A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants with Primary Hyperoxaluria Type 1 (PH1)

A single infusion of the genome-editing therapy ABO-101 aims to shut down a key enzyme in the liver that drives kidney destruction in people with primary hyperoxaluria type 1 (PH1). PH1 is a rare genetic disorder in which the liver overproduces oxalate, a waste chemical that crystallises in the kidneys and urinary tract, causing progressive kidney failure. Current treatments are limited: patients must drink huge volumes of water, take drugs that only modestly reduce oxalate, or receive a combined liver–kidney transplant. ABO-101 targets the *HAO1* gene, which codes for glycolate oxidase, an enzyme on the oxalate production pathway. By editing that gene, the therapy is designed to permanently lower oxalate output. If the trial shows ABO-101 is safe and reduces urinary oxalate excretion, it could offer PH1 patients a one-time treatment that prevents kidney damage without lifelong medication or major surgery. The study enrols participants aged 6 to 64, monitors them for about two years, and follows them for up to 15 years to track long-term safety and efficacy. Success would transform a disease that currently demands constant management into one that might be controlled with a single intervention.