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LE051

Development stage: Phase 1
Lead developer: EdiGene
Modality: Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies
Administration: Intravenous

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.

Targets

DMD (Dystrophin)

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