Delivery system for Dualase and testing of potential for gene therapy in partnership with Specific Biologics

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Specific Biologics Inc., a Toronto based start up, has a novel gene editing technique that they have validated for various research applications and are currently developing for use in the treatment of cystic fibrosis. The technique has greater specificity and fewer off target effects than currently available technologies. Based on the considerable interest in the use of gene therapy in ocular applications, SBI is interested in examining the potential of this protein based technology for its potential in the eye which is currently widely touted as a potential gene therapy target. In partnership with the Sheardown lab from McMaster University, which brings expertise in ophthalmic materials and drug delivery, the project will develop a method of delivering the protein based gene editor to the ocular tissue based on previously established ocular delivery systems and will examine the potential of the system to alter the genome of ocular cells in vitro and in vivo. Specifically the work will look at the specificity of the gene editing and the level of off target effects. Ultimately, this work will allow SBI to assess the ophthalmic potential of their system and to optimize the system for the treatment of a variety conditions and for research applications in the eye.