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.

ProteinQure is a revenue-generating (over $20M CAD total), clinical-stage biotech company developing peptides for precision therapeutic delivery. They combine machine learning and generative AI to design best-in-class therapeutics, both through fee-for-service partnerships with large pharmaceutical companies and via an internal drug discovery pipeline.

PQ203, is a peptide-drug conjugate for triple-negative breast cancer, now in a Phase I clinical trial at Princess Margaret Cancer Centre in Toronto. ProteinQure has experience across 80+ clinical trials and advanced PQ203 from discovery to IND-enabling studies in just two years—and into clinical trials 18 months later—demonstrating exceptional execution in translating preclinical programs into clinical assets.

Building on the innovation of PQ203, an AI-designed SORT1-binding peptide, they further modified and optimized this scaffold for CNS delivery of therapeutic payloads, having the potential to unlock an estimated $100B global market across neurology and neurodegenerative disease indications.

The CNS-delivery program leverages this optimized SORT1-binding peptide to deliver siRNA molecules for targeted gene knockdown in the brain. The program is supported by an advanced data package: best-in-class delivery across multiple in vivo models, including rodents and, more recently, non-human primates. For the lead CNS indication, they selected Glioblastoma Multiforme (GBM), a lethal brain cancer with relatively high incidence in Canada and few effective treatment options.

The interest in glioblastoma (GBM) is guided by ProteinQure’s existing expertise in cancer biology and, importantly, through a partnership with Dr. Stéphane Angers, Director of the Donnelly Centre at the University of Toronto and an internationally recognized leader in GBM genetics. Dr. Angers brings unparalleled leadership, drug development experience, and state-of-the-art tools that will support the advancement of our therapeutic candidate for GBM.

Also on the project team is Dr. Peter Dirks, a world-renowned neurosurgeon at SickKids and pioneer in brain cancer stem cell biology, whose lab has developed and validated in vivo GBM models. Dr. Douglas Cook, Professor and Chair of Neurosurgery at Queen’s University, adds expertise in translational neuroscience and access to a world-class non-human primate facility with established models for CNS therapeutics.

ProteinQure has a proven track record advancing oncology programs into the clinic, and unlike more prevalent diseases such as Alzheimer’s, GBM represents an area to independently advance and commercialize a program. By leveraging Toronto’s world-class ecosystem in GBM and oncology clinical trials, the same roadmap that was successfully executed with PQ203 can be applied to develop and commercialize a life-changing therapy for patients with GBM, who face a dire need for new treatment options.