Khondrion, a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that it has established a Scientific Advisory Board (SAB) comprising world-leading experts in mitochondrial disease.

The SAB will advise Khondrion on its current and future research and development strategy and clinical programmes, including the development of its lead asset, Sonlicromanol. Sonlicromanol is a potentially first-in-class oral small molecule currently in Phase IIb clinical development for the treatment of a range of mitochondrial diseases, including MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) and MIDD (maternally inherited diabetes and deafness) syndromes.

The SAB will be led by Sir Doug Turnbull, Emeritus Professor of Neurology, Newcastle University, UK and former Director of the Wellcome Centre for Mitochondrial Research. Doug has made the understanding of mitochondrial disease his life’s work, having cared for over 1000 patients and their families. In 2012, Doug’s work was recognised when he and his colleagues were awarded the coveted Wellcome Trust Centre status, one of only nine in the UK. As Director, Doug has built an internationally renowned team of clinicians and researchers, with patients opting to travel from all over the country to be under his care.

Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: Attracting such an esteemed and diverse group of mitochondrial disease experts to the Khondrion team is a testament to the strength of the work we are doing in finding potential new medicines for patients with mitochondrial disease. Each member of the SAB brings unrivalled scientific expertise and in-depth knowledge that will help us to reach our goal and their guidance will be particularly invaluable as we progress our lead asset, Sonlicromanol, through clinical development.”

Professor Sir Doug Turnbull, Chairman of Khondrion’s Scientific Advisory Board, commented: “I am delighted to have been appointed as Chairman of Khondrion’s Scientific Advisory Board. The Scientific Advisory Board includes some of the most respected experts across the field of mitochondrial research. Khondrion is an exciting company that is working hard to bring effective treatments to patients living with rare mitochondrial diseases. I look forward to advising the Khondrion team in their drive to improve the lives of patients with mitochondrial disease.”

The Khondrion SAB members are as follows:

  • Sir Prof. Dr. Emeritus Doug Turnbull, FRCP FMedSci FRS (Chairman of the SAB), Professor of Neurology, Newcastle University
  • Dr. Marni Falk, Executive Director, CHOP Mitochondrial Medicine Frontier Program and Professor, Division of Human Genetics, Department of Pediatrics, University of Pennsylvania Perelman School of Medicine
  • Dr. Michelangelo Mancuso, Department of Clinical and Experimental Medicine, Neurological Institute, University of Pisa; Chair, Neurogenetics and Rare Diseases Panel – Italian Society of Neurology; Chair, Neurogenetics Panel of the European Academy of Neurology
  • Dr. Akira Ohtake, Auditor, Japan Pediatric Society; Chairmain, IRB; Vice-president, Centre for Intractable Diseases, Department of Paediatrics & Clnical Genomics, Faculty of Medicine, Saitama Medical University
  • Dr. Shamima Rahman, Professor of Paediatric Metabolic Medicine, UCL Great Ormond Street Institute of Child Health
  • Dr. Anu Suomalainen Wartiovaara, Academy Professor in Clinical Molecular Medicine, Faculty of Medicine, University of Helsinki; Chief Physician, HUSlab, Helsinki University Hospital, Helsinki, Finland

Additional background on all SAB members can be found on the Khondrion website here.