> SPECIFIC BIOLOGICS

DEVELOPING A NEXT-GENERATION GENE EDITING TECHNOLOGY TO TREAT SERIOUS GENETIC DISEASES

Gene editing holds the potential to cure a wide range of genetic diseases by altering DNA in the genome inside cells – but today’s gene editors have limitations. To better realize the significant opportunity of this therapeutic approach, and allow for its application to new therapeutic areas, Specific Biologics (“Specific”) has developed the unique next-generation Dualase™ gene editing platform which can more accurately alter DNA sequences by cutting DNA in two spots as a single molecule. Specific has demonstrated the benefit of this two-site mechanism in proof-of-concept studies in disease-relevant models.

Based on the early work done at Specific, the Dualase™ technology should have broad utility in many diseases with high unmet medical need. The potential of current gene editing technologies is constrained by low on-target efficiency, coupled with possible off-targets which limit their applications for a variety of disease-relevant genetic mutations. Dualase™ gene editors cut DNA in a way that optimally exploits the cell’s naturally occurring DNA repair pathways. This enables two gene editing outcomes, precise DNA deletions to disrupt genes or increased repair to correct genes. Specific has also developed application-specific lipid nanoparticles to deliver the gene editor to certain target cells.

Specific is back by industry leaders Lumira Ventures and adMare BioInnovations who bring significant expertise in building and scaling life sciences companies. Operating from the JLABS @ Toronto incubator in the heart of Toronto’s MaRS Discovery District, our research labs are equipped with state-of the art equipment and are ideally located adjacent to world-class research institutions and hospitals to attract the best talent. Built on the pioneering work of Dr. David Edgell at Western University’s Schulich
School of Medicine & Dentistry, Specific is a great example of realizing the potential of innovative Canadian technologies.

The Specific team has identified a growing list of 100’s of clinically relevant genetic mutations where Dualase’s™ two-site mechanism provides an advantage over existing technologies. We have been actively building a pipeline of preclinical Dualase™-based therapeutics at several of these targets in areas of high unmet medical need to unlock the potential of gene editing for patients in need.

SPECIFIC BIOLOGICS

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