TORONTO, March 18, 2026 — Specific Biologics announced a new CAD $1.8 million grant from Genome Canada and Ontario Genomics to develop a machine learning–enabled platform aimed at accelerating the design of its proprietary Dualase® genome editors. The program, in collaboration with Western University, marks a strategic step toward scaling precision genome editing for a wide range of genetic diseases.
At the core of the company’s approach is the Dualase® genome editing system, a next-generation platform engineered to overcome key limitations of traditional gene editing technologies. Unlike conventional editors, Dualase® uses a unique two-site mechanism, enabling precise and programmable DNA modifications, including the removal, repair, or insertion of both small and large genetic sequences. This dual-cutting strategy creates distinct DNA ends that guide natural cellular repair pathways toward predictable and high-fidelity outcomes.
A defining advantage of Dualase® is its high specificity and minimal off-target activity, achieved through the requirement for coordinated binding at two genomic sites. This design significantly reduces unintended edits while maintaining strong editing efficiency across diverse mutation types, including insertions, deletions, and repeat expansions.
Importantly, the platform is compact (~3.7 kb), allowing it to be packaged into a single adeno-associated virus (AAV) vector or delivered as RNA via lipid nanoparticles. This flexibility supports both viral and non-viral delivery strategies and enables targeting of challenging tissues such as the central nervous system.
From a translational perspective, Dualase® supports two key editing modalities:
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Precise excision of large pathogenic DNA sequences, including toxic repeat expansions, using dual-guided cleavage and efficient non-homologous end joining (NHEJ) repair.
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RNA-templated repair, enabling seamless correction or insertion of genetic material without requiring bulky auxiliary proteins, enhancing deliverability and reducing complexity.
These features position Dualase® as a highly versatile platform for in vivo genome editing, with demonstrated applicability across multiple tissues, including CNS, lung, and liver. The ability to deliver the system using a single AAV vector simplifies manufacturing and dosing, aligning with clinically validated gene therapy approaches while maintaining scalability.
The newly funded machine learning platform will integrate genomic datasets, structural modeling, and experimental validation to predict optimal Dualase® editor designs. This is expected to significantly shorten development timelines by enabling rapid identification of highly potent and specific editing constructs for new targets.
Specific Biologics’ lead program focuses on C9ORF72-associated amyotrophic lateral sclerosis (ALS), where Dualase® is designed to precisely collapse toxic DNA repeat expansions—the most common genetic cause of ALS. Beyond ALS, the platform has broader potential in other repeat expansion disorders, such as Huntington’s disease, as well as additional high-unmet-need genetic conditions.
By combining AI-driven design, high-precision genome editing, and flexible AAV-based delivery, Specific Biologics is advancing a differentiated approach to next-generation genetic medicines. The integration of these capabilities aims to enable durable, one-time treatments targeting the root genetic causes of disease, reinforcing the company’s position at the forefront of in vivo genome editing innovation.
