Alnylam Pharmaceuticals Files Clinical Trial Application to Initiate a Phase I Study for ALN-AT3, a Subcutaneously Administered RNAi Therapeutic Targeting Antithrombin (AT) for the Treatment of Hemophilia A and B

Published: Oct 29, 2013

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase I clinical trial with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia, including people with “inhibitors.” Hemophilia is a hereditary disorder caused by genetic deficiencies in various blood clotting factors, resulting in recurrent bleeds into joints, muscles, and other major internal organs. By knocking down AT, ALN-AT3 administration is expected to increase thrombin generation and reduce disease burden, including annualized bleeding rate, severity of bleeding, and requirement for replacement factor or bypass agent, and to potentially improve quality of life. ALN-AT3 is a key program in the company’s “Alnylam 5x15” product development and commercialization strategy, in which the company aims to advance five RNAi therapeutic programs toward genetically validated disease targets into clinical development, including programs in advanced stages, by the end of 2015.

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