Ionis Pharmaceuticals, Inc., a RNA-targeted drug discovery and development company, focuses on developing drugs for patients with severe and rare diseases. It markets KYNAMRO for the treatment of homozygous familial hypercholesterolemia. The company's drugs in Phase III development include volanesorsen for the treatment of patients with familial chylomicronemia syndrome and familial partial lipodystrophy; IONIS-TTRRx, a drug for the treatment of patients with various forms of TTR amyloidosis, including familial amyloid polyneuropathy, familial amyloid cardiomyopathy, and wild-type TTR amyloidosis; and nusinersen, a drug for the treatment of infants and children with spinal muscular atrophy. It is also developing Custirsen, which is in two Phase III studies to treat cancer; and Plazomicin that is Phase III study for the treatment of severe bacterial infection. In addition, the company is developing products, which are Phase II clinical trials include IONIS-DMPK-2.5Rx for myotonic dystrophy type 1; IONIS-HTTR for huntington's disease; ATL1103 for acromegaly; IONIS-FXIRx (BAY 2306001) for clotting disorders; IONIS-APO(a)-LRx for high Lp; IONIS-ANGPTL3-LRx for mixed dyslipidemias; Apatorsen (OGX-427) and IONIS-STAT3-2.5Rx (AZD9150) for cancers; EXC 001 (PF-06473871) for scarring; ATL1102 for multiple sclerosis; and RG-101 for hepatitis C virus infection, as well as products for the treatment of diabetes and obesity. Further, its products in Phase I clinical include IONIS-GCCRRx for cushing's syndrome; IONIS-PKKRx for hereditary angioedema; RG-012 for alport syndrome; IONIS-AR-2.5Rx (AZD5312) for prostate cancer; IONIS-HBVRx for hepatitis B virus infection; IONIS-GSK4-LRx for ocular disease; and IONIS-DGAT2Rx for nonalcoholic steatohepatitis. 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.
Cancer Organ Systems Diabetes Cancers Drug Discovery Prostate Cancer Disorders Multiple Sclerosis Obesity Genetics Neurological Disorders Huntington's Disease Spinal Muscular Atrophy Treatment Of Diabetes Orphan Drugs Acromegaly Cushing's Syndrome Hepatitis C Virus Infection Hereditary Angioedema Therapeutic Gene Modulation Antisense RNA Familial Amyloid Polyneuropathy Severe And Rare Diseases Familial Chylomicronemia Syndrome Familial Partial Lipodystrophy Homozygous Familial Hypercholesterolemia Myotonic Dystrophy TTR Amyloidosis