Grant: Canada First Research Excellence Fund

The power of combining genomics with RNA technology is exemplified by the remarkable and rapid success of mRNA vaccines against SARS-CoV-2. We are at the beginning of a revolution that will enable the rapid, low-cost development of genome-guided RNA-based medicines for a wide spectrum of diseases, such as those caused by emerging viruses that threaten our society with pandemics and their associated catastrophic health and economic consequences, high-burden diseases such as cancer, which is the number one cause of death in Canada, and rare genetic disorders that are incurable or prohibitively expensive to treat. The D2R program is anchored in the strength and excellence of researchers at McGill University, McMaster University, Université de Sherbrooke, University of Ottawa, and University of British Columbia. D2R is based on a multi-disciplinary work package approach to guide the development of RNA-based drugs to silence or edit genes, replace defective proteins, and improve cell-based therapeutics. Novel computational and statistical methods, including big data technologies, analytics, and AI, will enable these advances by supporting the analysis and interpretation of large datasets and drug design. A strong network of clinical and pharmaceutical/biotechnology industry collaborations will conduct proof-of-concept clinical trials and other activities to accelerate commercialization and clinical translation. Based on the immense burden they place on human health and the economy, and on their amenability to the design of RNA therapeutics, we will focus initially on three disease areas: viral infections, precision oncology and rare genetic disorders. Economic impact will stem from the creation of a nexus for RNA-based drug development in Canada as a hub for partnerships with pharma and biotech to accelerate progression towards regulatory approval. To successfully implement and deploy these medicines, particularly for underserved and equity-seeking populations, including Canada's Indigenous communities, and within vulnerable groups such as the elderly, we will create and analyze diverse socio-genomic datasets and investigate and address ethical, legal and societal issues such as public perception, discrimination and equitable access. These mechanisms will provide a voice for Canadians through engagement and consultation with patient associations, Indigenous communities, and other equity-seeking groups, as well as provide policy recommendations to government.