Ionis Pharmaceuticals, Inc. engages in discovering and developing RNA-targeted therapeutics. The company markets SPINRAZA, an antisense drug for spinal muscular atrophy in pediatric and adult patients; and Kynamro an oligonucleotide inhibitor for use in patients with homozygous familial hypercholesterolemia to reduce low density lipoprotein-cholesterol, apolipoprotein B, total cholesterol, and non-high density lipoprotein-cholesterol, and as an adjunct to lipid lowering medications and diet. Its drugs in Phase III development include volanesorsen for the treatment of patients with familial chylomicronemia syndrome and familial partial lipodystrophy; and IONIS-TTRRx, an antisense drug to treat all forms of transthyretin amyloidosis. The company also develops IONIS-HTTRx, an antisense drug to treat patients with Huntington’s disease; IONIS-SOD1Rx, an antisense drug to treat patients with an inherited form of amyotrophic lateral sclerosis; AKCEA-ANGPTL3-LRx, a LICA drug for treating multiple lipid disorders or mixed dyslipidemias; IONIS-PKKRx, an antisense drug that treats patients with hereditary angioedema; IONIS-FXIRx, an antisense drug for the treatment of thrombosis; AKCEA-APO(a)-LRx, a LICA drug that reduces apolipoprotein(a) in the liver to offer a direct approach for reducing lipoprotein(a); and AKCEA-APOCIII-LRx, an antisense drug for cardiometabolic disease. In addition, it develops IONIS-AR-2.5Rx, an antisense drug that treats patients with prostate cancer; IONIS-STAT3-2.5Rx, a cancer drug; AKCEA-ANGPTL3-LRx, a LICA drug to treat multiple lipid disorders; IONIS-GCGRRx, an antisense drug to treat patients with type 2 diabetes; and IONIS-DGAT2Rx, an antisense drug to treat patients with liver disease. The company was formerly known as Isis Pharmaceuticals, Inc. and changed its name to Ionis Pharmaceuticals, Inc. in December 2015. Ionis Pharmaceuticals, Inc. was founded in 1989 and is headquartered in Carlsbad, California.
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