Learn about
an investigational clinical study
for people living with
Primary Hyperoxaluria Type 1 (PH1)

If you or someone you care for is living with PH1, this page provides information about the YOLT-203 clinical study and how to learn more about participation.

See if you may be eligible，please visit www.clinicaltrials.gov (NCT07587021) or pre-screen here.

What Is PH1?

PH1 is a rare genetic disease in which mutations in the AGXT gene impair the liver’s ability to properly process glyoxylate, leading to excess oxalate production. Elevated oxalate levels may result in recurrent kidney stones, nephrocalcinosis, and progressive kidney damage. In severe cases, systemic oxalosis may occur as oxalate accumulates throughout the body.

Current management approaches for PH1 may include intensive hydration, vitamin B6 supplementation in certain patients, RNA interference therapies, dialysis, and liver and/or kidney transplantation. Despite available management approaches, PH1 can remain associated with significant disease burden.

What Is YOLT-203?

YOLT-203 is an investigational gene editing therapy designed to target glycolate oxidase (GO), an enzyme encoded by the HAO1 gene that is involved in oxalate production pathways in the liver.

YOLT-203 uses a lipid nanoparticle (LNP) delivery approach to transport gene editing components to liver cells following a single intravenous infusion. The investigational therapy is intended to reduce hepatic oxalate production by editing the HAO1 gene.

Preclinical studies evaluating YOLT-203 have been presented at scientific meetings and in company disclosures. YOLT-203 is currently being evaluated in clinical studies, and its safety and efficacy have not been established.

Study at a Glance

√ Global Phase 2 clinical study

√ Children and adults aged 6 years and older may be eligible

√ One-time intravenous infusion

√ Randomized, placebo-controlled study