NOW Enrolling: GM2 gangliosidosis Gene Therapy Clinical Trial at Queens University

Global enrollment is now open for the TSHA-101 clinical trial at Queens University in Kingston, Ontario, Canada. TSHA-101 is a gene therapy for GM2 gangliosidosis (Tay Sachs and Sandhoff Disease).

To learn more about the clinical trial, visit www.TSD-SDGTXtrial.com

Please see the community letter sent by the Taysha team on 12 March 2021.

Dear Tay-Sachs and Sandhoff Community,

We are pleased to announce that a Phase 1/2 clinical trial of TSHA-101 is currently enrolling patients with infantile GM2 gangliosidosis, also known as Tay-Sachs and Sandhoff diseases. The clinical trial will be conducted by the Kingston Health Sciences Centre at Queen’s University in Ontario, Canada. The trial received financial support from the Canadian Glycomics Network (GlycoNet) and Taysha Gene Therapies, Inc., a patient-centric gene therapy company focused on monogenic diseases of the central nervous system.

First and foremost, the purpose of this clinical trial is to evaluate the safety of TSHA-101, an investigational AAV9-based gene therapy. The trial will also help determine if there is an improvement in symptoms and overall survival in children who have received TSHA-101.

Children 12 months and younger who have been diagnosed with GM2 gangliosidosis may be eligible to enroll in this study. The trial will enroll children wherever they and their caregivers reside, including outside Canada.

However, all clinical trial visits will be conducted in person at Kingston Health Sciences Center at Queen’s University in Kingston, Ontario, Canada. TSHA-101 is an innovative approach to gene therapy as it is designed to deliver both HEX genes (Hex A and Hex B) at the same time in the same cell. TSHA-101 is delivered as a single injection through the spinal fluid, known as intrathecal delivery. We are hopeful for the outcomes in this initial study of TSHA-101, but the safety of our patients continues to be our highest priority.

We encourage you to visit www.tsd-sdgtxtrial.com for more information regarding the TSHA-101 clinical trial. If interested in learning more about eligibility criteria, you may contact the research team at info@TSD-SDGtxtrial.com.

We are grateful for the support of the GM2 gangliosidosis communities and for Queen’s University for their continued efforts to advance TSHA-101 into the clinic. We look forward to providing updates about the TSHA-101 clinical trial and its progress.

Warm regards,

The Taysha Team