Alnylam Pharmaceuticals Initiates Phase 1 Clinical Trial With ALN-AT3, a Subcutaneously Administered RNAi Therapeutic Targeting Antithrombin (AT) In Development For The Treatment Of Hemophilia And Rare Bleeding Disorders (RBD)
Published: Jan 22, 2014
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated a Phase 1 study with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD). ALN-AT3 has demonstrated efficacy in animal models of hemophilia, including in non-human primate models of induced hemophilia. Moreover, pre-clinical studies of ALN-AT3 support a wide therapeutic index in the hemophilia setting. ALN-AT3 is a key program in the company’s “Alnylam 5x15” product strategy, which is aimed at advancing multiple RNAi therapeutic genetic medicine programs into clinical development.
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