Conquering Diseases

Study Of An Experimental Gene Therapy For Tay-Sachs Or Sandhoff Disease


Seeking children aged 6 months and older with Tay-Sachs or Sandhoff disease for a study of the safety of an experimental gene therapy and whether it improves their ability to move.


Participants in this study will receive either a low, mid or high dose of the investigational gene therapy. There will be about 13 different study visits over one year, including an overnight hospital stay after being given the experimental gene therapy.

What we're hoping for

We hope to evaluate how safe and effective an experimental gene therapy for infants and children with Tay-Sachs or Sandhoff disease is in improving the child's ability to move.

Additional Information Identifier: NCT04669535

 Principal Investigator

Terence R Flotte, MD

University of Massachusetts Medical School

 Study Contact

Jaclyn  Longtine



UMMS University Campus

55 Lake Ave North 

Worcester,MA 01655