KRRO-111

KRRO-111 is a proprietary GalNAc-conjugated oligonucleotide developed by Korro Bio as an RNA editing therapeutic for Alpha-1 Antitrypsin Deficiency (AATD). Utilizing the company's proprietary OPERA® (Oligonucleotide Promoted Editing of RNA) platform, KRRO-111 is designed for subcutaneous delivery to hepatocytes. Once inside the cell, the therapeutic co-opts the endogenous Adenosine Deaminase Acting on RNA (ADAR) enzyme to perform site-specific adenosine-to-inosine (A-to-I) editing on the SERPINA1 mRNA. This repair of the pathogenic single nucleotide variant aims to restore the production of functional M-AAT protein, potentially addressing both the pulmonary emphysema and hepatic cirrhosis associated with the disease. Preclinical studies in PiZZ mouse models have demonstrated high levels of transcript editing and significant restoration of functional protein levels.