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May 11, 2014
17:41 EDTALNYAlnylam presents key scientific data on ESC,-GalNAc-conjugate delivery platform
Alnylam Pharmaceuticals announced that it is presenting key scientific data on its Enhanced Stabilization Chemistry, ESC,-GalNAc-conjugate delivery platform. Specifically, the company will be presenting data showing that chemical modifications of siRNA that enhance in vitro stability result in higher liver exposure in vivo and lead to a significantly increased potency and durability of effect in pre-clinical studies. As compared with the “standard template chemistry” ,STC,-GalNAc-conjugate approach used in ALN-TTRsc – a subcutaneously administered RNAi therapeutic targeting transthyretin, TTR, for the treatment of TTR cardiac amyloidosis – ESC-GalNAc-siRNA conjugates demonstrated a 10-fold increased potency in non-human primate, NHP, studies, and a durability of effect that supports once-monthly or potentially even less frequent subcutaneous dosing regimens. The ESC-GalNAc-conjugate technology is being used in a wide range of Alnylam development programs, including ALN-AT3 – an RNAi therapeutic targeting antithrombin, AT, for the treatment of hemophilia and rare bleeding disorders – which is currently in a Phase 1 clinical study.
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