Alnylam Pharmaceuticals Inc (Alnylam Pharmaceuticals) is a biopharmaceutical company that focuses on developing novel therapeutics based on ribonucleic acid interference (RNAi). RNAi is a biological pathway for gene expression regulation, and its RNAi therapeutics aim to silence messenger RNA (mRNA) to prevent disease-causing proteins from being produced. The company has developed five first-in-class RNAi-based medicines and is advancing over 40 clinical programs across various disease areas. It focuses on therapeutic areas such as genetic medicines, cardio-metabolic diseases, and CNS/ocular diseases. The company operates through a single reporting segment, which focuses on the discovery, development, and commercialization of RNAi therapeutics. It develops RNAi therapeutics for treating genetic diseases, cardio-metabolic diseases, and CNS/Ocular Diseases. Alnylam Pharmaceuticals markets RNA therapies, including Onpattro (patisiran), a lipid complex injection approved in the US and Canada for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults; Onpattro is approved in the European Union (EU), Switzerland, Brazil, and Israel for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy and in Japan for the treatment of transthyretin (TTR) type familial amyloidosis with polyneuropathy. Givlaari (givosiran), an injection approved in the US, Brazil, and Canada for the treatment of acute hepatic porphyria (AHP); and in the EU for the treatment of AHP in adults and adolescents aged 12 years and older. Oxlumo (lumasiran), an injection that targets hydroxyacid oxidase 1 (HAO1), is approved in the US and EU for the treatment of Primary Hyperoxaluria Type 1 (PH1). Amvuttra (vutrisiran), is a treatment for ATTR amyloidosis with cardiomyopathy and hemophilia. Leqvio (inclisiran) is being developed and commercialized by Novartis and given marketing authorization by the European Commission for the treatment of adults with hypercholesterolemia. The company’s pipeline products for treating genetic diseases include patisiran and vutrisiran for ATTR amyloidosis, fitusiran for hemophilia and rare bleeding disorders, cemdisiran for complement-mediated diseases and lumasiran for severe PH1 recurrent renal stones.

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The company is focusing on advancing its RNAi platform and clinical pipeline through significant investments in research and development. The company is progressing late-stage programs, including the Phase 3 TRITON-PN and TRITON-CM clinical trials of nucresiran and the Phase 3 ZENITH cardiovascular outcomes trial of zilebesiran. Additionally, Alnylam is advancing its early-stage clinical pipeline, including CNS and metabolic programs, and is working on delivering RNAi therapeutics to additional tissues and treating new disease areas. The company is also pursuing complementary modalities through business development to expand its efforts in discovering, developing, and commercializing the next wave of RNAi therapeutics. Its R&D unit is responsible for the discovery, development, and supply of products. This unit manages and allocates resources at the global corporate level, enabling the CEO to assess the overall level of resources available and how to best deploy these resources in line with the company's overarching long-term corporate-wide strategic goals. The company has more than 20 clinical programs in its pipeline and has entered into major technology alliances with companies such as Roche, Regeneron, and Novartis. The company has six marketed products, including two commercialized by collaborators. In FY2025, the company spent US$1,319.7 million on its R&D activities, which as a percentage of revenue stood at 35.5%, and grew 17.1% YoY.