Alnylam Pharmaceuticals Presents New Pre-Clinical Data on RNAi Therapeutics for the Treatment of Alpha-1 Antitrypsin (AAT) Deficiency
Published: Nov 14, 2012
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) , a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of liver disease associated with AAT deficiency. These data were presented at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, “The Liver Meeting”) held November 9-13, 2012 in Boston. AAT deficiency is a rare genetic disease that can result in severe lung and liver pathology; approximately 10,000 patients are diagnosed worldwide. In a presentation titled “Developing an RNAi Therapeutic for Liver Disease Associated with Alpha-1 Antitrypsin Deficiency,” Alnylam scientists presented results showing robust RNAi-mediated silencing of AAT liver mRNA and serum protein in a transgenic mouse model of mutant AAT (“Z-AAT”) protein overexpression. The new RNAi therapeutic program, ALN-AAT, represents a novel approach for the treatment of liver disease associated with AAT deficiency.