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sector_ico_Health_trans Human Health

CanEuCre – Genomic Resources Advancing Therapies for Brain Disorders

  • Project Leaders: Elizabeth Simpson
  • Institutions: University of British Columbia (UBC)
  • Budget: $10560878
  • Program/Competition: Applied Genomics Programs
  • Genome Centre(s): Genome British Columbia
  • Status: Closed

The mouse is the premier model organism for human genetic and genomic medical research. Large scale efforts in mouse-functional genomics are currently ongoing in the US, Canada, and the EU. Research is focused on the development of mutations in all the genes of the genome, in order to discover the function of those genes.  These powerful new mutations depend upon “activation” by a molecule called “cre-recombinase” (cre). “CanEuCre” is a Canadian partnership with the European Commission (EU); EU scientists will create cre resources for a wide variety of other organs, while BC scientists will create resources for the brain, thus adding to, and building upon, our strengths.  Therapies for brain disorders are a major unmet need and gene therapy is emerging worldwide, with virus treatment trials currently underway for Alzheimer and Parkinson Disease. This project and the previously funded Pleiades Promoter Project have been highly successful in generating Brain MiniPromoters (tools for specific targeting of molecules to the brain) for use in neuroscience research and gene therapy. The group has attracted $1.4 million from GlaxoSmithKline (GSK), and spinoff grants totaling almost $1.9 M.

The most advanced use of the MiniPromoter-AAV technology is in the collaboration with Dr. Shannon Boye (University of Florida). One of the developed MiniPromoters has enabled “curing” congenital blindness in a mice model. A joint patent covering this therapeutic virus is planned. It’s worthwhile to note that In the last five years the Jackson Laboratory (JAX) through the Mutant Mouse Regional Resources Centre (MMRRC) have filled 19 orders, distributing 41 Pleiades mice to establish breeding colonies throughout the world. Also, the prompter plasmids collection at Addgene has been extremely popular and to date Addgene has sold 170 MiniPromoter plasmids to 84 researchers around the world. Please note that the group expects outcomes of this project will trigger further interactions with Industry groups including: Life Technologies, Novartis, GSK, Pfizer, Miami Mice Research Corp., Genzyme, Genable Technologies, Shire, ReGenX BioSciences, RetroSense Therapeutics, Genvec Theraeutics, Eos Neuroscience, Genentech, Addgene, MMRRC & Applied Biological Materials. Currently, the group has 4 issued US patents for the MiniPromoters, 1 notice of allowance, 6 utility patents and 12 provisional patent applications currently under review. The group has received payment for a non-exclusive six-month license with Genentech, Inc. and are anticipating an additional license.