MGE-101

MGE-101 is an investigational in vivo gene editing therapy developed by Primera Therapeutics for the treatment of rare and orphan mitochondrially driven disorders. The therapy utilizes a targeted gene editing platform designed to precisely modify or eliminate pathogenic mutations within the mitochondrial DNA (mtDNA). By addressing the underlying genetic cause of mitochondrial dysfunction, MGE-101 aims to restore normal cellular energy production and improve clinical outcomes for patients with orphan mitochondrial diseases. The program is currently in early-stage clinical development, with pipeline data indicating it has reached Phase 1.