TOPIC 397 - MICROFLUIDIC VORTEX SHEDDING: A LOW-COST, HIGH EFFICIENCY METHOD FOR GENETIC MODIFICATION TO SUPPORT CELL ENGINEERING FOR CELL-BASED IMMUNOTHERAPIES

这份合同提案的目标是提炼和商业化水孔，这是一种低成本、高效率的方法，以支持基于细胞的癌症免疫疗法的制造。实现这一目标将导致一种技术，从早期到临床研究，使细胞疗法的开发现代化，从而加快向有需要的患者提供治疗。在第一阶段，水孔技术在改进CAR-T细胞的制造工艺方面证明是可行的。在第二阶段，该提案计划(1)横向扩展水力孔性能，以优化构建基因编辑CAR-T细胞的效率，(2)通过开发具有&gt；5 x 10^8单元处理能力的大型水力孔消耗性设备来扩大水力孔容量，以及(3)验证水力孔平台和过程分析。经过处理的细胞将通过既定的方法进行严格的评估，包括流式细胞术、体外分析和体内(小鼠)功效研究。还将建立质量控制参数。最终交付产品定义为：(1)用于大容量细胞治疗应用的可稳定处理5x10^8T细胞的水力孔仪器和设备，以及(2)制造水力孔仪器和消耗品的标准操作程序。

The objective of this contract proposal is to refine and commercialize Hydropore, a low cost, high efficiency method to support manufacture of cell-based cancer immunotherapies. Meeting this objective will result in a technology that will modernize cell therapy development from early stage through clincical studies, thereby expediting the delivery of treatments to patients in need. In Phase 1 the Hydropore technology demonstrated feasability in improving the manufacturing processes of CAR-T cells. In Phase II the proposal plans to (1) scale-out Hydropore performance to optimize the efficiency of constructing gene-edited CAR-T cells, (2) scale-up Hydropore capacity with the development of a large scale Hydropore consumbable device, with >5 x 10^8 cell processing capability, and (3) validate the Hydropore platform and process analytics. Processed cells will be rigorously evaluated through established approaches, including flow cytometery, in vitro assays, and in vivo (mouse) efficacy studies. Quality control parameters will also be established. The final deliverable is defined as (1) Hydropore instrumentation and devices capable of robustly processing >5 x 10^8 T cells for large volume cell therapy applications and (2) Standard Operating Procedures for manufacturing Hydropore instruments and consumables.