Mitochondrial dysfunction is a major hallmark of various neurodegenerative disorders including Alzheimer’s disease, Parkinson’s disease, Huntington’s diseases or Amyotrophic Lateral Sclerosis (ALS). However linking mitochondrial dysfunction to the pathogenesis of some of these diseases still needs to be elucidated. Furthermore, in pathologies where this link is already established, the question remains whether specific targets or mechanisms involved in mitochondrial dysfunction will be amenable to therapeutic intervention. MitoTarget is an ambitious project aimed at providing solid data to better understand and exploit the circumstantial evidence linking mitochondrial dysfunction with neuronal dysfunction culminating in neurodegenerative disease. A 36 months translational research program will bring together a unique partnership between basic scientists, a seasoned team of clinical investigators and a SME that has identified a first-in-class compound, TRO19622, that targets mitochondria and has powerful neuroprotective and neuroregenerative activities. In parallel, and orchestrated by the SME that is the coordinator of the project, MitoTarget will bring together a more comprehensive insight into the mechanisms leading to mitochondrial impairments and establish their clinical relevance in a severe orphan neurodegenerative disease, ALS. If successful, it is expected that from this proof of principle a new class of therapeutic agents targeting the underlying mitochondrial dysfunction in neurons or their supporting cells will emerge. Results of the project have the potential to create a new paradigm for the drug discovery for neurodegenerative diseases.