SickKids Scientist Receives $1M Transformative Research Award to Advance Huntington’s Disease Treatment

SickKids IP&C is excited to congratulate Dr. Christopher Pearson, Senior Scientist in the Genetics & Genome Biology program at SickKids, on receiving the 2025 Transformative Research Award from the Hereditary Disease Foundation (HDF). This prestigious award includes $1 million in funding to support the continued development of a novel therapeutic strategy for Huntington’s disease (HD).

A recognized leader in the study of repeat expansion disorders, Pearson was among the first to show that repeat expansion variations continue to grow over time in affected brain regions. This ongoing expansion leads to the formation of abnormal DNA structures that drive the progression of many neurodegenerative conditions, including HD. Building on this discovery, his team developed a small molecule-based approach to target these structures and promote their contraction. In preclinical models of HD, treatment with these compounds led to improvements in motor function, muscle strength, behaviour and brain health.

Pearson’s work points towards a promising new class of disease-modifying therapeutics that could not only slow the progression of Huntington’s disease, but potentially reverse its effects at the genetic level.

The Industry Partnerships & Commercialization (IP&C) office played a pivotal role in the proposal development process, offering strategic guidance and hands-on support throughout. Team members also contributed to scientific discussions, assessed translational potential, led commercialization planning, and explored investment opportunities to advance the project. This sustained, cross-functional engagement was instrumental in shaping a competitive and strategically positioned application.

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