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LE051
- Development stage
- Phase 1
- Lead developer
- EdiGene
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
LE051 is an RNA-editing therapy for Duchenne muscular dystrophy (DMD) that incorporates EdiGene's LEAPER 2.0 platform. It uses circular ADAR-recruiting RNA to edit RNA, specifically targeting exon 51 skipping to restore dystrophin expression. This approach aims to treat approximately 13% of DMD patients with genetic mutations amenable to exon 51 skipping. Early clinical data shows improvements in motor function and confirmed dystrophin expression in treated patients.
02
Targets
DMD (Dystrophin)
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