ALN RNAi therapeutic program represents novel approach for treatment of AAT deficiency

Published on November 15, 2012 at 4:33 AM · No Comments

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.

Liposome mediated delivery of siRNA to hepatic stellate cells”

"In line with our 'Alnylam 5x15' product strategy, we are excited about the potential for RNAi therapeutics for the treatment of AAT deficiency, a rare, genetic condition that causes lung and liver disease," said Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development at Alnylam. "We are very encouraged by these new pre-clinical data with ALN-AAT showing potent knockdown of AAT that results in significant improvements in liver pathology in disease models."

"Approximately 10,000 people worldwide have been diagnosed with severe AAT deficiency, a rare genetic disease that results in severe lung and liver pathology. AAT patients can develop early onset emphysema as well as liver disease, including hepatitis, cirrhosis, and hepatocellular carcinoma. While lung disease associated with AAT deficiency has been addressed with AAT replacement therapy, there are limited treatment options for patients with liver disease," said Jeffrey Teckman, M.D., Professor in the Department of Pediatrics and Director of Gastroenterology and Hepatology at Saint Louis University School of Medicine. "We are very encouraged by these data with ALN-AAT and look forward to further advancement of an RNAi therapeutic approach for the treatment of this genetic disease."

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