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LX3010
- Development stage
- Unknown
- Lead developer
- Lexeo Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
LX3010 (formerly MTL-104) is an oral small molecule combination therapy being developed by Lexeo Therapeutics (acquired from Mantle Therapeutics) for the treatment of Friedreich's ataxia (FA). It works through two complementary mechanisms: histone deacetylase (HDAC) inhibition and Nrf2 pathway activation. These mechanisms are designed to increase frataxin protein expression while addressing mitochondrial dysfunction and oxidative stress. Early clinical data across 11 FA patients showed an approximately 6-point improvement in mFARS (modified Friedreich Ataxia Rating Scale) scores at 16 weeks and a mean nine-fold increase in frataxin protein levels in muscle biopsies.
02
Targets
FXN (Frataxin)HDAC (HDAC family)NFE2L2 (Nuclear factor (erythroid-derived 2)-like 2)
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