Scientific Leadership

We are presenting key scientific data on our Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform at the TIDES 2014 meeting held May 12 – 15, 2014 in Providence, Rhode Island. This technology enables subcutaneous dosing of RNAi therapeutics with increased potency and durability, and a wide therapeutic index.  Data show 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 our ALN-TTRsc program for the treatment of transthyretin (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 possibly even less frequent subcutaneous dosing regimens. [spotlight-link icon="release" href="" type=" "] Read the press release[/spotlight-link] [spotlight-link icon="presentation" href="" type="(1.3 MB PDF)"] View our presentation [/spotlight-link]

On July 11, we hosted an R&D Day in New York City. Alnylam scientists and management reviewed progress with our Alnylam 5x15 pipeline for the development and commercialization of RNAi therapeutics. The event featured presentations by experts in the following disease areas: TTR-Mediated Amyloidosis, Hemophilia and Rare Bleeding Disorders, and Acute Intermittent Porphyria. [spotlight-link icon="podcast" href="" type=" "] Listen to the webcast replay[/spotlight-link] [spotlight-link icon="presentation" href="" type="(3.4 MB PDF)"] View the complete presentation[/spotlight-link] TTR-Mediated Amyloidosis [spotlight-link icon="podcast" href="" type="(24.7 MP3)"] Listen to Dr. Philip Hawkins discuss TTR-Mediated Amyloidosis[/spotlight-link][spotlight-link icon="presentation" href="" type="(1.8 MB PDF)"] View the presentation[/spotlight-link][spotlight-link icon="podcast" href="" type="(25.4 MP3)"] Listen to the ALN-TTR program overview[/spotlight-link][spotlight-link icon="presentation" href="" type="(0.6 MB PDF)"] View the presentation[/spotlight-link] Hemophilia and Rare Bleeding Disorders (RBD) [spotlight-link icon="podcast" href="" type="(28.6 MP3)"] Listen to Dr. Craig Kessler discuss Hemophilia and RBDs[/spotlight-link][spotlight-link icon="presentation" href="" type="(0.8 MB PDF)"] View the presentation[/spotlight-link]


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