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).
About YOLT-203
YOLT-203 is an investigational in vivo gene-editing therapy designed to be a once-and-done treatment for PH1, a rare genetic disorder that leads to recurrent kidney stone formation and progress to kidney failure. The therapy aims 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
Built on next-generation CRISPR/Cas and LNP technologies, YolTech Therapeutics is pioneering in vivo gene-editing medicines with the potential for one-time, lifelong benefit. The company’s expanding clinical pipeline targets genetic, metabolic, cardiovascular, and autoimmune diseases, with initial results supporting the potential for durable and transformative therapeutic benefit.
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Contacts
YolTech Therapeutics
Ally Yu
xiaolingyu@yoltx.com