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.

基因治疗研究的进展可以为多种疾病提供有效和快速的治疗，是美国国立卫生研究院（NIH）目前的优先事项。 NIH内部研究计划（IRP）的协调努力需要增加对基因疗法进步和新产品开发能力的支持，以测试治疗。 校内研究人员面临着主要的基因治疗挑战。 首先，随着生物制药公司越来越少地使用罕见病作为概念证明，非营利性转化科学家在推进罕见病治疗方面变得越来越重要。其次，这是一个困难的过程，内部研究人员发起和具有挑战性的导航，提出了一个主要的障碍，为那些新的基因疗法的翻译到临床。 没有先前经验或商业合作伙伴的校内研究人员缺乏支持和指导，无法开展临床基因治疗项目。 由于这些挑战和推进基因治疗研究的机会，NIH校内研究计划（IRP）成立了一个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方案。 工作组的重点是确定潜在的合同开发和制造组织（CDMO），以提供更全面的细胞和基因疗法的商业制造。 为了评估这种方法的可行性，工作队正在寻求对目标CDMO进行能力测试，作为能力清除的概念验证。 样本CDMO应具有临床前至商业化生产能力，包括细胞和基因疗法、组分原材料、工艺开发、质粒DNA、病毒载体、细胞库、细胞处理，并在一个研究机构内支持设计应用和使用的检测能力。 CDMO应能够协助缺乏知识或基础设施的研究者将创新项目引入临床。