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CGX-195 is an oral small-molecule pharmacological corrector designed to treat alpha-1 antitrypsin deficiency (AATD). Developed by Congruence Therapeutics using its proprietary Revenir computational drug discovery platform, CGX-195 targets mutant alpha-1 antitrypsin (A1AT, encoded by the SERPINA1 gene), specifically stabilizing the misfolded Z-AAT protein. By preventing its polymerization and accumulation in hepatocytes, CGX-195 aims to mitigate liver fibrosis while simultaneously increasing the secretion of functional A1AT into the plasma to protect against lung emphysema.
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