YolTech Therapeutics, a pioneering clinical-stage gene editing company dedicated to delivering lifelong cures, today announced a major milestone: the first patient has been dosed with YOLT-203, the company’s cutting-edge in vivo genome editing candidate. This marks the commencement of an Investigator-Initiated Trial (IIT) and represents a global first in the application of in vivo gene editing therapies for Primary Hyperoxaluria Type 1 (PH1). YOLT-203 is designed as a single-dose, potentially curative therapy, harnessing the power of YolTech’s proprietary YolCas12™ editor. This trial sets a new standard in the field, pushing the boundaries of what gene editing can achieve in treating life-threatening genetic diseases.
Primary hyperoxaluria is a genetic disorder associated with hepatic oxalate overproduction, leading to increased renal oxalate excretion. PH1 as the most commonly diagnosed subtype, is a childhood-onset autosomal recessive disease form caused by AGXT gene mutations and absence or dysfunction of alanine-glyoxylate aminotransferase. Most patients ultimately progress to renal failure and require intensive hemodialysis as a bridge to a dual liver/kidney transplant to resolve the metabolic defect in the liver and replace the terminally damaged kidneys.
