New licence agreement between Queen’s University and Taysha Gene Therapies could lead to development of treatments for rare diseases in children

News / General / Children’s Health / Research / Discovery / Breakthrough / Pediatric Care

Monday, Sept. 28, 2020

KINGSTON, Ont. – Queen’s University announced that it has entered into a research collaboration and licensing agreement with Taysha Gene Therapies, Inc., for the development and commercialization of gene therapy for GM2 gangliosidoses.

The GM2 gangliosidoses are a group of degenerative/inflammatory diseases in which a fat called GM2 ganglioside accumulates, especially affecting the brain. Two of the more common diseases in this group, TSD and SD, are characterized by rapid neurological deterioration and, typically, death before four years old. The disease can also present in older children and adults.

Queen’s University researcher and Kingston Health Sciences Centre (KHSC) staff member Jagdeep S. Walia (Pediatrics), has generated preclinical evidence for a gene therapy approach that may be a potential treatment for GM2 gangliosidoses, also referred to as Tay-Sachs (TSD) and/or Sandhoff disease(SD).


“We are developing new genetic treatment approaches for rare disorders.  One such treatment has shown promising preliminary results for TSD and SD, which have laid foundations for a clinical trial. Outcomes from our research will help not only patients in Ontario and Canada, but globally. Our research may position Queen’s University and our province as a leader in such gene therapies."

- Dr. Jagdeep S Walia, Medical Genetics, Pediatrics, Queen’s University and Kingston Health Sciences Centre

“Congratulations to Dr. Walia and Taysha on their outstanding collaboration. We look forward to the exciting and important translation of the research to the clinic. Queen’s would like to acknowledge all the funders who have supported this innovative work, including the Cure Tay-Sachs Foundation and GlycoNet. Their support has made this work possible.”

- Dr. Kimberly Woodhouse, VP Research, Queen’s University

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Media Contacts:
Anne Craig 
Media Relations Officer, Queen’s University

Kristen Lipscombe
Strategic Communications Advisor, Kingston Health Sciences Centre