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ML0426
- Development stage
- Preclinical
- Lead developer
- Triplex Therapeutics
- Modality
- DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules
01
Overview
ML0426 (also known as ML1 polyamide) is a synthetic DNA-binding compound belonging to the pyrrole-imidazole polyamide class, developed by Triplex Therapeutics. It is designed to treat Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) by targeting the A3243G mutation in mitochondrial DNA (mtDNA). The compound is mitochondria-penetrant and selectively binds to the mutated mtDNA sequence, thereby suppressing its replication and preferentially promoting the replication of wild-type mtDNA. This shift in heteroplasmy has been demonstrated in patient-derived fibroblasts and cybrid cells, suggesting potential for curative treatment of mitochondrial diseases caused by specific point mutations.
- Other names
- ML1 polyamideML-1 polyamideML 1 polyamide
02
Targets
tRNA (Transfer RNA)
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