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PX578
- Development stage
- Unknown
- Lead developer
- Pretzel Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
PX578 is a first-in-class, orally administered small molecule designed to activate mitochondrial DNA polymerase gamma (POLG), the enzyme responsible for replication and repair of mitochondrial DNA (mtDNA). By acting as a POLG agonist, PX578 aims to restore or enhance mtDNA copy number and function, thereby improving cellular bioenergetics. This mechanism is particularly relevant for treating mitochondrial DNA depletion syndromes (MDDS) and potentially broader neurodegenerative diseases such as Parkinson’s disease and Alzheimer’s disease. The drug is CNS penetrant, taken once daily, and has been optimized for human use. Developed by Pretzel Therapeutics, it represents a novel approach in targeting mitochondrial dysfunction—a key driver in various rare genetic disorders as well as common neurodegenerative conditions[1][3][4][6][7][10].
- Other names
- POLG – SM
02
Targets
POLG (Mitochondrial DNA polymerase gamma)
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