Clinical Trial Update: Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial [Phase 1]

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Summary

A Phase 1 prenatal gene therapy trial using an AAV9 vector delivering human beta-galactosidase for GM1 gangliosidosis type I and type II; currently not yet recr…

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Diseases / indications

• Gm1 Gangliosidosis Type
• Gm1 Gangliosidosis Type Ii

Drugs mentioned

• AAV9-hBGAL gene therapy (AAV9 vector expressing human beta-galactosidase)

Snippet

Sponsor: Tippi Mackenzie | Conditions: GM1 Gangliosidoses, GM1 Gangliosidosis, Type I | Phase 1 | Status: NOT_YET_RECRUITING