Development of an Industrial Manufacturing Platform for Adeno-Associated Virus (AAV) production to support Regenerative Gene Therapy products

开发用于腺相关病毒 (AAV) 生产的工业制造平台，以支持再生基因治疗产品

• 批准号: 102568
• 金额: $ 178.85万
• 依托单位: COBRA BIOLOGICS LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2016
• 资助国家: 英国
• 起止时间: 2016 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=102568
• 关键词: Development; Industrial; Manufacturing; Platform; Adeno

Gene therapy is becoming an increasingly important method of treatment for a variety of major unmet medicalneeds especially in the areas of inherited and rare diseases and diseases of the eye, conditions which are lifethreatening or significantly diminish quality of life. Adeno-associated virus (AAV) vectors are currently thedelivery vehicle of choice for gene therapy treatments but the advancement of these treatments into clinicaltrials is currently hampered by the lack of scalabilty needed to manufacture these vectors.The proposed collaboration between Cobra and CPI will develop the scientific understanding to allow scalableflexibile process to be developed to manufacture AAV vectors. This will enable the acceleration of morepotential products into clinical testing and ultimately new medicinces. This in turn will increase the chances oftreatment being developed for a whole range of these currently intractable diseases.

基因治疗正在成为一种日益重要的治疗方法，用于治疗各种主要的未满足的医疗需求，特别是在遗传性和罕见疾病以及眼部疾病、危及生命或显着降低生活质量的疾病领域。腺相关病毒 (AAV) 载体目前是基因治疗的首选递送载体，但由于缺乏制造这些载体所需的可扩展性，目前这些治疗方法进入临床试验的进展受到阻碍。Cobra 和 CPI 之间拟议的合作将发展科学认识，从而开发出可扩展的灵活工艺来制造 AAV 载体。这将使更具潜力的产品加速进入临床测试并最终成为新药物。这反过来又会增加开发针对一系列目前棘手疾病的治疗方法的机会。