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Alnylam Pharmaceuticals (ALNY) Presents New Pre-clinical Data on ALN-AAT, an RNAi Therapeutic Targeting Alpha-1-Antitrypsin (AAT) for the Treatment of AAT Deficiency Liver Disease


11/4/2013 6:38:22 AM

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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development for the treatment of liver disease associated with AAT deficiency. These data were presented in a poster entitled “Developing an RNAi Therapeutic for Liver Disease Associated with Alpha-1-Antitrypsin Deficiency” at the 64th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, “The Liver Meeting”) held November 1 – 5, 2013 in Washington, D.C. AAT deficiency liver disease is a rare genetic disorder, with the most common mutation being in the “Z-allele,” which results in the accumulation of the mutant AAT protein (Z-AAT) in liver tissue with subsequent liver injury, fibrosis, and, in some cases, hepatocellular carcinoma. There are approximately 12,000 people in the U.S. and E.U. with liver pathology associated with AAT deficiency.

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