(RTTNews) - Korro Bio Inc. (KRRO), a biopharmaceutical company, has selected KRRO-111 as a development candidate for clinical advancement in the potential treatment of alpha-1 antitrypsin deficiency.
Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder that causes the depletion of protein in the lungs and liver, leading to pulmonary emphysema and/or hepatic cirrhosis. Affecting nearly 3.4 million people worldwide, the only current FDA-approved treatment for AATD is a once-weekly infusion of human plasma-derived AAT protein called augmentation therapy.
KRRO-111 is a proprietary, GaINAc-conjugate oligonucleotide designed for subcutaneous delivery to liver cells.
In preclinical studies, the drug has demonstrated successful RNA-editing by more than 90% in cells, correcting AAT transcription in liver cells. In mice models, the production of circulating M-AAT protein indicates the potential to improve lung and liver manifestations of the disease.
The company plans to present data on the preclinical validation of KRRO-111 as a candidate in treating AATD.
KRRO is currently trading at $9.32, up 1.86%.
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