Alnylam Pharmaceuticals this week presented preclinical data on its investigational treatment for alpha-1 antitrypsin deficiency-associated liver disorders.
AAT is a liver-produced serine proteinase inhibitor that protects the lungs from neutrophil elastase and other irritants that cause inflammation, according to Alnylam. In the liver, misfolding of the mutant form of the protein disrupts its normal release into the blood, causing it to aggregate in hepatocytes. The result can be liver injury, fibrosis, cirrhosis, and liver cancer.
Alnylam's drug, called ALN-AAT, is designed to inhibit AAT. In a mouse model triggered rapid, potent, dose-dependent, and durable knockdown of its target of greater than 90 percent, Alnylam said. A significant reduction in fibrosis and the incidence of liver tumors was also observed.
Alnylam, which presented these data at the annual meeting of the American Association for the Study of Liver Diseases, has previously said that it will only advance ALN-AAT into human testing with a partner.