Grant: Project Grant

Brain diseases affect around 10% of Canadians, but treating them has been challenging since the brain is difficult to target with regular medicines. Exciting new technologies that carry genetic drugs, similar to those used in COVID-19 vaccines, are changing the way we think about treatments. Our team is working on a unique approach using a novel technology called lipid nanoparticle-messenger RNA (LNP-mRNA) to help heal the brain from within. Decades of research in neuroscience have shown that many brain diseases occur because the brain lacks certain proteins. We believe we can fix this by giving the brain the missing proteins it needs to get better. But there's a problem: a protective barrier called the blood-brain barrier (BBB) makes it almost impossible for proteins to reach the brain. With LNP-mRNA, we have a solution. We can instruct brain cells to make and release the missing proteins inside the BBB, effectively treating these diseases. We aim to develop and optimize LNP-mRNA as a brain treatment that can ultimately be used in the clinic. To do this, we will study (i) the dosing and toxicity for LNP-mRNA in mouse and rat models, (ii) what types of therapeutic proteins can be produced in the brain using this technology, and (iii) make sure LNP-mRNA is safe in non-human primates. Our strategy aims to deliver therapeutic proteins exactly where and when they are needed in the brain, leading to more effective treatments for brain disorders. Our technology is versatile, so we plan to develop medicines for various brain diseases like stroke, cancer, neurodegenerative diseases, and rare genetic diseases. Through a recently incorporated company, we will preclinically develop drug products, which can be licensed to clinical partners for further development. Our unique strategy will unlock the potential of LNP-mRNA technology for neurological disorders and pave the way for transformative brain treatments to improve the quality of life for those in need.