YOLT-203 Receives Orphan Drug Designation from EMA

July 28, 2025 – Shanghai, China — YolTech Therapeutics, a clinical-stage biotechnology company developing next-generation in vivo gene editing therapies, today announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designation (ODD) to YOLT-203, an investigational in vivo gene editing therapy for the treatment of Primary Hyperoxaluria Type 1 (PH1).

This designation follows the Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) previously granted by the U.S. Food and Drug Administration (FDA) in 2024.

YOLT-203 is currently being evaluated in an ongoing investigator-initiated clinical trial. (ClinicalTrials.gov ID: NCT06511349), with interim clinical data demonstrating excellent safety and strong potential to significantly reduce urinary oxalate levels in patients with PH1.

About YOLT-203

YOLT-203 is an investigational in vivo gene-editing therapy designed to be a one-time treatment for PH1, a rare genetic disorder that causes kidney stone accumulation and potentially kidney failure. YOLT-203 has the potential to reduce the oxalate overproduction in PH1 patients by deactivating glycolate oxidase (GO), an enzyme encoded by HAO1 gene and suppressing the synthesis of oxalate precursors.

About YolTech

YolTech Therapeutics is a clinical-stage in vivo gene editing company committed to pioneering the next generation of precision genetic medicines. Our approach combines innovative gene editing technologies with an advanced lipid nanoparticle (LNP) delivery system, creating a versatile platform designed to address a wide range of serious diseases. Central to our mission is the development of internal capabilities, including end-to-end manufacturing, to ensure the highest standards of quality and scalability. Our lead candidate, targeting ATTR, marks a significant milestone as China's first LNP-mediated in vivo gene editing therapy to enter clinical development. With promising early clinical outcomes, YolTech is also advancing therapies for familial hypercholesterolemia (FH) and primary hyperoxaluria type 1 (PH1). As a company dedicated to transforming the treatment landscape, YolTech continues to push the boundaries of what is possible in gene editing.