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Alnylam

Archives: Publications

April 13, 2015

Pre-Clinical Results with ALN-AT3 Published in “Nature Medicine”

We have published pre-clinical results with ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia, in Nature Medicine. The paper documents a broad set of pre-clinical data supporting the clinical advancement of ALN-AT3. Among the many findings reported, subcutaneous administration of ALN-AT3 led to potent, dose-dependent, and durable knockdown of AT Read More

January 10, 2015

Welcome to Alnylam

Alnylam is developing an entirely new class of medicines based on the breakthrough discovery in biology known as RNA interference, or RNAi. Alnylam believes that RNAi therapeutics are a promising approach to silence disease-causing genes, and represent an opportunity to transform the treatment of many diseases.

December 1, 2014

New Paper Documenting Alnylam’s Pioneering Discovery of GalNAc-Conjugated siRNA

We have published an article in the Journal of the American Chemical Society describing the discovery of GalNAc-conjugated siRNA as a novel strategy for delivery of RNAi therapeutics. This publication as a JACS “Communication” documents the landmark discovery by our scientists of GalNAc-conjugates as a potent and durable approach for subcutaneous administration of RNAi therapeutics Read More

May 21, 2014

Pre-Clinical Results with RNAi Therapeutics Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) Published in Proceedings of the National Academy of Sciences (PNAS)

We have published pre-clinical results with RNAi therapeutics targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic porphyrias in the Proceedings of the National Academy of Sciences (PNAS). The new paper highlights work that was done in collaboration with researchers at the Icahn School of Medicine at Mount Sinai in New York City. Data Read More

December 23, 2013

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 Read More

November 16, 2013

ALN-ANG Historical Press Releases

For your convenienence we have collected a list of historical press releases that cover the historical progess of our clinical program for mixed hyperlipidema and severe hypertriglyceridemia 2013

October 3, 2013

Positive Phase I Clinical Trial Results with ALN-PCS Published in “The Lancet”

We have published complete study results from our Phase I trial with ALN-PCS in The Lancet. As reported in the paper, ALN-PCS administration resulted in a rapid, dose-dependent reduction in plasma PCSK9 of up to 84% relative to baseline and placebo, with a corresponding reduction in serum levels of low-density lipoprotein cholesterol (LDL-C) – or Read More

September 14, 2013

Safety and Efficacy of RNAi Therapy of RNAi Therapy for Transthyretin Amyloidosis

Coelho et al., NEJM. 2013 Sept13 (Epub doi: 10.1056/NEJMoa1208760 )

August 28, 2013

Positive Clinical Trial Results with ALN-TTR01 and ALN-TTR02 Published in the “New England Journal of Medicine”

We have published complete study results from Phase I trials with ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine. RNAi therapeutics targeting transthyretin (TTR) achieved rapid, dose-dependent, durable, and specific knockdown of TTR, the disease-causing protein in TTR-mediated amyloidosis (ATTR). The Phase I studies of ALN-TTR01 and ALN-TTR02 demonstrate key human proof of Read More

June 25, 2013

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

Maier et al., Mol Ther. 2013 Jun25 (Epub doi: 10.1038/mt.2013.124)