By Fineline Cube 
China-based YolTech Therapeutics 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 underscores the potential of YOLT-203 to address a significant unmet medical need in this rare genetic disorder.
YOLT-203 Mechanism
YOLT-203 is designed to be a one-time treatment for PH1, a rare genetic disorder that causes kidney stone accumulation and potentially kidney failure. The therapy works by deactivating glycolate oxidase (GO), an enzyme encoded by the HAO1 gene, thereby reducing oxalate overproduction in PH1 patients and suppressing the synthesis of oxalate precursors.
Clinical Trial Progress
YOLT-203 is currently being evaluated in an ongoing investigator-initiated clinical trial (NCT06511349). Interim data from the trial have demonstrated excellent safety and a strong potential to significantly reduce urinary oxalate levels in patients with PH1. These findings highlight YOLT-203’s promise as a transformative treatment option for PH1 patients.-Fineline Info & Tech