1.1 本指南旨在为参与细胞治疗产品（CTP）开发、生产、交付和监管的个人和组织提供资源，包括转基因细胞、组织工程医疗产品（TEMP）和组合产品，其中细胞活性是最终产品的功能成分。 1.2 本指南的制定包括了从之前发布的几份指导文件和标准（第节）中得出的输入 2.4 ). 本指南旨在反映CTP效价测定的当前观点。 1.3 CTP可以通过局部或系统治疗疾病或病理来提供治疗。 1.4 这些产品可能提供相对较短的治疗，可能是暂时的，也可能是永久性的，并提供长期治疗。 1.5 这些产物可以是单独的细胞、与瞬时载体结合的细胞、或与支架或在整体治疗中起作用的其他成分结合的细胞。 1.6 效价测定可能是 体外试验 或 体内 旨在确定特定产品效力的分析。 体内 分析可能对研究治疗的作用机制（MOA）特别有用，但对于最终产品质量控制可能不可取，因为它们可能耗时且昂贵，并且 体外试验 化验可能更可取。 1.7 可能会进行多次化验，也可能同时进行两次化验 体外试验 和 体内 分析，将需要提供一个广泛的衡量效力。然而 体外试验 分析可能是产品释放分析的首选方法，因此确定效价分析的研究应强调 体外试验 与临床前或临床结果相关或预测的分析。 1.8 效价测定应在产品开发周期内进行，因此与产品开发和测试开始时相比，在该周期结束时可能更全面。 建议在产品开发周期中尽早开发效价测定( 无花果。1和 2. ). 图1 逐步实施效价测定 图2 产品开发阶段流程图，显示何时开发和引入效价分析 1.9 效价测量是细胞和基于细胞的产品测试的一部分，以证明产品批次在发布供临床使用时符合规定的规范。 1.10 应在产品开发过程中制定保质期规范，以包括效价测量。 1.11 本标准指南不适用于药物或基因治疗产品。然而，基因修饰的细胞疗法，例如嵌合抗原受体-T（CAR-T）细胞疗法，美国FDA将其归类为基因疗法，是适用的。 1.12 本标准并非旨在解决与其使用相关的所有安全问题（如有）。本标准的用户有责任在使用前制定适当的安全、健康和环境实践，并确定监管限制的适用性。 1.13 本国际标准是根据世界贸易组织技术性贸易壁垒（TBT）委员会发布的《关于制定国际标准、指南和建议的原则的决定》中确立的国际公认标准化原则制定的。 ====意义和用途====== 5.1 细胞治疗产品可用于治疗临床疾病，例如再生医学（例如I型糖尿病、急性心肌梗死、小儿先天性心脏病、慢性缺血性心力衰竭、癌症、克罗恩病、慢性伤口修复、神经和脊髓损伤、肌肉骨骼修复），并可用于免疫治疗（例如。 g、 移植物抗宿主病，CAR-T治疗）。 5.2 自体、异体和异种细胞可用于制造产品。 5.3 产品可能仅为细胞、与惰性载体结合的细胞、细胞外基质内的细胞或合成支架内的细胞，并将包括含有细胞的组织工程医疗产品。 5.4 细胞可能是基因修饰的细胞。 5.5 细胞可以是成人干细胞或胚胎干细胞。 5.6 细胞可能受到最小程度的操纵。

1.1 This guide is intended as a resource for individuals and organizations involved in the development, production, delivery, and regulation of cellular therapy products (CTPs) including genetically modified cells, tissue engineered medical products (TEMPs) and combination products where cell activity is a functional component of the final product. 1.2 This Guide was developed to include input derived from several previously published guidance documents and standards (section 2.4 ). It is the intent of this Guide is to reflect the current perspectives for CTP potency assays. 1.3 CTPs can provide therapy by localized or systemic treatment of a disease or pathology. 1.4 The products may provide a relatively short therapy, may be transient, or may be permanent and provide long-term therapy. 1.5 The products may be cells alone, cells combined with a carrier that is transient, or cells combined with a scaffold or other components that function in the overall therapy. 1.6 Potency assays may be in-vitro or in-vivo assays designed to determine the potency of a specific product. In-vivo assays are likely to be particularly useful to study the mechanism of action (MOA) of the therapy, but may not be desirable for final product quality control where they may be time-consuming and expensive, and where in-vitro assays may be preferable. 1.7 It is likely that multiple assays, and possibly both in-vitro and in-vivo assays, will be required to provide a broad measure of potency. However, in-vitro assays are likely to be preferred as release assays for products, and so studies to identify potency assays should emphasize in-vitro assays that are correlative or predictive of preclinical or clinical results. 1.8 Potency assays should be developed during the product development cycle and therefore are likely to be more comprehensive at the end of that cycle compared to the beginning of product development and testing. It is recommended that potency assays be developed as early as possible in the product development cycle ( Figs. 1 and 2 ). FIG. 1 Progressive Implementation of Potency Assays FIG. 2 Flow Chart for Stages in Product Development Showing When Potency Assays Will Be Developed and Introduced 1.9 Potency measurements are used as part of the testing for cell and cell-based products to demonstrate that product lots meet defined specifications when released for clinical use. 1.10 Shelf life specifications should be developed during the product development process to include potency measurements. 1.11 This standard guide is not intended to apply to drug or gene therapy products. However, genetically modified cell therapies, for example the chimeric antigen receptor-T (CAR-T) cell therapy, which the United States FDA classifies as gene therapy, are applicable. 1.12 This standard does not purport to address all of the safety concerns, if any, associated with its use. It is the responsibility of the user of this standard to establish appropriate safety, health, and environmental practices and determine the applicability of regulatory limitations prior to use. 1.13 This international standard was developed in accordance with internationally recognized principles on standardization established in the Decision on Principles for the Development of International Standards, Guides and Recommendations issued by the World Trade Organization Technical Barriers to Trade (TBT) Committee. ====== Significance And Use ====== 5.1 Cell Therapy Products may be used to treat clinical conditions, for example in regenerative medicine (e.g. type I diabetes, acute myocardial infarction, pediatric congenital heart disease, chronic ischemic heart failure, cancer, Crohn’s disease, chronic wound repair, nerve and spinal cord injury, musculoskeletal repair), and may be used for immunotherapy (e.g. graft versus host disease, CAR-T therapy). 5.2 Autologous, allogeneic, and xenogeneic cells may be used to make a product. 5.3 A product may be cells only, cells combined with an inert carrier, cells within an extracellular matrix, or cells within a synthetic scaffold, and will include tissue engineered medical products containing cells. 5.4 Cells may be gene-modified cells. 5.5 Cells may be adult or embryonic stem cells. 5.6 Cells may be minimally manipulated.