Alnylam Pharmaceuticals is a leader in the study of RNA interference (RNAi) therapeutics. RNAi is a naturally occurring biological pathway within cells for sequence-specific silencing and regulation of gene expression. Alnylam has five drugs on the market: Onpattro and Amvuttra for hATTR amyloidosis, Givlaari for acute hepatic porphyria, Oxlumo for primary hyperoxaluria type 1, and Leqvio for hypercholesterolemia. It also has several clinical programs across various therapeutic areas: genetic medicines; cardio-metabolic diseases; hepatic infectious diseases, and CNS/ocular diseases. Upfront fees from research partnerships have boosted Alnylam's cash levels, and the company stands to see additional milestones and royalties from drugs commercialized under collaboration partnerships.
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