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Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to the exploration, advancement, and marketing of innovative therapies rooted in RNA interference (RNAi) technology. The company's research pipeline for RNAi treatments targets a diverse array of medical conditions, encompassing inherited disorders, cardiovascular and metabolic diseases, hepatic infections, and illnesses affecting the central nervous system (CNS) and eyes. Alnylam's commercialized products include ONPATTRO (patisiran), an injectable lipid complex prescribed for adults experiencing polyneuropathy due to hereditary transthyretin-mediated amyloidosis. Also marketed are GIVLAARI, used to treat acute hepatic porphyria (AHP) in adult patients, and OXLUMO (lumasiran), indicated for primary hyperoxaluria type 1 (PH1). In terms of its developmental programs, the company is progressing several candidates: Givosiran, for the treatment of AHP in adolescent patients. Patisiran, being investigated for transthyretin amyloidosis (ATTR amyloidosis) when accompanied by cardiomyopathy. Cemdisiran, aimed at complement-mediated diseases. ALN-AAT02, for liver disease associated with AAT deficiency. ALN-HBV02, targeting chronic hepatitis B virus infection. Zilebesiran, for the management of hypertension. ALN-HSD, for non-alcoholic steatohepatitis (NASH). Fitusiran, addressing hemophilia and various rare bleeding disorders. Inclisiran, developed to treat hypercholesterolemia. Lumasiran, for advanced PH1 and recurrent kidney stones. Vutrisiran, currently in Phase 3 clinical trials for ATTR amyloidosis. Alnylam Pharmaceuticals maintains strategic alliances with several key players in the industry. It collaborates with Regeneron Pharmaceuticals, Inc. on the discovery, development, and commercialization of RNAi therapeutics for ocular and CNS targets. Another significant partnership exists with Sanofi Genzyme, focusing on the broader development and commercialization of RNAi treatments. Furthermore, the company holds licensing and collaboration agreements with Novartis AG, Vir Biotechnology, Inc., Dicerna Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., and PeptiDream, Inc. The company was established in 2002 and operates from its headquarters in Cambridge, Massachusetts.
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