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Therapeutics

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PX578

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Investigational POLG activator designed to raise mitochondrial DNA levels and restore mitochondrial function in diseases driven by mtDNA depletion.

Modality
oral CNS-penetrant small molecule
Availability (US)
Completed Phase 1 healthy-volunteer study; Phase 2 planned for late 2026
Biomarkers
mtDNA levels; mitochondrial function
Hallmark targeted
mitochondrial dysfunction, genomic instability
Kind
investigational drug