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LX3030
- Development stage
- Preclinical
- Lead developer
- Lexeo Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
LX3030 (formerly MTL-707) is a preclinical-stage, oral, tissue-penetrant small molecule histone deacetylase (HDAC) inhibitor being developed for the treatment of Friedreich's ataxia (FA). Acquired by Lexeo Therapeutics from Mantle Therapeutics, LX3030 is a third-generation benzamide designed to cross the blood-brain barrier and increase the production of endogenous frataxin in the central nervous system (CNS). By inhibiting HDAC, the drug promotes epigenetic modulation and chromatin acetylation at the *FXN* gene locus, thereby upregulating frataxin protein levels to address the mitochondrial energy deficiency and iron imbalance characteristic of FA.
02
Targets
HDAC (HDAC family)
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