary disease IONIS-PKK-LRx for hereditary angioedema Danvatirsen for cancer IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity IONIS-HBVRx for hepatitis B virus IONIS-AR-2.5Rx for prostate cancer IONIS-FB-LRx for age-related macular degeneration and IgA nephropathy and ION357 for retinitis pigmentosa. It has a collaboration agreement with AstraZeneca GlaxoSmithKline plc Janssen Biotech, Inc. Bayer AG Novartis AG Pfizer, Inc. Biogen Inc. and Roche. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.