Business Summary

Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company. It is developing therapeutics based on ribonucleic acid interference (RNAi). Its pipeline includes four products and multiple late and early-stage investigational RNAi therapeutics, that is focused in four strategic therapeutic areas (STArs): Genetic Medicines; Cardio-Metabolic Diseases; Hepatic Infectious Diseases; and CNS/Ocular Diseases. Its RNAi-based medicines include ONPATTRO (patisiran), 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. Leqvio is used for the treatment of adults with hypercholesterolemia.