Translational Cell Therapy Core

摘要 我们对干细胞和淋巴细胞生物学的理解已经大大增加，包括 了解它们的发育、生长要求和功能。再加上重大改进， 在细胞分离和培养技术方面，这些进展为临床应用创造了令人兴奋的机会。 使用细胞疗法的转化研究。然而，技术和技术从一个 基础研究实验室到当前符合良好生产规范（cGMP）的实验室是复杂的， 细微差别，无论是基础/翻译研究人员还是临床医生都不熟悉FDA的要求。 Core C的主要功能是促进基本/平移观测的快速高效传输 转化为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 开发早期阶段的过程，实验室感兴趣的细胞群，我们将解释如何成员 核心C将与研究者合作，通过以下方式使基本实验室技术适应cGMP合规方法： 一个前cGMP“集结区”。核心C成员将提供必要的文件，以支持FDA IND提交，包括化学、生产和控制（CMC）部分的开发， 相关文件。我们将描述三种细胞产物（T调节细胞、记忆样NK细胞）的产生。 细胞和T祖细胞），其在项目1和3中描述。核心C还将确保适当的 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、给药和效价测定的建议 适当的发展。

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.