The SmartBio System for the Improved Preservation of Human Hematopoietic Stem Cells

用于改善人类造血干细胞保存的 SmartBio 系统

• 批准号: 9228432
• 负责人: John M BAUST
• 金额: $ 26.4万
• 依托单位: CELL PRESERVATION SERVICES, INC.
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-09-13 至 2018-07-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9228432
• 关键词: Ablation; Area; Bathing; Binding; Biochemical; Biological Assay; Biological Preservation; Biological Sciences; Biotechnology; Blood; Cell Death; Cell Survival; Cell Therapy; Cell physiology; Cells; Cellular Stress; Cellular biology; Characteristics; Chinese Hamster Ovary Cell; Clinical; Computer software; Coupling; Cryopreservation; Cryopreserved Cell; Cryosurgery; Data; Development; Device Designs; Device or Instrument Development; Devices; Documentation; Engineering; FDA approved; Freezing; Future; Goals; Hematopoietic stem cells; Homing; Human; Human Characteristics; Human Resources; Industry; Industry Standard; Investigation; Letters; Malignant Neoplasms; Malignant neoplasm of pancreas; Marketing; Medical; Medical Device; Molecular; Molecular Analysis; Nature; Outcome; Pathway interactions; Performance; Phase; Play; Positioning Attribute; Procedures; Process; Protocols documentation; Publications; Reagent; Reproducibility; Research; Sampling; Series; Stem cells; Stress; System; Technology; Testing; Time; Tissues; Vial device; Water; base; biological research; bioprocess; conditioning; cost effective; cryobiology; design; experience; improved; improved outcome; in vivo; innovation; instrument; interest; knowledge base; medical schools; novel; phase 1 study; product development; programs; prototype; response; success; targeted agent; technology development; user-friendly

CPSI Biotech is a technology development company focused on the design of devices and molecular modulation strategies in support of the biomedical and bioprocessing industries. Through these efforts we have developed the PSN and SCN cryoablation systems for targeted ablation of various cancers including pancreatic cancer. This unique medical device is now being tested at Johns Hopkins School of Medicine. Given the favorable freezing and engineering characteristics as well as performance profiles compared to comparable systems, CPSI has re- tooled the PSN technology into a device, SmartFreeze, for the rapid and effective cryopreservation of human cells and tissues beyond what is possible today. CPSI has also built a prototype device, SmartThaw, designed for the enhanced, documentable and programmable dry thawing of human cell products. This system is designed such that it has advantages over traditional water bath thawing such as operator/software control design for unique thawing programs. Furthermore, SmartThaw Gen7 demonstrates a 3 fold increase in post-thaw viability of CHO cells compared to a standard water bath - a result that argues well for improved post- thaw outcome when applied to stem cells. The SmartFreeze and SmartThaw devices collectively make up the SmartBio platform. The unique features and documentable nature uniquely position the SmartBio for inclusion for cell processing in FDA approved stem cell INDs. Given the potential of SmartBio, under this project we will apply our extensive experience and knowledge base on the cell biology, cryobiology and cryoengineering to develop a new class of devices and strategies to enable the rapid and controlled freezing and thawing of human hematopoietic stem cells (hHSCs). Phase 1 Specific Aims are the following: SA1 - Develop the SmartThaw system for the improved thawing of hHSCs. SA2 - Couple the SmartThaw to the SmartFreeze system developing cell specific freezing and thawing profiles that result in optimal hHSC viability. SA3 - Test agents that target and ameliorate select cell stress pathways activated in hHSCs following the freezing and thawing process; SA4 - Analyze functionality and differentiation of hHSCs subsequent to the optimal freeze/thaw process. Phase 2 will focus on device optimization, developing an hHSC-specific cryopreservation strategy for the SmartBio system, performing homing studies and subsequent beta testing. The overall goal of the SmartBio project is to improve hHSC post-thaw outcome by ≥50% over the current industry standard. This proposal represents an integrative approach to product development coupling engineering and life sciences that will yield the first tandem operator controlled and documentable freezing and thawing systems specifically designed for the cell therapy arena.

CPSI Biotech 是一家专注于设备设计和 支持生物医学和生物加工行业的分子调制策略。 通过这些努力，我们开发了 PSN 和 SCN 冷冻消融系统，用于靶向治疗 这种独特的医疗设备目前正在消融各种癌症，包括胰腺癌。 鉴于有利的冷冻和工程，在约翰霍普金斯大学医学院进行了测试。 与同类系统相比，CPSI 已重新 将 PSN 技术融入到 SmartFreeze 设备中，以实现快速有效的 CPSI 还建立了超越当今可能的人体细胞和组织冷冻保存技术。 原型设备 SmartThaw，专为增强型、可记录和可编程的干燥 该系统的设计使其具有优于人类细胞产品的优点。 传统的洗澡水解冻，例如用于独特解冻的操作员/软件控制设计 在进一步的项目中，SmartThaw Gen7 的解冻后活力提高了 3 倍 与标准水浴相比，CHO 细胞的细胞数 - 这一结果充分证明了后处理的改善 应用于干细胞时的解冻结果。 共同构成了SmartBio平台的独特功能和可记录性。 独特地将 SmartBio 纳入 FDA 批准的干细胞 IND 中的细胞处理。 鉴于 SmartBio 的潜力，在这个项目下，我们将运用我们丰富的经验和 以细胞生物学、低温生物学和低温工程为基础的知识库，开发一类新的 能够快速、受控冷冻和解冻人体的装置和策略 造血干细胞 (hHSC) 第一阶段的具体目标如下： SA1 - 开发 用于改善 hHSC 解冻的 SmartThaw 系统 - 将 SmartThaw 连接到 SA2。 SmartFreeze 系统开发细胞特异性冷冻和解冻曲线，从而获得最佳效果 hHSC 活力。SA3 - 针对和改善激活的选定细胞应激途径的测试药物。 在冷冻和解冻过程后的 hHSC 中 - 分析功能和 最佳冷冻/解冻过程之后 hHSC 的分化将集中于第二阶段。 设备优化，为 SmartBio 开发 hHSC 特异性冷冻保存策略 系统，执行归航研究和随后的 Beta 测试。 SmartBio 项目旨在将 hHSC 解冻后结果比当前行业提高 ≥50% 该提案代表了产品开发耦合的综合方法。 工程和生命科学将产生第一个串联操作员 专为细胞治疗领域设计的可记录冷冻和解冻系统。