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 its RNAi therapeutic for the treatment of hemophilia at the World Federation of Hemophilia World Congress being held July 8-12, 2012 in Paris. Alnylam scientists presented data comparing robust RNAi-mediated silencing of protein C (PC) and antithrombin (AT), two natural anticoagulant proteins. Studies showed that AT, an endogenous inhibitor of thrombin generation, is the optimal anticoagulant target and Alnylam has now designated ALN-AT3, an RNAi therapeutic targeting AT, as the development candidate for its hemophilia program. ALN-AT3 utilizes Alnylam’s proprietary GalNAc-siRNA conjugate delivery approach enabling subcutaneous administration with the potential for a once-weekly or twice-monthly dosing regimen. In pre-clinical animal models of hemophilia, administration of ALN-AT3 resulted in increased thrombin generation demonstrating proof of concept for this novel strategy. Alnylam expects to file an investigational new drug (IND) application for ALN-AT3 in 2013.
