Widespread vector distribution in the brain and vector serotype selection have presented significant challenges to gene therapy for lysosomal storage diseases such as GM1 Gangliosidosis. This new grant will fund the development of a minimally invasive AAV-based therapeutic platform. Data essential to the submission of an Investigative New Drug (IND) application to the United States Food and Drug Administration (FDA) should enable conduct of a clinical study in GM1 patients.

This two-year project leverages the considerable translational experience and know-how of the Bankiewicz Laboratory at the University of California San Francisco. The Bankiewicz Laboratory has initiated so far 3 gene therapy clinical trials in neurological diseases.


This new project will build upon work in GM1 gene therapy and leverage the Bankiewicz Laboratory’s considerable translational experience.