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Participating in a clinical trial may be an option for some families caring for a loved one with GM2 gangliosidosis (Tay-Sachs disease or Sandhoff disease)—including yours.
That’s why we wanted to share information with you about a study being conducted to learn if an investigational therapy called TSHA-101 can help children with Tay-Sachs disease or Sandhoff disease. This will be the first time that TSHA-101 is studied in humans.


Want more information on the clinical trial? Download a brochure to learn more:

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Doctor Examining CT Scan

What will the clinical trial evaluate?

The clinical trial will investigate: 

  • Safety of TSHA-101 in humans

  • Effectiveness of TSHA-101 in improving survival and symptoms of Tay-Sachs disease and Sandhoff disease

  • Every qualified child enrolled in the clinical trial will receive TSHA-101, the one-time investigational therapy.

Who can participate in the clinical trial? 

The clinical trial will enroll children who: 

  • Are aged 15 months or younger at the time of dosing

  • Have been diagnosed with Tay-Sachs disease or Sandhoff disease

The clinical trial team will do assessments and discuss other factors with you to ensure the trial is appropriate for your child.

The trial will enroll children wherever they and their caregivers reside. Clinical trial visits will be conducted in person at Kingston Health Sciences Center at Queen’s University in Kingston, Ontario, Canada. 

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Doctor Analyzing X-Rays

Who is conducting the clinical trial?

The clinical trial is sponsored and conducted by Queen’s University in Ontario, Canada.


The trial received financial support from the Canadian Glycomics Network (GlycoNet) and from Taysha Gene Therapies, Inc.


Want more information on the clinical trial? Download a brochure to learn more about: 

•    The risks and benefits of participating in this clinical trial

•    What a family can expect to happen during the clinical trial

•    The types of assessments your child will receive

•    How the therapy is administered

In addition, you can contact the research team at or visit and search for NCT04798235.

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