Overview
Specific Biologics Inc. (SBI) is developing Dualase®, a two-site genome editing platform designed to precisely remove, repair, or insert DNA sequences as therapeutic for serious genetic diseases. Unlike other genome editing systems like CRISPR-Cas, base editing or prime editing, Dualase enables accurate, programmable outcomes at diverse therapeutic targets unlocking the market for genome editing that is rapidly growing (+15.7% per year) and expected to reach over $40 billion/year by 2034.
The key objective of this project is to build a machine-learning (ML)-enabled prediction tool to design bespoke Dualase editors that maximize on-target activity and minimize off-target risk and identify highly active Dualase editors for therapeutic targets. It draws on the expertise of Dr. David Edgell at Western University, whose lab discovered Dualase editors, and SBI whose have developed therapeutics based in Dualase genome editors to 10+ therapeutic targets to date.
The ML tool will rapidly generate and identify bespoke Dualase editors in silico, reducing design-build-test cycles that require considerable wet lab empirical evaluation. This increases the probability of successfully identifying potent editors and enables SBI to advance more therapeutic programs in parallel internally, and together with partners.
The key aims of the project include generation of novel variants of Dualase directed to therapeutically relevant sequences using directed evolution, developing and validating the predictive ML model, and piloting new editors across multiple high-value disease targets.
Commercialization will follow a staged pathway. Internally, the tool will accelerate Specific Biologics’ own pipeline toward the clinic. Externally, it will accelerate partnerships by allowing design of Dualase constructs, creating new licensing and co-development revenues.
The expected benefits to Canada include high-skilled job creation in AI/ML, computational biology, and advanced therapeutics; expansion of Canada’s leadership in genome editing and machine learning; and enhance SBI commercialization capacity through new intellectual property and licensable therapeutics positioning SBI as an end-to-end therapeutic anchor company in Canada.
The project will also deliver a powerful capability to accelerate research timeline in the Canadian research community through non-commercial licensing, saving months of wet lab experimentation and reduce R&D costs. Importantly, the tool will bring innovative treatments to patients sooner, and strengthen SBI’s asset value and partnership potential, all while contributing to improved health outcomes for patients in Ontario, Canada and globally.