Alnylam Pharmaceuticals Inc (Alnylam Pharmaceuticals) is a biopharmaceutical company, which focuses on the development and sale of innovative medicines based on RNA interference (RNAi), which is a naturally occurring biological pathway in cells for selectively silencing and regulating the expression of specific genes. The company developing medicines on four therapeutic areas such as genetic medicines; cardio-metabolic diseases; hepatic infectious diseases; and CNS/ocular diseases.

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The company’s R&D focus is on developing its pipeline and advancing its product candidates including partnered programs into later-stage development. Its R&D focuses on developing RNAi products in four areas including genetic medicines, cardio-metabolic disease, hepatic infectious disease, and CNS/ocular diseases. Genetic medicine development pipeline focuses on developing RNAi therapies for the treatment of rare diseases including ATTR amyloidosis, haemophilia and rare bleeding disorders, acute hepatic porphyria, severe PH1, recurrent renal stones, and alpha 1 liver disease; primary hyperoxaluria porphyria, primary hyperoxaluria, hypercholesterolemia, Cardio-metabolic disease development pipeline focuses on developing RNAi therapies for hypercholesterolemia; Hepatic infectious disease development pipeline focuses on developing a pipeline for hepatitis B virus infection. In FY2023, the company spent US$883 million on its R&D activities, which as a percentage of revenue stood at 85.1%.