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Scientific Leadership

These are exciting times for RNAi therapeutics.  Important scientific and clinical advancements are being made at a very rapid pace. The discovery of RNAi has been widely acknowledged as a major breakthrough in biology, and the belief that this technology has the potential to make a broad and significant impact in medicine is stronger than ever.   Alnylam is leading the effort in translating the science of RNAi into a sustainable product pipeline of innovative medicines.

At Alnylam, we are unwavering in our commitment to scientific excellence, and we are driven by our dedication to the highest scientific standards. A testament to this underlying tenet at Alnylam is the publication of our research in world-leading, peer-reviewed scientific journals, such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet, amongst others.  We believe our numerous publications – numbering over 200 to date – and collaborations with some of the world’s best research labs are important measures of our position as the scientific and clinical leaders in the RNAi
field. Our scientific and clinical papers also serve to demonstrate to leading academic scientists, clinicians, and pharmaceutical executives the potential impact of our discoveries and the opportunity for RNAi therapeutics to become a new therapeutic approach in medicine, in addition to Alnylam’s commitment to and leadership in translating and advancing the field.

Summer RNAi Roundtable Series

We hosted a series of online “RNAi Roundtables” during July and August, at which Alnylam scientists and key clinical collaborators reviewed recent progress from several “Alnylam 5×15” programs and discussed the related disease areas.

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Key Scientific Data on Enhanced Stabilization Chemistry (ESC)-GalNAc-Conjugate Technology

Key Scientific Data on Enhanced Stabilization Chemistry (ESC)-GalNAc-Conjugate Technology

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.



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Novel Method for Detection of Tissue-Specific, RNAi-Mediated Gene Silencing Published in “RNA”

Novel Method for Detection of Tissue-Specific, RNAi-Mediated Gene Silencing Published in “RNA”

We have published a paper in the journal RNA describing a novel method that enables the quantification of circulating messenger RNA (mRNA) and micro-RNA (miRNA) as a way of monitoring tissue-specific RNA silencing, and demonstrating RNAi mechanism of action.  The method, called circulating Extracellular RNA Detection (cERD), could have broad applicability in clinical studies since it allows for monitoring of tissue-specific mRNA levels using a non-invasive technique.



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