NATIONAL INSTITUTES OF HEALTH (NIH), INTRAMURAL RESEARCH PROGRAM (IRP), GENE THERAPY TASK FORCE (GTTF), CONTRACT DEVELOPMENT AND MANUFACTURING ORGANIZATIONS (CDMOS), SPECIALIZED SUPPORT SERVICESS4 AX

美国国立卫生研究院 (NIH)、校内研究计划 (IRP)、基因治疗工作组 (GTTF)、合同开发和制造组织 (CDMOS)、专业支持服务4 AX

• 批准号: 10950093
• 负责人: GARY MAYS
• 金额: $ 155.67万
• 依托单位: AXLE INFORMATICS, LLC
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-06-22 至 2024-06-21
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10950093
• 关键词: NATIONAL; INSTITUTES; HEALTH; NIH; INTRAMURAL

The advancement of gene therapy research can offer effective and rapid treatments for multiple disorders and is a current priority for the National Institutes of Health (NIH). Concerted efforts across the NIH Intramural Research Program (IRP) are requiring increased support for advancement of gene therapies and new product development capabilities to test treatments. There are primary gene therapy challenges facing intramural investigators. First, as biopharmaceutical companies use fewer rare diseases as proofs of concept, the non-profit translational scientist is becoming more significant in advancing therapies for rare diseases. Second, this is a difficult process for intramural investigators to initiate and challenging to navigate, presenting a major barrier for those new to translation of gene therapies to the clinic. Intramural investigators without prior experience or commercial partners lack support and guidance to embark on clinical gene therapy projects. As a result of these challenges and the opportunities to advance gene therapy research, the NIH Intramural Research Program (IRP) formed a Gene Therapy Task Force (GTTF) of senior scientists to assess viable options to meet this demand. The Task Force focused on identifying potential contract development and manufacturing organizations (CDMOs) to provide commercial manufacturing of cell and gene therapies at a more comprehensive level. To assess the viability of this approach, the Task Force is seeking a capability test of a target CDMO as proof-of-concept for capability clearance. The sample CDMO shall have preclinical through commercial manufacturing capabilities of cell and gene therapies, component raw materials, process development, plasmid DNA, viral vectors, cell banking, cell processing, and support testing capabilities within one research facility for design application and use. The CDMO shall have the capability to assist investigators who lack the knowledge or infrastructure to bring innovative projects to the clinic.

基因治疗研究的进步可以为多种疾病提供有效、快速的治疗，是美国国立卫生研究院 (NIH) 当前的首要任务。 NIH 校内研究计划 (IRP) 的共同努力需要加大对基因疗法进步和测试治疗的新产品开发能力的支持。 校内研究人员面临着基因治疗的主要挑战。 首先，随着生物制药公司越来越少地使用罕见疾病作为概念证明，非营利性转化科学家在推进罕见疾病治疗方面变得越来越重要。其次，对于校内研究人员来说，这是一个艰难的过程，并且具有挑战性，这对那些刚开始将基因疗法转化为临床的人来说是一个主要障碍。 没有先前经验的内部研究人员或商业合作伙伴缺乏开展临床基因治疗项目的支持和指导。 由于这些挑战和推进基因治疗研究的机遇，NIH 校内研究计划 (IRP) 成立了由资深科学家组成的基因治疗工作组 (GTTF)，以评估满足这一需求的可行选择。 该工作组的重点是确定潜在的合同开发和制造组织（CDMO），以在更全面的水平上提供细胞和基因疗法的商业制造。 为了评估这种方法的可行性，工作组正在寻求对目标 CDMO 进行能力测试，作为能力许可的概念验证。 样本 CDMO 应具有细胞和基因疗法、成分原材料、工艺开发、质粒 DNA、病毒载体、细胞库、细胞处理的临床前直至商业制造能力，并在一个研究机构内支持设计应用和使用的测试能力。 CDMO 应有能力协助缺乏知识或基础设施的研究者将创新项目引入临床。