YolTech Therapeutics, a clinical-stage biotechnology company developing next-generation in vivo gene editing therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for YOLT-203, an in vivo gene-editing therapy for the treatment of Primary Hyperoxaluria Type 1 (PH1).

The global multicenter, randomized, double-blind, placebo-controlled study is the first pivotal trial of an in vivo gene-editing therapy for PH1, aiming to evaluate the safety and efficacy of YOLT-203 in reducing urinary oxalate levels and improving long-term renal outcomes.

YOLT-203 has also received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. FDA, as well as Orphan Drug Designation from the European Medicines Agency (EMA).