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An estimated 3.8 million Canadians live with a neurological condition, with a projected two-fold increase in this number by 2031. Brain disorders are often progressive, debilitating and require continued care, imposing a significant financial burden on patients, caregivers and the healthcare system. Therapeutic development for brain disorders of the central nervous system (CNS) is particularly challenging due to the high R&D costs involved and the need to engineer therapeutics to cross the blood-brain barrier for effective biodistribution while minimizing toxicity.

This funding will support CereCura Nanotherapeutics Inc. (CCNTx), a spin off company from UBC, that brings together experts in neuroscience, Dr.MacVicar, Dr.Weilinger and Dr.Bernier in collaboration with Dr Pieter Cullis, who is recognized globally for his contributions to Lipid Nanoparticle (LNP) delivery systems. The project aims to test next-generation nanomedicines to develop therapies for neuronopathic Gaucher disease, a rare and fatal monogenic neurological disorder with no effective treatments currently available. In this novel approach, the team proposes to temporarily transform certain brain cells into therapeutic protein “factories,” with the vision of enabling a patient’s brain to produce its own medicine.

The project will provide key insights into development of nanomedicine-based therapeutics for Gaucher disease and will inform feasibility of translating it to humans and potentially to other neurological disorders. In addition, this funding will support the development of an early-stage biotechnology company based in British Columbia (BC) and position it for future fundraising and/or partnerships that will foster growth and job creation in BC.