YOLT-203

YOLT-203 is an investigational in vivo gene-editing therapy developed by YolTech Therapeutics for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production leading to kidney stones and potential kidney failure. The therapy utilizes YolTech's proprietary YolCas12™ system, a novel CRISPR/Cas-based gene editing tool delivered via lipid nanoparticles (LNPs) that target liver cells. Once inside hepatocytes, the system uses guide RNA to direct the YolCas12™ nuclease to the HAO1 gene, inactivating glycolate oxidase (GO), an enzyme critical for oxalate production. By disrupting HAO1 and reducing GO activity, YOLT-203 aims to lower harmful oxalate levels and provide a one-time curative treatment for PH1. Early phase 1 clinical data show promising reductions in urinary oxalate with good tolerability[1][4][5][6][8].