Innovation of Biobanking for Cellular Adoptive Immunotherapies Based on a First-in-Class Biocompatible Cryopreservation Technology for T and NK Cells and Their CAR-Engineered Products

摘要 之前获得NIH SBIR和库尔特基金会奖的支持使CryoCrate团队能够开发出第一个- 同类生物相容冷冻保存介质产品，不需要任何细胞渗透和毒性 冷冻保护剂。该产品注册为OdinSol®，并已获得批准提供显著的 提高以细胞为基础的过继免疫疗法所必需的细胞类型的冷冻存储效率。这个 OdinSol®技术还消除了对血清和人体产品的要求，并确保了长期储存 在普通的-80摄氏度的冷冻室里。 基于我们以前在FDA 510(K)提交方面的经验，在这个项目中，我们组织了与 我们的监管顾问(Cardinal Health)、代工组织(Bryllan)、合同研究 组织(无锡AppTec)，学术分包协作者(大学精准医学中心 密苏里州)，以实现以下里程碑： I.OdinSol®的生产符合GMP资格和生物兼容性评估显示 遵守FDA关于将510(K)作为可输血细胞储存装置提交的指南； 体外和体内的安全性和有效性评估表明，OdinSol®技术是 在保存活性和功能方面优于现有的冷冻保存协议 T细胞、NK细胞和CAR-T细胞； 根据这些资格，我们将完成510(K)提交并最终确定产品 在项目期结束之前，应根据FDA的进一步反馈确定项目。 我们预计在FDA批准后三年内，OdinSol®技术将在临床上使用，提供 拥有高效生物兼容冷冻存储平台的细胞治疗行业。

SUMMARY Previous support from NIH SBIR and Coulter Foundation awards allowed the CryoCrate team to develop a first- in-class biocompatible cryopreservation medium product that doesn’t need any cell permeating and toxic cryoprotectant. The product is trademarked as OdinSol®, and has been approved to provide significantly improved efficiency in cryostorage of cell types that are essential for cell-based adoptive immunotherapies. The OdinSol® technology also removes the requirement of serum and human products and secures long-term storage in regular -80oC deep freezers. Based on our former experience in FDA 510(k) submission, in this project, we organized the collaboration with our regulatory consultant (Cardinal Health), contract manufacturing organizations (Bryllan), contract research organization (WuXi AppTec), academic subcontract collaborator (the Center for Precision Medicine at University of Missouri), to achieve the following milestones: I. The production of OdinSol® meets GMP qualifications and biocompatibility assessment shows compliance with FDA guidance for 510(k) submission as a storage device for transfusable cells; II. The in vitro and in vivo evaluation of safety and efficacy demonstrates the OdinSol® technology to be superior to existing cryopreservation protocols with respect to preserving viability and functionalities of T, NK and CAR-T cells; III. Based on those qualifications, we will complete of the 510(k) submission and finalize of the production line based on further FDA feedback before the end of the project period. We expect future clinical use of the OdinSol® technology within three years after the FDA clearance, providing the industry of cell therapies with a highly efficient biocompatible cryostorage platform.