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

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，这是美国国立卫生研究院(NIH)目前的优先事项。整个NIH内壁研究计划(IRP)的协调努力要求为基因治疗的进步和测试治疗的新产品开发能力提供更多支持。 内部调查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疾病作为概念的证据，这位非营利性的翻译科学家在推进罕见疾病的治疗方面变得越来越重要。其次，对于内部研究人员来说，这是一个难以启动和具有挑战性的过程，对于那些将基因疗法转化为临床的新手来说，这是一个主要的障碍。没有先前经验或商业合作伙伴的内部调查人员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，美国国立卫生研究院内部研究计划(IRP)成立了一个由资深科学家组成的基因治疗特别工作组(GTTF)，以评估满足这一需求的可行方案。工作队侧重于确定潜在的合同开发和制造组织，以便在更全面的层面上提供细胞和基因疗法的商业制造。为了评估这一方法的可行性，工作队正在寻求对目标CDMO进行能力测试，作为能力核准的概念证明。 样品CDMO应具有通过商业制造细胞和基因疗法、成分原材料、过程开发、质粒DNA、病毒载体、细胞库、细胞加工的临床前能力，并在一个研究设施内支持测试能力，以供设计、应用和使用。CDMO应有能力协助缺乏知识或基础设施的研究人员将创新项目带到临床。

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.