Korro Bio, Inc. has announced the dosing of the first cohort of participants in its Phase 1/2 clinical trial, ANCHOR, for the company's investigational drug KRRO-121. The trial is designed to evaluate the safety, tolerability, pharmacokinetics, and clinical activity of KRRO-121 in patients with urea cycle disorders (UCDs) and hepatic encephalopathy (HE).
The ANCHOR program is a global, two-part study. Part 1 consists of a Phase 1 single ascending dose (SAD) study followed by a multiple ascending dose (MAD) study in healthy volunteers. The SAD portion is currently enrolling up to 30 healthy volunteers across five cohorts, while the MAD portion will enroll up to 20 subjects across two cohorts. Both parts are placebo-controlled. The first cohort received a single dose of 2mg/kg of KRRO-121.
Part 2 of the program is a Phase 2 multiple dose study in up to 15 patients with UCDs across all genotypes. Korro expects to begin dosing UCD patients in the first half of 2027, with an interim clinical readout anticipated in the second half of 2027.
KRRO-121 is a GalNAc-conjugated RNA-editing oligonucleotide developed using Korro's proprietary OPERA platform. The therapy is designed to generate a stabilized, de novo glutamine synthetase protein to assist in ammonia clearance. Korro is based in Cambridge, Massachusetts, and its common stock trades on the Nasdaq Capital Market under the symbol KRRO.
