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的技术开发公司 分子调制策略支持生物医学和生物处理行业。 通过这些努力，我们开发了针对目标的PSN和SCN冷冻系统 包括胰腺癌在内的各种癌症的消融。现在，这种独特的医疗设备正在 在约翰·霍普金斯医学院测试。鉴于有利的冻结和工程 与可比系统相比，特征和性能概况，CPSI具有 将PSN技术的工具设置为SmartFreeze，以快速有效 对人类细胞和组织的冷冻保存超出了当今可能。 CPSI也建立了 原型设备，SmartThaw，专为增强，可记录和可编程的干燥设计 解冻人类细胞产品。该系统的设计使其具有优势 传统的水浴融化，例如用于独特解冻的操作员/软件控制设计 程序。此外，SmartThaw Gen7表现出3倍的速度生存能力增加 与标准水浴相比 将结果融化，当应用于干细胞。 SmartFreeze和SmartThaw设备 集体组成SmartBio平台。独特的功能和可记录的性质 独特地将SMARTBIO定位，以纳入FDA认可的干细胞IND中的细胞处理。 鉴于Smartbio的潜力，在此项目下，我们将运用我们的丰富经验， 细胞生物学，冷冻生物学和冷冻工程的知识基础，以开发新的类别 设备和策略，以使人类快速而受控的冰冻和融化 造血干细胞（HHSC）。第1阶段的特定目的如下：SA1-开发 改善HHSC的SmartThaw系统。 SA2-将SmartThaw搭配到 SmartFreeze系统开发细胞特定的冷冻和解冻轮廓，从而最佳 HHSC的生存能力。 SA3-靶向和改善精选细胞应力途径激活的测试剂 在冷冻和解冻过程之后的HHSC中； SA4-分析功能和 最佳冻结/解冻过程之后的HHSC的分化。第2阶段将重点关注 设备优化，为SmartBio制定HHSC特定的冷冻保存策略 系统，进行归巢研究以及随后的Beta测试。总体目标 SmartBio项目将与当前行业相比，将HHSC的HHSC后结果提高≥50％ 标准。该建议代表了产品开发耦合的一种综合方法 工程和生命科学将产生第一个串联操作员的控制和 可记录的冻结和融化系统专为细胞疗法而设计。