Alnylam Pharmaceuticals

Pioniere RNA interference terapeutico per malattie rare.

Alnylam Pharmaceuticals, Inc. discovers, develops, manufactures, and commercializes therapeutics based on ribonucleic acid interference in the United States, Europe, and internationally. The company offers ONPATTRO for hereditary transthyretin-mediated (hATTR) amyloidosis; AMVUTTRA for ATTR and hATTR amyloidosis; Leqvio for hypercholesterolemia; Qfitlia for hemophilia A or B; GIVLAARI for acute hepatic porphyria; and OXLUMO for primary hyperoxaluria type 1. It also develops a range of products in Phase 3 trial, such as Nucresiran for ATTR amyloidosis; Zilebesiran for hypertension; Cemdisiran for myasthenia gravis, paroxysmal nocturnal hemoglobinuria, and geographic atrophy; and Elebsiran for hepatitis D virus infections. In addition, the company is developing various products in Phase 2 trial, including Rapirosiran for metabolic dysfunction-associated steatohepatitis; ALN-ANG3 for diabetic kidney disease; ALN-4324 for type 2 diabetes mellitus; Mivelsiran for cerebral amyloid angiopathy, as well as in Phase 1 trial for Alzheimer's disease; and ALN-6400 for bleeding disorders. Further, it develops a range of products in Phase 1 trial, such as ALN-2232 for obesity and weight management; ALN-PNP for non-alcoholic fatty liver disease; ALN-APOC3 for dyslipidemia; ALN-CIDEB for metabolic dysfunction-associated steatohepatitis; ALN-HTT02 for Huntington's disease; ALN-5288 for Alzheimer's disease; ALN-SOD for SOD1 amyotrophic lateral sclerosis; ALN-SNCA for Parkinson's disease; AG-236 for polycythemia vera; ALN-CFB for paroxysmal nocturnal hemoglobinuria; ALN-BCAT for hepatocellular carcinoma; and ALN-4285, ALN-4915, and ALN-F1202 for healthy volunteers. The company has collaborations with Regeneron Pharmaceuticals, Inc.; Roche Holding AG; Sanofi S.A.; Novartis AG; PeptiDream, Inc; Dicerna Pharmaceuticals, Inc.; and Ionis Pharmaceuticals, Inc. Alnylam Pharmaceuticals, Inc. was founded in 2002 and is headquartered in Cambridge, Massachusetts.