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LX3050
- Development stage
- Preclinical
- Lead developer
- Lexeo Therapeutics
- Modality
- Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
01
Overview
LX3050 (formerly MTL-501) is a preclinical-stage recombinant fusion protein being developed by Lexeo Therapeutics for the treatment of Friedreich's ataxia (FA). It consists of a recombinant human frataxin protein fused to a proprietary anti-transferrin receptor 1 (TfR1) Fab fragment, functioning as a frataxin replacement therapy. The anti-TfR1 Fab serves as a 'brain shuttle' designed to facilitate crossing of the blood-brain barrier and increase frataxin delivery to the central nervous system. Its biological target is the frataxin protein (FXN), which is deficient in FA patients due to an expanded GAA trinucleotide repeat on the FXN gene. In vitro studies have shown dose-responsive improvements in mitochondrial function.
02
Targets
TFRC (Transferrin Receptor)
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