Mission Therapeutics awarded research grant from MJFF

Published: 13-Apr-2017

Collaborative team to progress novel USP30 inhibitors to develop potential therapies for Parkinson’s disease

Mission Therapeutics has been awarded a grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF).

The company works in drug discovery and development, focused on selectively targeting deubiquitylating enzymes (DUBs) to treat neurodegenerative diseases, cancer and other diseases with high unmet medical need.

This research grant will support the testing of Mission Therapeutics’ potent and selective USP30-targeted inhibitors in translationally relevant stem cell-derived Parkinson’s disease models.

The models were developed by Professor Richard Wade-Martins and his research group at the University of Oxford.


Parkinson's disease is a chronic, degenerative neurological disorder that affects one in 100 people older than 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 US, and more than five million worldwide, have Parkinson's disease.


USP30, a mitochondrial associated DUB, has been implicated in the control of mitophagy – a process where dysfunctional mitochondria are selectively cleared from the cell.

Failure of mitochondrial quality control may lead to degeneration of the highly active substantia nigra neurons in the brain, a pathological mechanism which results in Parkinson’s disease.

The inhibition of USP30 is being studied by Mission Therapeutics to see if this promotes mitophagy and thus improves cellular resilience in this and other neurodegenerative diseases.

The objective of the research collaboration with Professor Richard Wade-Martins is to test Mission’s potent and selective USP30 inhibitors in a range of disease models – induced Pluripotent Stem Cells (iPSC)-derived from patients with sporadic and familial Parkinson’s disease.

Shalini Padmanabhan, Associate Director of Research Programmes at MJFF, said: “USP30 is one of the more promising DUBs associated with mitophagy, in terms of published data and feasibility of compound development.”

“We hope that this collaboration will promote our understanding of the mechanisms and consequences of USP30 inhibition in Parkinson’s disease.”

You may also like