D2R will focus initially on three disease areas:

Infectious diseases

Nowhere is the impact of RNA therapeutics currently more evident than in anti-viral vaccines. With international partners, D2R will prioritize RNA viruses known or predicted to represent the many established and emerging pandemic threats (e.g., coronaviruses, hantavirus, dengue fever, West Nile virus, Zika virus, non-poliovirus enteroviruses, influenza A, Chikungunya virus, Ebola, Marburg, and Nipah virus). A range of RNA-based vaccines and therapeutics will dramatically improve Canada鈥檚 pandemic preparedness and allow rapid responses to emerging viruses.

Precision oncology

RNA-based therapies promise to address the problems of traditionally 鈥渦ndruggable鈥� targets, which have severely hindered the growth of precision oncology. We will expand the therapeutic arsenal to match the heterogeneity of cancer, using genomic and transcriptional data to design RNA-based therapies (e.g., RNAi, RNAa, aptamers) that silence genes involved in cancer progression and target oncogenic mutations and gene fusions. Our approach will integrate other emerging technologies in precision oncology, such the development of cell-based therapies (e.g., RNA-based CAR-T programming) and RNA vaccines for cancer. We will ensure that comprehensive genomic profiling and clinical trials include underserved minorities.

Rare diseases

The approximately 6,000 known inherited genetic disorders, while individually rare, have a cumulative prevalence of ~10% of the population. Yet, therapies exist only for a handful of these. RNA-based therapies provide unique opportunities to address these disorders (e.g., small interfering RNAs have already been approved by the US Food and Drug Administration for several rare genetic diseases). In Canada, founder effects (i.e., concentrating rare mutations) in specific populations such as in Quebec, the Atlantic provinces, and isolated Indigenous communities, contribute to one of the world鈥檚 largest cohorts of rare genetic disorders. Collaborative networks will be mobilized to identify targets for RNA-based therapies.