The US Food and Drug Administration (FDA) has cleared the research team at the University of Massachusetts (UMass) Chan Medical School to proceed with a phase 1/2 clinical trial of an investigational second-generation AAV gene therapy for GM2.
This significant advance would not have been possible without the generous support of members of the NTSAD community. The organisation thanks a number of funding sources, among them 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.
NTSAD is collaborating on this research with the Blu Genes Foundation, the Cure Tay-Sachs Foundation and the Mathew Forbes Romer Foundation. The team from UMass, Massachusetts General Hospital and the National Institutes of Health is working with patient organisations to launch the clinical trial — further information will be published as soon as details are known.
Congratulations to Dr Gray-Edwards, Dr Sena-Esteves and the entire UMass Chan research team on this important achievement, and thank you for their efforts in advancing GM2 research.