Mission Therapeutics presents at Michael J Fox Foundation’s Parkinson’s Disease conference

Mission Therapeutics announced that its Medical Director, CNS Translational Medicine, Dr Paul Thompson will be delivering a presentation today at the Parkinson’s Disease Therapeutics Conference in New York City.

  • Preclinical data will be presented on novel USP30 inhibitors being developed as potential therapies for Parkinson’s disease.
  • Programme of research supported by grant from The Michael J. Fox Foundation for Parkinson’s Research.

Mission Therapeutics is the leading drug discovery and development company focused on selectively targeting deubiquitylating enzymes (DUBs) to treat neurodegenerative diseases, cancer, and other diseases with high unmet medical need.

The conference is The Michael J. Fox Foundation for Parkinson’s Research (MJFF)’s 11th annual scientific conference and the only one in the world focused exclusively on Parkinson’s disease drug development.

Mission Therapeutics’ presentation, entitled “Validation of USP30 as a Therapeutic Target for Parkinson’s Disease Modification,” will be given during the “Emerging Targets Advancing Treatments Session,” between 9.00h-10.40h ET.

In his presentation, Dr Thompson will share preclinical data generated by Mission Therapeutics as part of their development of potent and selective USP30 inhibitors, ranging from simple in vitro systems through to translationally relevant stem cell-derived Parkinson’s disease models. He will outline some of the scientific mechanisms as well as the clinical potential of USP30 inhibitors in Parkinson’s disease.

USP30 is a mitochondrial-associated DUB that has been highlighted as a promising new target in Parkinson’s disease. It has been implicated in the control of mitophagy – a process that regulates the selective clearance of poorly functioning mitochondria by modifying levels of a protein called ubiquitin. Failure of mitochondrial quality control may lead to degeneration of the highly active substantia nigra neurons in the brain, a pathological mechanism that can result in Parkinson’s disease.

Dr Anker Lundemose, Chief Executive Officer of Mission Therapeutics, said: “Being invited to speak at The Michael J. Fox Foundation’s Parkinson’s Disease Therapeutics Conference, as well as receiving a grant from the organisation, is a great honour. It is also an endorsement of the potential clinical utility of inhibiting USP30 as a treatment for Parkinson’s disease. In the immediate term the data from our studies are promoting our understanding of the scientific mechanisms and consequences of USP30 inhibition in Parkinson’s disease. They also inform our active USP30 inhibitor preclinical development strategy.”

Mission Therapeutics and the University of Oxford were awarded a grant from MJFF in April 2017.

Parkinson’s Disease
Parkinson’s disease is a chronic, degenerative neurological disorder that affects one in 100 people over age 60. There is no objective test, or biomarker for Parkinson’s disease, so the rate of misdiagnosis can be relatively high. Estimates of the number of people living with the disease therefore vary, but recent research indicates that at least one million people in the United States, and more than five million worldwide, have the disease.

About the Parkinson’s Disease Therapeutics Conference
The Parkinson’s Disease Therapeutics Conference brings together 300 research and business development professionals from both academia and industry and showcases the most exciting and innovative research from The Michael J. Fox Foundation’s research portfolio. Novel advances in basic and translational research from both academic and industry labs are highlighted through speaker presentations and a poster session. The event is a platform for field leaders to share new and unpublished results and for fostering new relationships and collaborations.

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