Alnylam Presents Preclinical Data on AAT Deficiency Drug | GenomeWeb

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.

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