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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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February 27, 2015
09:58 EDTALNYOn The Fly: Analyst Initiation Summary
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06:59 EDTALNYAlnylam initiated with an Outperform at Cowen
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February 23, 2015
07:22 EDTALNYSunTrust to hold a conference
2015 Biotechnology and Pharmaceutical 1:1 Orphan Drug Day is being held in New York on February 23.

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