Translational Cell Therapy Core

转化细胞治疗核心

• 批准号: 8931149
• 负责人: Michael R. Verneris
• 金额: $ 30.79万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 1997
• 资助国家: 美国
• 起止时间: 1997-09-15 至 2020-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8931149
• 关键词: Animal Model; Annual Reports; Area; Basic Science; Biological Process; Biological Response Modifier Therapy; Biology; Cell Culture Techniques; Cell Separation; Cell Therapy; Cells; Chemistry; Clinic; Clinical; Clinical Investigator; Clinical Trials; Complex; Coupled; Development; Documentation; Dose; Ensure; Face; Funding; Goals; Growth and Development function; Health; Hematopoietic Stem Cell Transplantation; Human; Human Resources; Immune; Knowledge; Laboratories; Laboratory Research; Lead; Lymphocyte; Lymphocyte Subset; Malignant Neoplasms; Mediating; Medical; Memory; Methodology; Minnesota; Molecular; Movement; Mus; Natural Killer Cells; Patient Care; Phase; Population; Preparation; Procedures; Process; Production; Qualifying; Records; Regulatory T-Lymphocyte; Reporting; Research; Research Infrastructure; Research Personnel; Resources; Safety; Scientist; Series; Services; Speed; Staging; Stem cells; Techniques; Testing; Therapeutic; Therapeutic Studies; Translational Research; Translations; Universities; Work; abstracting; assay development; cell type; clinical care; data management; graft vs host disease; hematopoietic cell transplantation; innovation; interest; member; novel; pre-clinical; product development; progenitor; programs; public health relevance; quality assurance; reconstitution; research clinical testing; stem cell biology; technology/technique; translational medicine

Abstract Our understanding of the biology of stem cells and lymphocytes has increased dramatically, including knowledge about their development, growth requirements and function. Coupled with significant improvements in cell isolation and culture techniques, these advances have created exciting opportunities for clinical translational research using cellular therapy. However, the transition of technology and techniques from a basic research laboratory to a current good manufacture compliant (cGMP) laboratory is complex and nuanced, and neither the basic/translational researcher nor clinician is well versed in the FDA requirements. The main function of Core C is to facilitate the rapid and efficient transfer of basic/translational observations into meaningful and effective cellular therapeutics. Through a series of specific aims, we will describe our process of developing an early stage, laboratory cell population of interest and we will explain how members of Core C will work with investigators to adapt basic laboratory techniques to a cGMP compliant methodology via a pre-cGMP “staging area”. Members of Core C will provide the necessary documentation in support of FDA IND submissions, including the development of the chemistry, manufacturing and control (CMC) section and related documents. We will describe the production of three cell products (T regulatory cells, memory-like NK cells and T progenitors) which are described in Projects 1 and 3. Core C will also ensure that appropriate quality standards both for the product and facility. Lastly, Core C will interact with project and core investigators, providing cell products for testing as well as advice on cell delivery, dosing and potency assay development as appropriate.

抽象的 我们对干细胞和淋巴细胞生物学的了解急剧增加，包括 了解它们的发育、生长要求和功能。加上显着的改进 在细胞分离和培养技术方面，这些进步为临床创造了令人兴奋的机会 使用细胞疗法的转化研究。然而，技术和工艺的转变 基础研究实验室到当前符合良好生产规范 (cGMP) 的实验室是复杂且复杂的 细致入微，基础/转化研究人员和临床医生都不熟悉 FDA 的要求。 Core C的主要功能是促进基础/翻译观察的快速高效传输 转化为有意义且有效的细胞疗法。通过一系列具体目标，我们将描述我们的 开发感兴趣的早期阶段实验室细胞群的过程，我们将解释成员如何 核心 C 将与研究人员合作，通过以下方式使基本实验室技术适应 cGMP 合规方法 cGMP 之前的“暂存区”。 Core C 成员将提供必要的文件来支持 FDA IND 提交，包括化学、制造和控制 (CMC) 部分的开发以及 相关文件。我们将描述三种细胞产品（T 调节细胞、记忆样 NK 细胞）的生产 细胞和 T 祖细胞），如项目 1 和 3 中所述。核心 C 还将确保适当的 产品和设施的质量标准。最后，Core C 将与项目和核心交互 研究人员，提供用于测试的细胞产品以及有关细胞递送、剂量和效力测定的建议 酌情发展。