An Important Milestone for the GM2 Community - Taken From NTSAD

An Important Milestone for the GM2 Community The U.S. Food and Drug Administration (FDA) has authorized a University of Massachusetts (UMass) Chan Medical School research team to proceed with a second-generation investigational AAV gene therapy Phase 1/2 clinical trial for GM2.

This significant advancement would not have been possible without the generous support of members of the NTSAD community. We are deeply grateful to and wish to recognize the families and donor-restricted funds whose commitment made this important research possible, including the Cameron & Hayden Lord Foundation, the Heringer Family Research Fund, the Katie & Allie Buryk Research Fund, the LOTS Research & Education Fund, the New York Area Fund, Susan and Alan Roden, the Sussman Family Fund, and the Vera Pesotchinsky Research Fund.

Combined with support from the NTSAD Research Initiative Fund, NTSAD is proud to support this research in partnership with the Blu Genes Foundation, the Cure Tay-Sachs Foundation, and the Mathew Forbes Romer Foundation. Together, our collaborative investment in this pivotal research is helping drive meaningful progress toward approved treatments for Tay-Sachs and Sandhoff diseases.

 A team from UMass, Massachusetts General Hospital, and the National Institutes of Health – along with input from the patient advocacy organizations – are working on the necessary steps needed to launch the clinical trial. When additional details become available, NTSAD will share these updates with our Tay-Sachs and Sandhoff community.

We congratulate Dr. Gray-Edwards, Dr. Sena-Esteves, and the entire UMass Chan research team on this important achievement and extend our deepest gratitude for their dedication and determination to advance research for the GM2 community.