KRRO-110

KRRO-110 is an investigational RNA editing oligonucleotide therapy developed by Korro Bio. It is the first clinical candidate from Korro’s proprietary OPERA platform, designed to treat Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder caused by mutations in the SERPINA1 gene. The drug uses endogenous adenosine deaminase acting on RNA (ADAR) enzymes to edit mutant SERPINA1 mRNA, aiming to restore production of functional alpha-1 antitrypsin protein and address both liver and lung manifestations of AATD. Preclinical studies demonstrated that KRRO-110 can repair misfolded AAT protein containing the Z mutation and reduce liver aggregates in mouse models. The REWRITE Phase 1/2a clinical trial is ongoing, with interim results expected in late 2025[2][3][5][6][9].