Pretzel Therapeutics presents PX578 data supporting POLG disease treatment
PX578, a first-in-class small molecule activator of mitochondrial DNA polymerase gamma, demonstrated preclinical efficacy in restoring mitochondrial DNA levels and cellular energy production across multiple POLG disease models. The compound addresses the underlying genetic defect in mitochondrial DNA depletion syndromes, for which no disease-modifying treatments currently exist.

