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Compounds for the treatment of Mitochondrial disorders

Leigh syndromeMELAS syndromemitochondrial respiratory dysfunctionorphan drugParkinsonsredox cyclers


This patent identifies various compounds and their derivatives that are suitable for the development of a class of drugs for the treatment of diseases due to the dysfunction of mitochondrial respiratory chain complexes, such as complex I and III. The most known pathologies linked to dysfunction of respiratory chain complexes include the Leigh syndrome, MELAS, Gracile syndrome and Parkinson disease.

Technical features

Mitochondrial disorders are debilitating and life-threatening diseases that still lack effective treatment. They are due to genetic defects affecting oxidative phosphorylation, thus mitochondrial fitness and ATP production. Patients with complex III and I dysfunction develop progressive neurological impairment, myopathy and meet an early death. Currently, no treatment for such pathologies exist.

A pharmaceutical strategy to “replace” the defective complexes III and I has been identified: redox cyclers (compounds that undergo reduction to form radical species that can then react with oxygen, thus regenerating the parent molecule) such as pyocyanin, were found to recover the mitochondrial function in patient-derived cells. These molecules as well as some derivatives improved the pathological phenotypes and motor impairment in animal models with complex III or complex I deficiency.

TRL: 4

Possible Applications

  • A drug for treatment of diseases due to the dysfunction of mitochondrial electron transfer chain complexes, such as complex I and III.
  • Possible drug treatment for Gracile syndrome, mitochondrial dysfunction-linked myopathies,Parkinsons and Leigh syndrome.


  • No existing treatment for mitochondrial diseases linked to complex III dysfunction;
  • A treatment for rare diseases, eligible for Orphan drug designation;
  • Pyocyanin is a small molecule of bacterial origin able to penetrate biological membranes and cross the blood-brain barrier.