Alpha-1 antitrypsin deficiency (AATD) is a genetic condition caused by changes in the SERPINA1 gene, which plays a role in creating alpha-1 antitrypsin (A1AT), a protein that is normally made by the liver. In some people, the protein does not fold properly and builds up in the liver, where it can cause progressive damage.

Learn more about our active alpha-1 antitrypsin deficiency clinical trials below.