01 Dec, 2014 New Paper Documenting Alnylam’s Pioneering Discovery of GalNAc-Conjugated siRNA
We have published an article in the Journal of the American Chemical Society describing the discovery of GalNAc-conjugated siRNA as a novel strategy for delivery of RNAi therapeutics. This publication as a JACS “Communication” documents the landmark discovery by our scientists of GalNAc-conjugates as a potent and durable approach for subcutaneous administration of RNAi therapeutics with a wide therapeutic index.
GalNAc-siRNA conjugates are a clinically validated, proprietary Alnylam delivery platform and are designed to achieve targeted delivery of RNA therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor (ASGPR). We believe that this approach has emerged as the optimal strategy across the entire industry for RNA therapeutics targeting hepatocyte-expressed disease genes. We’re proud to have pioneered this important innovation in the RNA therapeutics field, and we look forward to realizing the full impact of our science on the treatment of human disease.