At Alnylam Pharmaceuticals, we have advanced two delivery platforms for RNA interference (RNAi) based human therapeutics for liver-based disease molecular targets. The first one is lipid nanoparticles (LNPs) formulation containing siRNAs and used for intravenous administration. The second one, in which siRNAs are conjugated to trivalent GalNAc sugar to target asialoglycoprotein receptor (ASGPR) of hepatocytes are emerging as a potential new class of medicine supporting a broad clinical pipeline across multiple therapeutic targets by subcutaneous administration. Furthermore, we have been able to continuously optimize the siRNA chemical modifications and design resulting in the Enhanced Stabilization Chemistry (ESC) platform exhibiting improved efficacy and extended duration lasting for several months. Our progress in these areas and applications will be presented.

Advances in RNAi therapeutics: Chemistry, mechanism and applications

253rd National Meeting (2017)

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