Alnylam Pharmaceuticals Inc

Alnylam Pharmaceuticals is a global biopharmaceutical company and pioneer of RNA interference (RNAi) therapeutics. The company develops and commercializes medicines that silence specific messenger RNA to prevent disease-causing proteins from being made. Alnylam has four commercially approved products: AMVUTTRA (vutrisiran) for the treatment of transthyretin-mediated amyloidosis encompassing both polyneuropathy and cardiomyopathy; ONPATTRO (patisiran) for hereditary transthyretin amyloidosis with polyneuropathy; GIVLAARI (givosiran) for acute hepatic porphyria; and OXLUMO (lumasiran) for primary hyperoxaluria type 1. AMVUTTRA is the company's primary growth driver, approved for the full spectrum of ATTR amyloidosis including ATTR-CM, the larger market opportunity. Alnylam maintains a deep clinical pipeline spanning neurology (mivelsiran for Alzheimer's disease and cerebral amyloid angiopathy, ALN-HTT02 for Huntington's disease), cardiovascular (zilebesiran for hypertension), hematology (ALN-6400 for von Willebrand disease and hereditary hemorrhagic telangiectasia), and metabolic diseases (ALN-6222 for obesity). The company has strategic collaborations with Regeneron, Roche, Novartis, and Sanofi, and earns royalties on Novartis's Leqvio. Alnylam's medicines are available in more than 70 countries, with the ATTR-CM indication representing a significant commercial opportunity as diagnosis rates improve. Updated Jul 30, 2026