YolTech Therapeutics Receives U.S. FDA Rare Pediatric Disease Designation for YOLT-203 in Treating Primary Hyperoxaluria Type 1.

YolTech Therapeutics has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for their gene editing therapy, YOLT-203, in the treatment of Primary Hyperoxaluria Type 1 (PH1). PH1 is a genetic disorder that causes increased renal oxalate excretion and can lead to renal failure. YOLT-203 is the first in vivo gene editing therapy developed specifically for PH1 and aims to correct the genetic mutations responsible for the disease. The therapy has shown promising results in preclinical models, and if approved by the FDA, YolTech will be eligible to receive a priority review voucher. [Extracted from the article]