New Pre-Clinical Data on ALN-AAT for the Treatment of Alpha-1-Antitrypsin (AAT) Deficiency Liver Disease

New Pre-Clinical Data on ALN-AAT for the Treatment of Alpha-1-Antitrypsin (AAT) Deficiency Liver Disease

We presented new pre-clinical data on 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 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 a transgenic mouse model of Z-AAT protein over-expression, 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.



AAT deficiency liver disease can lead to hepatitis, cirrhosis, and hepatocellular carcinoma, with liver transplantation being the only treatment option currently available. There exists a very high unmet need for therapies toward this disease. We are very encouraged by these new pre-clinical data, as they highlight the potential for ALN-AAT to become an important treatment option for the management of this devastating disease.



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