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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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August 27, 2014
08:02 EDTALNYAlnylam receives notice of allowance from USPTO for new patent
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August 21, 2014
09:16 EDTALNYAlnylam to hold a roundtable webinar
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August 20, 2014
08:07 EDTALNYAlnylam receives USPTO notice of allowance for new patent
Alnylam Pharmaceuticals announced that the United States Patent and Trademark Office has issued a Notice of Allowance for claims in the Tuschl et al. patent application 13/725,262. The ’262 patent application includes newly allowed claims directed to compositions that mediate RNAi, and comprising a double-stranded molecule with up to 25 base pairs and at least one nucleotide analogue. Specifically, the newly allowed patent application broadly covers small interfering RNA molecules of various designs, including so-called “dicer substrate” RNAi triggers (Amarzguioui et al., Nat Protoc. 2006;1(2):508-17; Rose et al., Nucleic Acids Res. 2005 Jul 26;33(13):4140-56) and double-stranded, RNAi-mediating molecules containing moieties that include “unlocked nucleobase analogs” amongst other naturally or non-naturally occurring nucleotide analogues. The allowed claims of the Tuschl ’262 patent application, as well as other granted, Alnylam-owned or -licensed patents, in aggregate broadly cover compositions, methods, and uses of RNAi therapeutics.
August 19, 2014
15:50 EDTALNYAlnylam to hold a roundtable webinar
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