Translational Cell Therapy Core

摘要 我们对干细胞和淋巴细胞生物学的了解有了显著的提高，包括 关于它们的发展、生长要求和功能的知识。再加上显著的改进 在细胞分离和培养技术方面，这些进展为临床创造了令人兴奋的机会 使用细胞疗法的转译研究。然而，技术和技术的过渡从一个 基础研究实验室到目前的良好制造合规(CGMP)实验室是复杂的和 细微差别，基础/翻译研究人员和临床医生都不太熟悉FDA的要求。 核心C的主要功能是促进基本/转换观测的快速高效传输 变成有意义和有效的细胞疗法。通过一系列具体目标，我们将描述我们的 发展早期感兴趣的实验室细胞群体的过程，我们将解释成员如何 Core C将与调查人员合作，通过以下方式使基本实验室技术适应cGMP合规方法学 CGMP前的“集结区”。核心C的成员将提供必要的文件来支持FDA IND提交的材料，包括化学、制造和控制(CMC)部分的开发和 相关文件。我们将描述三种细胞产品的生产(T调节细胞，记忆样NK 细胞和T祖细胞)。核心C还将确保适当 产品和设施的质量标准。最后，核心C将与项目和核心进行交互 研究人员，提供用于测试的细胞产品以及关于细胞递送、剂量和效力分析的建议 视情况发展。

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.