Strategies for development of novel adeno-associated viral vectors and production technology: towards national clinical-grade manufacturing capacity

新型腺相关病毒载体和生产技术的开发策略：迈向国家临床级生产能力

• 批准号: nhmrc : GNT1161583
• 金额: $ 78.25万
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2019
• 资助国家: 澳大利亚
• 起止时间: 2019-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/strategies-development-novel-manufacturing-capacity/1379182
• 关键词: Strategies; development; novel; adeno; associated

Gene therapy offers the possibility of new treatments for patients with genetic and acquired diseases. Modified non-pathogenic viruses can safely and efficiently deliver therapeutic DNA to affected cells inside a patient’s body, but manufacturing of these vectors is inefficient and expensive, hampering clinical development. This project focuses on further developing this technology to dramatically enhance manufacturing efficiency and build the foundations for an Australian production capacity.

基因治疗为遗传性和获得性疾病患者提供了新的治疗方法。经修饰的非致病性病毒可以安全有效地将治疗性DNA递送到患者体内受影响的细胞，但这些载体的制造效率低下且昂贵，阻碍了临床开发。该项目的重点是进一步开发这项技术，以大幅提高制造效率，并为澳大利亚的生产能力奠定基础。