Pioneers RNA interference (RNAi) therapeutics to treat a range of serious, genetically-defined diseases.
Alnylam Pharmaceuticals, Inc. is a pioneering biopharmaceutical company dedicated to advancing novel therapeutics through the innovative science of ribonucleic acid interference (RNAi). Headquartered in Cambridge, Massachusetts, Alnylam focuses on discovering, developing, and commercializing RNAi-based medicines across a diverse range of therapeutic areas. Its robust pipeline includes investigational RNAi therapeutics targeting genetic diseases, cardio-metabolic conditions, hepatic infectious diseases, and central nervous system (CNS) and ocular disorders.
Among its successful products, Alnylam markets ONPATTRO (patisiran), a lipid complex injection for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. GIVLAARI is another marketed product, indicated for adults with acute hepatic porphyria (AHP), alongside OXLUMO (lumasiran) for primary hyperoxaluria type 1 (PH1). The company is also advancing several therapies in clinical trials, including givosiran for adolescent AHP patients, patisiran for transthyretin amyloidosis with cardiomyopathy, and cemdisiran for complement-mediated diseases.
Alnylam's collaborative efforts are integral to its mission, partnering with industry leaders like Regeneron Pharmaceuticals, Inc. and Sanofi Genzyme to develop and commercialize RNAi therapies targeting diseases affecting the eye, CNS, and beyond. The company also engages in strategic alliances with Novartis AG, Vir Biotechnology, Inc., Dicerna Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., and PeptiDream, Inc. These partnerships underscore Alnylam's commitment to advancing RNAi therapeutics and addressing unmet medical needs globally. Founded in 2002, Alnylam Pharmaceuticals continues to lead the field of RNAi-based medicine with groundbreaking research and a dedication to transforming patient care.