14 Nov, 2012 Pre-clinical Data with ALN-AAT for the Treatment of Alpha-1 Antitrypsin Deficiency
At the Liver Meeting held this week in Boston, our scientists presented pre-clinical data from our ALN-AAT program for the treatment of liver disease associated with alpha-1 antitrypsin (AAT) deficiency. AAT deficiency is a rare, genetic condition characterized by misfolding of mutant AAT (“Z-AAT”) that causes lung and liver disease.
The new data demonstrated robust RNAi-mediated silencing of AAT liver mRNA and serum protein using an AAT-targeting siRNA formulated in a lipid nanoparticle (LNP). Knockdown of AAT resulted in significant improvements in liver pathology in disease models, and we are very encouraged by these pre-clinical results. The new RNAi therapeutic program represents a novel approach for the treatment of liver disease associated with AAT deficiency.