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AAV9.hMCOLN1co in Mucolipidosis Type IV
NCT07398872Phase 1Enrolling by invitation
- Sponsor
- Children's Hospital, Zhejiang University School of Medicine
- Start
- Jan 2026
- Primary completion
- Jan 2027
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of AAV9.hMCOLN1co in mucolipidosis type IV, sponsored by Children's Hospital, Zhejiang University School of Medicine. Primary endpoint: Adverse Events and Serious Adverse Events at 5 years. Started Jan 2026; status: enrolling by invitation.
02
Drugs
AAV9.hMCOLN1co
03
Conditions
mucolipidosis type IV
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