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First Parkinson's patient receives drug that clears damaged mitochondriaLongevity & Aging

First Parkinson's patient receives drug that clears damaged mitochondria

Mission Therapeutics has dosed the first patient in a Phase 1b trial of MTX325, an oral, brain-penetrant drug that blocks USP30, an enzyme that slows the removal of damaged mitochondria. Mitochondria are the cell's power plants, and cells clear broken ones through a cleanup process called mitophagy. By releasing that brake, the drug aims to help neurons stay healthy, reduce inflammation and possibly slow Parkinson's disease. The trial will enroll 36 people with mild to moderate Parkinson's in the UK, Europe and Australia. It is designed to test proof-of-mechanism, meaning whether the drug does in people what it did in mice, using biomarkers, imaging and safety measures rather than symptom relief. A data readout is expected in late 2027. Because mitochondrial decline is linked to aging, the approach could also be tested in other age-related conditions. It is still very early.

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