Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) is a biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Founded to translate the scientific discovery of RNAi into new medicines, Alnylam applies small interfering RNA (siRNA) technology to silence disease-causing genes. The company develops therapies designed to provide durable disease modification by targeting underlying genetic drivers across a range of rare and more prevalent conditions.
Alnylam has advanced multiple siRNA-based products into commercialization, initially using lipid nanoparticle delivery and more recently employing GalNAc-conjugate chemistry to enable targeted delivery to the liver with subcutaneous dosing. Notable approved products include patisiran (Onpattro) for hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy, givosiran (Givlaari) for acute hepatic porphyria, lumasiran (Oxlumo) for primary hyperoxaluria type 1 and vutrisiran (Amvuttra) as an additional therapeutic option for ATTR-related disease. The company also maintains a diverse clinical-stage pipeline addressing genetic, cardio-metabolic and liver-related disorders, with programs in various stages of development aimed at expanding the reach of RNAi medicines.
Headquartered in Cambridge, Massachusetts, Alnylam operates internationally and has pursued regulatory approvals and commercial launches in multiple regions, including the United States and Europe. The company supports commercialization through its own commercial organization and through partnerships designed to extend geographic reach and accelerate development. Manufacturing strategy combines in-house capabilities and contract manufacturing to supply both clinical programs and marketed products.
As a commercial-stage, research-driven biotechnology company, Alnylam’s business model emphasizes platform innovation, clinical development and patient access. Its work centers on leveraging RNAi as a modality to address unmet medical needs, particularly in rare genetic diseases, while exploring broader indications where targeted gene silencing can offer therapeutic benefit. The company’s ongoing efforts include advancing late-stage programs, expanding indications for approved medicines and optimizing delivery technologies to broaden the applicability of RNAi therapeutics.