Undisclosed Alpha-1 Antitrypsin Deficiency therapeutic / Prime Medicine - LARVOL DELTA

Prime Medicine Unveils Program for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) (GlobeNewswire) - "Prime Medicine, Inc...unveiled a preclinical program for the treatment of alpha-1 antitrypsin deficiency (AATD), the next program within its liver franchise. Prime Medicine expects to file an investigational new drug (IND) and/or clinical trial application (CTA) in mid-2026....In Prime Medicine’s initial in vivo data, LNP delivery of Prime Editors targeting the Pi*Z (E324K) mutation demonstrated up to 72% precise correction of the SERPINA1 gene in the hepatocytes of fully humanized mice. Importantly, this restored over 95% of serum AAT to the corrected isoform, with healthy AAT (M-AAT) protein in the serum at levels well above 20µM, indicating restoration of M-AAT to normal levels in a humanized mouse model."

IND • New molecule • New trial • Preclinical • Alpha-1 Antitrypsin Deficiency