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AAV9-GLB1 Gene Therapy in Type I and Type II GM1 Gangliosidosis
NCT03952637Phase 2Recruiting
- Sponsor
- National Human Genome Research Institute
- Start
- Aug 2019
- Primary completion
- Jan 2030
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
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Overview
Phase 2, open-label trial of bomtabegagene bavoparvovec in GM1 gangliosidosis (GM1) (2 arms), sponsored by National Human Genome Research Institute. Primary endpoint: Safety. Started Aug 2019; status: recruiting.
02
Drugs
bomtabegagene bavoparvovec
03
Conditions
GM1 gangliosidosis (GM1)
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