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Alnylam Pharmaceuticals (ALNY) Advances Development Candidate For ALN-AAT, A Subcutaneously Administered RNAi Therapeutic Targeting Alpha-1 Antitrypsin (AAT) In Development For The Treatment Of AAT Deficiency-Associated Liver Disease


5/6/2014 10:27:17 AM

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate (DC) for ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development for the treatment of AAT deficiency-associated liver disease. New pre-clinical data were presented in a Late-Breaking Abstract Session at Digestive Disease Week (DDW), held May 3 – 6, 2014 in Chicago, Illinois. ALN-AAT is one of Alnylam’s genetic medicine programs, which are RNAi therapeutics directed toward genetically defined targets for the treatment of diseases with high unmet medical need. AAT deficiency-associated liver disease is caused by accumulation of mutant AAT protein (“Z-allele” or “Z-AAT”) in liver tissue with subsequent liver injury, fibrosis, cirrhosis, and, in some cases, hepatocellular carcinoma. It is estimated that approximately 12,000 people with AAT deficiency in the U.S. and E.U. have associated liver pathology. The company now plans to initiate IND-enabling studies with the goal of filing an IND or IND equivalent for ALN-AAT in mid-2015.

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