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CO-2 is an orally active small molecule pharmacological corrector being developed by Congruence Therapeutics for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). Discovered using the proprietary Revenir™ computational platform, the drug targets the SERPINA1 gene product, alpha-1 antitrypsin (A1AT). Its mechanism of action involves stabilizing the protein to increase the secretion of functional AAT into the bloodstream while simultaneously reducing the toxic accumulation of polymerized mutant protein within hepatocytes. This dual approach aims to mitigate both the lung disease (emphysema) caused by AAT deficiency and the liver disease (fibrosis and cirrhosis) caused by protein aggregation. As of 2025, the program is in preclinical development.
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