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Alnylam Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company. The Company is engaged in developing therapeutics based on ribonucleic acid interference (RNAi). Its pipeline includes five marketed products and over ten clinical programs, including several in late-stage development, across four strategic therapeutic areas (STArs): genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and CNS/ocular diseases.
Its RNAi-based medicines include ONPATTRO (patisiran), AMVUTTRA (vutrisiran), GIVLAARI (givosiran), OXLUMO (lumasiran) and Leqvio (inclisiran). ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin amyloidosis. GIVLAARI is used to reduce induced liver aminolevulinic acid synthase 1 messenger RNA (mRNA), to reduce toxins associated with attacks and other disease manifestations of acute hepatic porphyria. OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 for the treatment of primary hyperoxaluria type 1. .