Persevere Therapeutics

Persevere Therapeutics is a clinical-stage biotechnology company dedicated to developing curative treatments for rare and devastating mitochondrial diseases. The company's primary focus is on POLG-related disorders, specifically Alpers-Huttenlocher Syndrome, a severe neurodegenerative condition affecting children. Their lead program, PTX-001, is a small molecule therapy designed to bypass the genetic defects in DNA polymerase gamma to restore mitochondrial function. By targeting the underlying metabolic and genetic drivers of these orphan diseases, Persevere Therapeutics aims to provide the first disease-modifying options for patients with limited treatment alternatives.