What if gene editing could be designed as precisely as a race car tuned to a specific track?
With new funding from Genome Canada and Ontario Genomics, Western researchers are working to do just that - using a machine-learning platform to accelerate the development of gene-editing tools in collaboration with Toronto-based biotechnology company Specific Biologics.
"A standard car can get you almost anywhere, but you wouldn't take your Honda Civic on a Formula 1 track," said David Edgell, professor in biochemistry at Western's Schulich School of Medicine & Dentistry. "You'd optimize it for that specific environment."
Edgell is applying that same principle to gene editing. Each disease-causing mutation, like each race track, requires its own customized editing tool.
But designing the right tool for each target has traditionally been slow, requiring months of trial and error in the lab. With this new project, researchers are turning to machine learning to predict which designs will work best - compressing timelines and accelerating the path to new therapies.
Edgell's lab has spent years building on CRISPR-Cas9, the Nobel Prize-winning technology that allows scientists to cut DNA at precise locations. While CRISPR has transformed biomedical research - leading to the first Health Canada-approved therapy for sickle-cell disease - Edgell's work aims to push the science even further.
His team is developing a novel class of gene-editing enzymes designed to enhance CRISPR's accuracy. Known as dual-cleaving nuclease, or Dualase®, the technology makes two targeted cuts in DNA rather than one. In doing so, it helps cells repair themselves more accurately and lowers the risk of unintended changes.
"First-generation editors are really good at breaking genes, but not fixing them," said Brent Stead, PhD'12, CEO of Specific Biologics. "We're developing gene editors that actually restore the genetic sequence."
Specific Biologics plays a critical role in translating that discovery into potential therapies. Built on Edgell's foundational research, the company focuses on applying and testing the technology in disease-relevant cell and animal models, helping move it closer to clinical use.
The new $1.8-million investment will help accelerate this work by supporting the machine-learning platform and enabling researchers to generate and test a wide range of new enzyme designs. This will build a robust dataset to improve predictions and expand the technology's potential across a broader range of diseases.
The translational focus was key to securing the funding. The project is supported through the Genomic Applications Partnership Program, which helps improve knowledge transfer from public institutions to Canadian companies to commercialize cutting-edge research.
The implications are significant. Dualase® technology is already being explored for amyotrophic lateral sclerosis (ALS), as well as other genetic disorders caused by repeat expansions, including Huntington's disease. These are conditions with limited treatment options, where correcting the underlying mutation could fundamentally change outcomes for patients.
"This kind of funding is incredibly important," said Edgell. "It's a strong signal that both the science and the partnership have real potential - not just in the lab, but in translating meaningful therapies."
Learn more about how Western is optimizing heath for all.
