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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.
News For ALNY From The Last 14 Days
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May 20, 2015
07:04 EDTALNYAlnylam receives orphan drug designation for revusiran
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May 19, 2015
11:03 EDTALNYAlnylam transthyretin amyloidosis treatment designated for FDA orphan status
Alnylam's revusiran was designated by the FDA for orphan drug status as a treatment of transthyretin amyloidosis. Reference Link
07:28 EDTALNYUBS to hold a conference
Global Healthcare Conference is being held in New York on May 18-20 with webcasted company presentations to begin on May 19 at 8 am; not all company presentations may be webcasted. Webcast Link
May 17, 2015
15:24 EDTALNYAlnylam files ALN-AAT trial application, reports positive results in primates
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May 12, 2015
07:52 EDTALNYLeerink to hold a tour
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