DNA to RNA: An Inclusive Canadian Approach to Genomic-based RNA Therapeutics (D2R)

DNA 到 RNA：基于基因组的 RNA 治疗的包容性加拿大方法 (D2R)

• 批准号: CFREF-2022-00045
• 负责人: Crago, Martha
• 金额: $ 68.37万
• 依托单位: McGill University
• 依托单位国家: 加拿大
• 项目类别: Canada First Research Excellence Fund
• 财政年份: 2022
• 资助国家: 加拿大
• 起止时间: 2022-01-01 至 2023-12-31
• 项目状态: 已结题
• 来源: https://www.nserc-crsng.gc.ca/ase-oro/Details-Detailles_eng.asp?id=755350
• 关键词: DNA; RNA; Inclusive; Canadian; Approach

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.

基因组学与RNA技术相结合的力量体现在针对SARS-CoV-2的mRNA疫苗的显著和快速成功上。我们正处于一场革命的开端，这场革命将使基因组指导的RNA药物的快速、低成本开发成为可能，用于广泛的疾病，例如那些由新出现的病毒引起的疾病，这些病毒威胁着我们的社会，造成流行病及其相关的灾难性健康和经济后果，高负担疾病，如癌症，这是加拿大的头号死因，D2 R项目依托于麦吉尔大学、麦克马斯特大学、舍布鲁克大学、渥太华大学和不列颠哥伦比亚省大学研究人员的实力和卓越。D2 R基于多学科工作包方法，以指导基于RNA的药物的开发，以沉默或编辑基因，替换有缺陷的蛋白质，并改善基于细胞的治疗方法。新的计算和统计方法，包括大数据技术，分析和人工智能，将通过支持大型数据集和药物设计的分析和解释来实现这些进步。一个强大的临床和制药/生物技术行业合作网络将进行概念验证临床试验和其他活动，以加速商业化和临床转化。基于它们对人类健康和经济造成的巨大负担，以及它们对RNA治疗设计的适应性，我们将首先关注三个疾病领域：病毒感染，精确肿瘤学和罕见遗传疾病。经济影响将来自于在加拿大建立基于RNA的药物开发联系，作为与制药和生物技术合作的中心，以加快监管批准的进展。为了成功地实施和部署这些药物，特别是对于服务不足和寻求公平的人群，包括加拿大的土著社区，以及老年人等弱势群体，我们将创建和分析不同的社会基因组数据集，并调查和解决道德，法律的和社会问题，如公众认知，歧视和公平获取。这些机制将通过与患者协会、土著社区和其他寻求平等的团体的接触和协商，为加拿大人提供发言权，并向政府提供政策建议。