Korro Bio announced positive preclinical results for KRRO-111, its development candidate for treating Alpha-1 Antitrypsin Deficiency (AATD). Data from mouse studies suggest the subcutaneously administered RNA-editing therapy has the potential to address both the lung and liver manifestations of the disease by restoring functional protein and reducing toxic protein accumulation.

Key Details

  • Efficacy in Mouse Models: In repeat-dose studies, KRRO-111 achieved over 95% SERPINA1 mRNA editing, increased total AAT levels by approximately 6-fold, and reduced pathogenic Z-AAT protein in the liver by up to 95%.
  • Therapeutic Approach: The therapy uses an RNA editing approach to correct the pathogenic SERPINA1 transcript, enabling the production of functional M-AAT while simultaneously reducing the synthesis of mutant Z-AAT that causes liver damage.
  • Clinical Timeline: The company expects to make a regulatory submission and advance KRRO-111 into clinical trials in the first half of 2027.