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

CPSI Biotech是一家专注于设备和设备设计的技术开发公司 支持生物医药和生物加工业的分子调控战略。 通过这些努力，我们已经开发出用于靶向的PSN和SCN冷冻消融系统 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被 在约翰·霍普金斯医学院进行了测试。考虑到有利的冰冻和工程 与同类系统相比，CPSI具有更高的性能 将PSN技术集成到SmartFreeze设备中，以实现快速有效的 超低温保存人类细胞和组织，这是今天所不可能的。CPSI还建立了一个 原型设备SmartThw，专为增强的、可记录的和可编程的DRY而设计 人类细胞产品的解冻。这个系统的设计是这样设计的，它比 传统的水浴解冻，如操作员/软件控制设计，具有独特的解冻效果 程序。此外，SmartThaw Gen7显示解冻后的生存能力提高了3倍 将CHO细胞与标准水浴进行比较--这一结果很好地证明了改进后的 当应用于干细胞时，解冻结果。SmartFreeze和SmartThw设备 共同组成了SmartBio平台。独一无二的特点和可记录的性质 将SmartBio定位为包括在FDA批准的干细胞IND中进行细胞处理的独特位置。 鉴于SmartBio的潜力，在这个项目下，我们将应用我们丰富的经验和 基于细胞生物学、低温生物学和低温工程的知识开发新一类 使人类能够快速和受控地冷冻和解冻的装置和策略 造血干细胞(HHSC)。阶段1的具体目标如下：SA1-制定 用于改善hHSCs解冻的SmartThaw系统。SA2-将SmartThaw连接到 SmartFreeze系统开发特定于细胞的冷冻和解冻配置文件，从而实现最佳 HHSC活性。SA3-靶向和改善激活的选定细胞应激途径的测试试剂 在冻结和解冻过程后的hHSC中；SA4-Analyze功能和 最佳冷冻/解冻过程后hHSC的分化。第二阶段将重点放在 设备优化，为SmartBio开发特定于HHSC的冷冻保存策略 系统，执行寻的研究和随后的Beta测试。该计划的总体目标 SmartBio项目是将HHSC解冻后的结果比目前的行业提高≥50% 标准的。这一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，将产生第一个受控和 专门为细胞治疗领域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.