Mission Therapeutics United Kingdom United States of America

Mission Therapeutics announces publication in Nature Reviews Drug Discovery of article highlighting how neurodegenerative diseases can be tackled by enhancing mitophagy

Review article in high impact journal outlines the evidence that neurodegenerative diseases –including Parkinson's, Alzheimer's, ALS and Huntington's – can be addressed by boosting mitophagy, the quality control system cells use to remove dysfunctional mitochondriaStrong evidence has emerged that deficient mitophagy is an underlying cause of neurodegenerative diseaseMomentum is building, with the first selective mitophagy enhancers aimed at modifying the course of neurodegenerative diseases entering clinical trials in the last 12 months is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy. In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson's disease (PD), heart failure, Duchenne's Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer's.

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria – consequently improving overall cellular health. Mission's USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction.

Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

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