Clinical Trial Update: A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis [Phase 1, Phase 2]

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Summary

An ongoing Phase 1/2 clinical trial is evaluating intravenous delivery of an AAV9 vector expressing human beta-galactosidase in patients with Type I and Type II…

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Diseases / indications

• Gm1 Gangliosidosis

Drugs mentioned

• AAV9 vector expressing human beta-galactosidase

Snippet

Sponsor: National Human Genome Research Institute (NHGRI) | Conditions: Lysosomal Diseases, Gangliosidosis | Phase 1, Phase 2 | Status: RECRUITING