- Potential best-in-class therapeutic delivered subcutaneously (GalNAc-conjugated oligonucleotide) for a debilitating genetic condition impacting both the liver and lungs
- Demonstrated >90% editing of SERPINA1 transcript in vivo translating into ~90% repaired functional alpha-1 antitrypsin (AAT) protein in plasma in a mouse model of AATD
- Demonstrated a near elimination of active Z-protein production while showing progressive clearance of pre-existing aggregates in a mouse model of AATD
- Pre-clinical data highlighted the possibility of repeat dose therapy to achieve the functional equivalent of a DNA modification without altering the genome
CAMBRIDGE, Mass., May 19, 2026 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (NASDAQ:KRRO), a biopharmaceutical company leveraging a novel Oligonucleotide Promoted Editing of RNA (OPERA®) platform to develop a new class of genetic medicines for rare and highly prevalent diseases, today announced the addition of KRRO-111 for the potential treatment of AATD to its pipeline of therapeutic programs in development. KRRO-111 is a proprietary GalNAc-conjugated oligonucleotide that is delivered subcutaneously to the liver cells, where it is engineered to co-opt the hepatocytes' endogenous Adenosine Deaminase Acting on RNA (ADAR) enzyme and repair a pathogenic single nucleotide variant (SNV) on AAT mRNA to restore production of normal AAT protein. This therapeutic profile along with KRRO-111's rapid onset of action and titratable dosing to treat this disorder, position the compound as a potential best-in-class candidate.