Alnylam Pharmaceuticals (NASDAQ:ALNY) is a biopharmaceutical company pioneering the development of ribonucleic acid interference (RNAi) therapeutics. Leveraging its proprietary RNAi delivery platforms, the company designs small interfering RNA (siRNA) molecules to selectively silence disease-causing genes. Headquartered in Cambridge, Massachusetts, Alnylam aims to transform the treatment landscape for a range of rare genetic, cardio-metabolic, and hepatic infectious diseases by addressing root genetic causes rather than symptoms alone.
The company’s commercial portfolio includes several approved products. ONPATTRO® (patisiran) was the first FDA-approved RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR), offering patients an option to slow disease progression. GIVLAARI® (givosiran) targets acute hepatic porphyria (AHP), providing targeted enzyme silencing to reduce the frequency of life-threatening attacks. More recently, Alnylam launched OXLUMO® (lumasiran) for primary hyperoxaluria type 1 (PH1) and AMVUTTRA™ (vutrisiran) for the polyneuropathy of hATTR amyloidosis, both employing advanced GalNAc conjugate technology for subcutaneous delivery and improved dosing convenience.
Beyond its marketed therapies, Alnylam maintains a robust pipeline spanning Phase 1 through Phase 3 development. Key programs include candidates targeting high-cholesterol conditions, chronic liver diseases, and viral infections. The company partners with major pharmaceutical firms, including Sanofi, Roche, and Vir Biotechnology, to advance RNAi research across diverse therapeutic areas. Alnylam’s research centers in the United States and Europe support innovation in RNAi chemistry, delivery approaches, and biomarker development.
Founded in 2002 by researchers from the Massachusetts Institute of Technology, Alnylam has grown under the leadership of industry veterans and scientists dedicated to translating gene-silencing science into medicines. Under the guidance of President and Chief Executive Officer Yvonne Greenstreet and a leadership team with deep experience in drug development and commercialization, Alnylam continues to expand its global footprint, with commercial operations and clinical trials conducted across North America, Europe, Asia, and Latin America.
