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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.
Takeda Pharmaceutical is Japan's largest pharmaceutical company, with revenue of JPY 4.6 trillion in fiscal 2024. The company's five core therapeutic areas are oncology, gastroenterology, neuroscience, rare diseases, and plasma-derived therapies, which account for more than 80% of revenue. Its geographic footprint is well diversified, with over 50% derived from the US, 20% from Japan, 20% from Europe and Canada.