GEN6050X is an investigational in vivo base-editing therapy for Duchenne muscular dystrophy caused by mutations amenable to exon 50 skipping. It is designed to modify the dystrophin pre-mRNA splicing pattern so that exon 50 is excluded, restoring the reading frame and enabling production of a shorter but potentially functional dystrophin protein. GEN6050X consists of two single-stranded AAV9 vectors that deliver the components of a CRISPR-guided base-editing system to skeletal and cardiac muscle following intravenous administration.
Disease: Duchenne Muscular Dystrophy (DMD) (NCT06392724)
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Candidate summary:
Disease/Disease category:
- Neuromuscular diseases
Official title:
A Single-arm, Open-label, Single-center Study to Evaluate the Safety and Tolerability of Intravenous GEN6050X Gene Therapy in Ambulatory Boys With Duchenne Muscular Dystrophy (DMD).
Phases:
- Phase 1
Status:
Active not recruiting
Country:
- China
