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Alnylam Pharmaceuticals (ALNY) and Collaborators Publish Clinical Trial Results With RNAi Therapeutics Targeting Transthyretin (TTR) for the Treatment of TTR-Mediated Amyloidosis (ATTR) in The New England Journal of Medicine


8/29/2013 10:16:58 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the publication of complete study results from Phase I trials with ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine. The paper, titled “Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis” (Coelho et al., N Engl J Med 2013;369:819-29), shows that RNAi therapeutics targeting transthyretin (TTR) achieved rapid, dose-dependent, durable, and specific knockdown of TTR, the disease-causing protein in TTR-mediated amyloidosis (ATTR). These results document the most robust proof of concept for RNAi therapy in man to date, including knockdown of serum TTR protein levels of up to 94% after just a single dose of drug.

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