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Alnylam Pharmaceuticals (ALNY) Presents New Pre-clinical Data on ALN-AT3, an RNAi Therapeutic Targeting Antithrombin (AT) for the Treatment of Hemophilia and Rare Bleeding Disorders


7/2/2013 8:40:17 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders. The data were presented at the XXIV Congress of the International Society on Thrombosis and Haemostasis (ISTH) being held June 29 – July 4, 2013 in Amsterdam. The new pre-clinical data demonstrate that ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), can normalize thrombin generation and improve hemostasis in hemophilia mice and fully correct thrombin generation in a non-human primate (NHP) hemophilia “inhibitor” model. ALN-AT3 is a key program in the company’s “Alnylam 5x15” product strategy, which is aimed at advancing five RNAi therapeutic programs directed toward genetically validated disease targets into clinical development – including programs in advanced stages – by the end of 2015. ALN-AT3 utilizes the company’s proprietary GalNAc conjugate delivery platform which enables subcutaneous dose administration.

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