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 four drugs on the market: Onpattro for hATTR amyloidosis, Givlaari fo...
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 four drugs on the market: Onpattro for hATTR amyloidosis, Givlaari for acute hepatic porphyria, Oxlumo for primary hyperoxaluria type 1, and Leqvio for hypercholesterolemia. The firm also has 12 clinical programs across the following therapeutic areas: genetic medicines; cardio-metabolic diseases; hepatic infectious diseases, and CNS/ocular diseases. Additionally, up-front 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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