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Alnylam Presents New Pre-clinical Data On ALN-AAT, An RNAi Therapeutic Targeting Alpha-1-Antitrypsin (AAT) For The Treatment Of AAT Deficiency Liver Disease

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 64 th 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.

Alnylam scientists and collaborators presented new results in a transgenic mouse model of Z-AAT protein over-expression showing that subcutaneous administration of a GalNAc-siRNA targeting AAT led to rapid, potent, dose-dependent, and durable knockdown of AAT of greater than 90%, as well as a significant reduction in fibrosis and the incidence of liver tumors. ALN-AAT is a program in the company’s “Alnylam 5x15” product development and commercialization strategy, by 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.

“We are excited about the potential for RNAi therapeutics for the treatment of liver disease associated with AAT deficiency, a rare genetic disorder. AAT deficiency liver disease is caused by the mutant ‘Z allele’ of the AAT gene, whose protein misfolds, accumulates in liver cells, and causes cellular damage. Our therapeutic hypothesis is that an RNAi therapeutic that silences the mutant Z-AAT will prevent the development and/or progression of liver disease,” said Rachel Meyers, Ph.D., Vice President of Research and RNAi Lead Development at Alnylam. “Our new pre-clinical data with a GalNAc-siRNA conjugate against AAT demonstrate rapid, potent, dose-dependent, and durable knockdown of AAT to greater than 90% resulting in a significant reduction of liver fibrosis and tumor formation. If these results extend in clinical studies, we believe that ALN-AAT could become an important treatment option for the management of disease in people with AAT deficiency.”

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