YolTech Therapeutics, a Shanghai-based startup specializing in lipid nanoparticle delivery and gene editing, announced positive results from an investigator-initiated trial (ITT) of YOLT-203 in patients with primary hyperoxaluria type 1 (PH1). The therapy marks the first in vivo gene-editing treatment to demonstrate positive clinical data for PH1, showing excellent safety and pharmacodynamics profiles and the potential to normalize urinary oxalate levels.
Trial Results
The ITT results highlight YOLT-203’s efficacy in reducing urinary oxalate levels, a key marker of PH1. The drug is designed as a one-time, life-long cure for the rare genetic disorder, which can lead to kidney stones and kidney failure.
FDA Designations
YOLT-203 entered clinical trials for PH1 in August 2024 and received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the US FDA one month later, underscoring its potential to address unmet medical needs.-Fineline Info & Tech
