Primary
Hyperoxaluria
(PH1)

Investigational in vivo gene editing therapy for PH1

YOLT-203 is an investigational in vivo gene editing therapy being evaluated for the treatment of Primary Hyperoxaluria Type 1 (PH1), a rare inherited metabolic disease caused by mutations in the AGXT gene. YOLT-203 is designed to reduce oxalate production by targeting the HAO1 gene in the liver. YOLT-203 has not been approved by any regulatory authority for any use.

About Primary Hyperoxaluria Type 1 (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.

About 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.

PH1-YOLT-203-2001 Clinical Trial

PH1-YOLT-203-2001 (NCT07587021) is a global Phase 2 randomized, double-blind, placebo-controlled clinical trial evaluating YOLT-203 in children and adults living with PH1.

The study is designed to evaluate the safety, tolerability, pharmacodynamics, and investigational activity of YOLT-203.

Key study features include:

 ✔  Participants ages 6 years and older

 ✔  Genetically confirmed PH1 diagnosis

 ✔  Randomized, placebo-controlled study design

 ✔  Global multi-center enrollment

Additional information about the study, including eligibility criteria and study locations, is available on ClinicalTrials.gov.

Important Information

YOLT-203 is an investigational therapy and has not been approved by the U.S. Food and Drug Administration, the European Medicines Agency, the UK Medicines and Healthcare products Regulatory Agency, or any other regulatory authority.

Participation in clinical trials involves potential risks and benefits. Eligibility and participation are determined by study investigators and protocol requirements.