Alnylam

Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company that develops RNA interference (RNAi) therapeutics. Founded in 2002 and headquartered in Cambridge, Massachusetts, the company focuses on medicines that silence disease-causing genes using RNA interference. Alnylam's core technology involves small interfering RNA (siRNA) molecules delivered to cells via N-acetylgalactosamine (GalNAc) ligands, enabling selective gene silencing in the liver and other tissues.

Alnylam went public in 2004 and has built partnerships to advance its medicines through development and commercialization. A landmark milestone was the approval of patisiran, marketed as Onpattro, for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy, marking the first FDA-approved RNAi therapeutic in 2018. Following this, the company achieved additional approvals, including givosiran (Givlaari) in 2019 for acute hepatic porphyrias, and lumasiran (Oxlumo) in 2020 for primary hyperoxaluria type 1, highlighting the clinical potential of RNAi medicines.

Alnylam maintains a pipeline of RNAi programs targeting genetic, hepatic, and metabolic diseases, with ongoing collaborations and internal research aimed at expanding tissue targeting and indications. The company operates globally, with research, manufacturing, and commercial activities designed to support its product portfolio and future growth in the RNA therapeutics field.