Alnylam Pharmaceuticals Inc (Alnylam Pharmaceuticals) is a biopharmaceutical company, which focuses on developing novel therapeutics based on ribonucleic acid interference (RNAi). RNAi is a biological pathway for gene expression regulation, and its RNAi therapeutics aim to silence messenger RNA (mRNA) to prevent disease-causing proteins from being produced. The company has developed five first-in-class RNAi-based medicines and is advancing over 20 clinical programs across various disease areas. It focuses on therapeutic areas such as genetic medicines; cardio-metabolic 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, and CNS/ocular diseases. It utilizes N-acetylgalactosamine (GalNAc) conjugate and lipid nanoparticle (LNP) approaches for hepatic delivery of siRNAs, and an alternative conjugate approach based on a hexadecyl (C16) moiety for delivery to the central nervous system and the eye. The company is also advancing delivery approaches for heart, skeletal muscle, and adipose tissue. 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 and hypercholesterolemia. Cardio-metabolic disease development pipeline focuses on developing RNAi therapies for hypercholesterolemia. In FY2024, the company spent US$1,126.2 million on its R&D activities, which as a percentage of revenue stood at 50.1%, and grew 12.1% YoY.