May 21, 2026 —
Queen’s University researcher Dr. Jagdeep Walia has received a $5 million contribution from the Hilary and Galen Weston Foundation to support the development of gene therapies for rare and often fatal genetic neurodegenerative disorders. The funding will help advance clinical translation of Walia’s work on GM2 gangliosidoses, a group of inherited disorders that includes Tay-Sachs disease, Sandhoff disease, and AB variant.
GM2 gangliosidoses are caused by defects that prevent the body from properly metabolizing specific brain lipids. As a result, toxic lipid material accumulates in brain cells, leading to progressive neurodegeneration. In the infantile form, affected children are often born appearing healthy, but begin showing developmental regression within the first months of life. The disease can lead to loss of motor function, impaired hearing and vision, severe neurological decline, and death, often by early childhood.
Walia’s current research is focused first on gene therapy approaches for infants diagnosed with these disorders, with plans to expand later into juvenile- and adult-onset forms. His team has developed gene therapy candidates in the laboratory at Queen’s, and animal studies have shown promising results, including disease correction in some treated animals. The next major step is to evaluate whether these findings can translate safely and effectively into humans.
The Weston Foundation funding, combined with additional support from the Canadian Institutes of Health Research, brings the project’s available funding to approximately $8 million. Walia hopes to use this support to launch a clinical trial for GM2 gangliosidoses in early 2027, with patients expected to participate from Canada and the United States.
The program reflects the broader promise and challenge of rare disease gene therapy. For disorders with no approved treatments and limited commercial incentives, academic research, philanthropic funding, and cross-border patient participation can be essential to moving therapies toward the clinic. Walia emphasized that rare disease research is costly and difficult, but also deeply needed for patients and families facing conditions with few or no therapeutic options.
