NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals announced this week that it has formally added ALN-AAT, a subcutaneously delivered therapy for alpha-1 antitrypsin (AAT) deficiency-associated liver disease, to its drug-development pipeline.
AAT deficiency-associated liver disease is caused by accumulation of mutant AAT protein in liver tissue with subsequent liver injury, fibrosis, cirrhosis, and potentially hepatocellular carcinoma, according to the company.
Recently released preclinical data showed that ALN-AAT triggered rapid, potent, dose-dependent, and durable knockdown of mutant AAT protein in the livers of transgenic mouse models, while improving liver histopathology. Treatment also resulted in significant improvements in liver pathology and function, along with reductions in the incidence of liver tumors.
Alnylam said that it anticipates filing an investigational new drug application for ALN-AAT in mid-2015. The drug is one of the company's genetic medicines programs and covered by its recently announced partnership with Genzyme.