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 先前来自 NIH SBIR 和库尔特基金会奖项的支持使 CryoCrate 团队能够开发出第一个- 一流的生物相容性冷冻保存介质产品，不需要任何细胞渗透性和毒性 冷冻保护剂。该产品的商标为 OdinSol®，并已被批准提供显着 提高细胞类型冷冻保存的效率，这对于基于细胞的过继免疫疗法至关重要。的 OdinSol® 技术还消除了对血清和人类产品的需求，并确保长期储存 在普通的 -80oC 冷冻柜中。 根据我们之前提交 FDA 510(k) 的经验，在这个项目中，我们组织了与 我们的监管顾问 (Cardinal Health)、合同制造组织 (Bryllan)、合同研究 组织（药明康德）、学术分包合作者（大学精准医学中心） 密苏里州），以实现以下里程碑： 一、OdinSol®的生产符合GMP资质及生物相容性评估表明 符合 FDA 关于作为可输注细胞存储设备提交 510(k) 的指导； 二.安全性和有效性的体外和体内评估表明 OdinSol® 技术是 在保留活力和功能方面优于现有的冷冻保存方案 T、NK 和 CAR-T 细胞； 三．根据这些资格，我们将完成 510(k) 提交并完成生产 线基于项目期结束前 FDA 的进一步反馈。 我们预计 OdinSol® 技术在获得 FDA 批准后三年内可用于未来的临床应用，从而提供 细胞治疗行业拥有高效的生物相容性低温储存平台。

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.