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New Alnylam Pharmaceuticals (ALNY) Pre-Clinical Results On ALN-CC5, An RNAi Therapeutic Targeting Complement Component C5 For The Treatment Of Complement-Mediated Diseases, Demonstrate Up To 98% Serum C5 Knockdown And Up To 94% Inhibition Of Hemolytic Activity In Non-Human Primates


12/9/2013 9:37:28 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 with ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. These data were presented at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 – 10, 2013 in New Orleans, and demonstrate that subcutaneous administration of ALN-CC5 in non-human primates (NHPs) led to an up to 98% knockdown of serum C5 and an up to 94% inhibition of hemolytic activity. Alnylam believes that ALN-CC5 – part of the company’s “Alnylam 5x15” product strategy – represents a novel approach for the treatment of complement-mediated diseases, with a potentially competitive profile compared with intravenously administered anti-C5 monoclonal antibody therapy. In addition, Alnylam presented two separate posters with new pre-clinical data on ALN-TMP, an RNAi therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders.

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