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Korro Selects KRRO-111 as Development Candidate for the Potential Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
Potential best-in-class therapeutic delivered subcutaneously (GalNAc-conjugated oligonucleotide) for a debilitating genetic condition impacting both the liver
About this update from Korro Bio, Inc.
[{"type":"text","content":"Potential best-in-class therapeutic delivered subcutaneously (GalNAc-conjugated oligonucleotide) for a debilitating genetic condition impacting both the liver and lungsDemonstrated >90% editing of SERPINA1 transcript in vivo translating into ~90% repaired functional alpha-1 antitrypsin (AAT) protein in plasma in a mouse model of AATDDemonstrated a near elimination of active Z-protein production while showing progressive clearance of pre-existing aggregates in a mouse model of AATDPre-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. \"The addition of KRRO-111 for AATD to our pipeline of transformative therapeutic candidates is the culmination of extensive and robust pre-clinical optimization, achieving the highest level of SERPINA1 editing reported to date using RNA editing. It appears to be a level of highly specific protein correction that functionally approaches what would be expected from a permanent specific DNA modification, while preserving the inherent safety advantages of working at the RNA level,\" commented Ram Aiyar, Ph.D., Chief Executive Officer and President of Korro Bio. \"For patients living with AATD, this represents a meaningful step toward a therapy designed to address the root cause of their disease. This is an important clinical and corporate development milestone for the Company as it highlights the...