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

CPSI Biotech是一家专注于设备设计的技术开发公司， 支持生物医学和生物加工行业的分子调节策略。 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。 包括胰腺癌在内的各种癌症的消融。这种独特的医疗设备现在正在 在约翰霍普金斯医学院进行测试。鉴于有利的冷冻和工程 特性以及性能配置文件相比，可比系统，CPSI已重新- 将PSN技术应用到SmartFreeze设备中，以快速有效地 人类细胞和组织的冷冻保存超出了今天的可能性。CPSI还建立了一个 原型设备SmartThaw，专为增强型、可记录和可编程的干燥而设计 解冻人类细胞产品。该系统的设计使其具有优于 传统的水浴解冻，如操作员/软件控制设计， 程序.此外，SmartThaw Gen 7显示解冻后活力增加3倍 的CHO细胞相比，标准水浴-一个结果，很好地证明了改善后， 解冻结果时，适用于干细胞。SmartFreeze和SmartThaw设备 共同组成了SmartBio平台。独特的功能和可记录的性质 在FDA批准的干细胞IND中唯一定位SmartBio用于细胞处理。 鉴于SmartBio的潜力，在这个项目中，我们将运用我们丰富的经验， 知识基础上的细胞生物学，低温生物学和低温工程开发一个新的类， 能够快速和受控地冷冻和解冻人体的装置和策略 造血干细胞（hHSC）。第1阶段的具体目标如下：SA 1-开发 SmartThaw系统用于改进hHSC的解冻。SA 2-将SmartThaw连接到 SmartFreeze系统开发细胞特异性冷冻和解冻曲线， hHSC活力。SA 3-测试靶向和改善激活的选择细胞应激途径的药物 在冻融过程后的hHSC中; SA 4-分析功能性和 在最佳冷冻/解冻过程之后的hHSC的分化。第二阶段将侧重于 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。的总体目标 SmartBio项目旨在将hHSC解冻后结局比当前行业改善≥50% 标准这个建议代表了一种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控制， 专门为细胞治疗竞技场设计的可记录的冷冻和解冻系统。

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.