Novel technology for quality control of viral vector particles

基因治疗的目标是修改或操纵基因的表达或改变活细胞的生物学特性以用于治疗。病毒载体是一种无致病性的修饰病毒，是基因治疗中传递遗传物质的理想载体。在过去的十年中，它们的设计和组成的重大改进提高了安全性，并将基于病毒载体的疗法置于现代医学的前沿。病毒载体已被用于治疗各种疾病，如代谢性疾病、心血管疾病、肌肉疾病、血液疾病、眼科疾病，并被用作疫苗递送的平台。虽然这些载体的制造也取得了进展，但需求的增加现在需要做出重大努力来提高产量和纯度。该项目建议利用加拿大affinit<e:1>仪器公司开发的技术，开发新的和负担得起的质量控制策略，以监测制造过程中病毒载体生产的效率。正如最近的“加拿大生物制造和生命科学战略”所传达的那样，加拿大迫切需要恢复灵活的生物制造能力，以应对我们面临的COVID-19等意想不到的挑战。在拟议项目背景下开发的技术将有助于共同努力，在加拿大重建强大、高效和有弹性的生物制造能力。

The goal of gene therapy is to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. Viral vectors, which are modified viruses devoid of pathogenicity, are ideal vehicles to deliver genetic material for the purpose of gene therapy. Over the last decade, significant improvements in their design and composition have enhanced safety and placed viral vector-based therapies at the forefront of modern medicine. Viral vectors have been employed for the treatment of various diseases such as metabolic, cardiovascular, muscular, hematologic, ophthalmologic, and are used as platforms for vaccine delivery. Although the manufacturing of these vectors has also progressed, increases in demand now requires significant efforts to increase yield and purity. This project proposes to exploit the technology developed by Affinité Instruments, a Canadian company, to develop new and affordable quality control strategies to monitor the efficiency of viral vector production during the manufacturing process. As communicated through the recent "Canada's Biomanufacturing and Life Sciences Strategy" Canada is in desperate need to re-instate a flexible biomanufacturing capacity to respond to unexpected challenges such as the one we are facing with COVID-19. The technologies developed in the context of the proposed project will contribute to the collective efforts to rebuild a strong, efficient, and resilient biomanufacturing capacity in Canada.