A preclinical study assessing Arrowhead Pharmaceuticals' (NASDAQ:ARWR) RNAi therapeutic ARO-AAT in a mouse model of alpha-1 antitrypsin deficiency (AATD) showed a positive effect. The data were presented at The International Liver Congress in Vienna.
Based on 33 weeks of treatment, ARO-AAT reduced a protein called Z-AAT which reversed the AATD disease phenotype, prevented inflammation and prevented/reduced fibrosis, oxidative stress and apoptosis (in addition to additional effects).
The company has completed a Phase 1 trial and plans to initiate a Phase 2/3 registrational study.
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