Expert Series: PX578: Understanding a “First-in Class”  POLG Activator

Mitochondrial DNA depletion syndromes, most often caused by mutations in the POLG gene, are devastating disorders with no approved disease-modifying treatments. This presentation introduces PX578, a first-in-class, oral, brain-penetrant activator of DNA polymerase γ that restores mtDNA replication across diverse patient genotypes in cell and animal models. This presentation will discuss the science behind PX578 and current understanding of its mechanism of action.