CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data on the pharmacology of GalNAc-siRNA conjugates at the 12th US-Japan Symposium on Drug Delivery Systems held December 16 – 20, 2013 in Lahaina, Maui, Hawaii. In a presentation titled “Advances in Systemic Delivery of RNAi Therapeutics,” Alnylam scientists presented new data on tissue drug levels and sustained target knockdown achieved with long-term chronic dosing of GalNAc-siRNA conjugates. GalNAc-siRNA conjugates are a proprietary Alnylam delivery platform, and are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. This targeted delivery platform enables subcutaneous dose administration with a wide therapeutic index, and has demonstrated potent and durable gene silencing, as well as a favorable tolerability profile, in clinical and pre-clinical studies from multiple programs in the company’s “Alnylam 5x15” product pipeline.
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