KRRO-121

KRRO-121 is a preclinical GalNAc-conjugated RNA-editing oligonucleotide being developed by Korro Bio for the treatment of hyperammonemia in patients with urea cycle disorders (UCD) of any mutational background and hepatic encephalopathy. It is designed using Korro’s OPERA RNA-editing platform to generate a de novo, stabilized variant of a critical enzyme involved in ammonia clearance, thereby activating a biological pathway that enhances ammonia detoxification and reduces hyperammonemic crises.[1][5] The agent is planned for subcutaneous administration and aims to offer a pan-UCD and hepatic encephalopathy therapy that does not depend on the underlying genetic mutation by stabilizing and enhancing activity of an endogenous ammonia-handling enzyme in the liver.[1][5]