Bond.az reports that Korro Bio Inc. (NASDAQ:KRRO) announced the addition of KRRO-111 to its pipeline for the potential treatment of alpha-1 antitrypsin deficiency (AATD).
KRRO-111 is a GalNAc-conjugated oligonucleotide delivered subcutaneously to liver cells, designed to repair a pathogenic single nucleotide variant on AAT mRNA to restore production of normal AAT protein.
AATD is a genetic disorder caused by a missense mutation in the SERPINA1 gene, leading to pulmonary emphysema and hepatic cirrhosis in adults. Over 95% of severe cases are homozygous for the PiZ mutation, affecting an estimated 3.4 million people worldwide.
The only FDA-approved treatment for AATD is augmentation therapy, a weekly infusion of plasma-derived AAT protein.
In preclinical studies, KRRO-111 demonstrated over 90% editing of AAT transcripts in liver cells, translating to approximately 90% repaired functional AAT protein in plasma in a mouse model.
A repeat-dose study in PiZZ mice at 3 mg/kg reduced non-inclusion Z-AAT by approximately 95% versus vehicle at days 28 and 56, with pre-existing aggregates progressively cleared.
The company plans to present additional data at a future scientific meeting. Korro Bio also advances KRRO-121 for hyperammonemia in urea cycle disorders.
