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Arrowhead Begins Phase I Trial of AAT Deficiency Drug


NEW YORK (GenomeWeb) – Arrowhead Research today announced that it has begun dosing in a Phase I trial of its siRNA-based alpha-1 antitrypsin (AAT) deficiency therapy ARC-AAT.

AAT deficiency is a genetic disease that leads to decreased activity of the protease inhibitor alpha-1 antitrypsin in the blood and lungs causing lung dysfunction. It can also feature the accumulation of a mutant AAT protein known as Z-AAT in liver tissue, which leads to liver injury, fibrosis, cirrhosis, and potentially liver cancer. ARC-AAT is designed to lower production of Z-AAT.

The trial is testing single doses of the drug, first in healthy volunteers and then in AAT deficient patients. Its objectives are to evaluate the compound pharmacokinetics and determine its effects on circulating levels of AAT.

Initial data from the study are expected later this year.