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LX3070
- Development stage
- Preclinical
- Lead developer
- Lexeo Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
01
Overview
LX3070 (formerly MTL-801) is a discovery-stage antisense oligonucleotide (ASO) Fab conjugate being developed by Lexeo Therapeutics for the treatment of Friedreich's ataxia (FA). The therapeutic candidate consists of an RNA-targeted ASO designed to stabilize FXN mRNA, thereby increasing the translation of endogenous frataxin protein to address the underlying frataxin deficiency caused by GAA trinucleotide repeat expansions in the FXN gene. To facilitate delivery to disease-relevant tissues, including crossing the blood-brain barrier, the ASO is conjugated to a proprietary Fab fragment targeting transferrin receptor 1 (TfR1). LX3070 was originally developed by Mantle Therapeutics and was acquired by Lexeo Therapeutics in September 2026.
02
Targets
TFRC (Transferrin Receptor)
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