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’s commercial RNAi therapeutic products include Onpattro and Amvuttra (for hATTR amyloidosis), Givlaari (for acute hepatic porphyria), and Oxlumo (for primary hyperoxaluria type 1), all developed and commercialized by Alnylam. Plus, Leqvio (for hypercholesterolemia) and Qfitlia (for hemophilia A or B), which are being commercialized by Alnylam’s partners, Novartis and Sanofi, respectively. It also has several clinical programs across various therapeutic areas, including cardio-metabolic diseases, neuroscience, and hematology.
