HG302 is an investigational in vivo CRISPR-based gene-editing therapy developed for the treatment of Duchenne muscular dystrophy (DMD). The therapy uses a single adeno-associated virus (AAV) vector to deliver a high-fidelity Cas12Max (hfCas12Max) DNA-editing system targeting the exon 51 splice donor site of the DMD gene, with the aim of restoring dystrophin expression in muscle fibres. HG302 is administered as a single intravenous infusion and is being evaluated in a first-in-human, open-label, dose-escalation study to assess safety, tolerability, and preliminary efficacy, including changes in dystrophin expression and motor function.
Disease: Duchenne Muscular Dystrophin, DMD, (NCT06594094)
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Candidate summary:
Disease/Disease category:
- Neuromuscular diseases
Official title:
An Investigator-initiated Clinical Study Evaluating the CRISPR-hfCas12Max Gene Editing Therapy in the Treatment of Duchenne Muscular Dystrophy (DMD)
Phases:
- Unspecified
Status:
Active recruiting
Country:
- China
