Alnylam launches trial of one of its RNAi candidates

RNAi specialist Alnylam ($ALNY) initiated Phase I/II trials of its candidate to treat AAT deficiency-associated liver disease (alpha-1 liver disease). "There is great unmet need for any specific treatment for patients with alpha-1-antitrypsin deficiency and liver disease, which is caused by misfolding of the mutated Z-antitrypsin protein; accumulation of the misfolded protein in liver cells causes irreversible hepatocyte damage which leads to advanced liver disease in many. Alpha-1 liver disease can manifest as cholestasis, chronic hepatitis, cirrhosis and hepatocellular carcinoma, and occurs in both children and adults where it is currently managed with supportive care or, in the case of liver failure, with liver transplantation," said Professor Graeme Alexander of the U.K's Cambridge University Hospitals. More

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