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

主题 397 - 微流控涡流脱落：一种低成本、高效的基因修饰方法，支持基于细胞的免疫疗法的细胞工程

• 批准号: 10708268
• 负责人: RYAN PAWELL
• 金额: $ 199.96万
• 依托单位: INDEE, INC
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-09-19 至 2024-09-18
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10708268
• 关键词: Cell Therapy; Cell Volumes; Cell physiology; Cells; Cellular immunotherapy; Computer Vision Systems; Computer software; Contracts; Development; Devices; Electrodes; Genes; Genetic; Gold; Knock-in; Knock-out; Measures; Methods; Microfluidics; Microscopy; Modernization; Modification; Mus; Optics; Patients; Performance; Phase; Phenotype; Procedures; Process; Quality Control; Signal Transduction; Supporting Cell; T-Lymphocyte; Technology; Width; base; cancer immunotherapy; cellular engineering; chimeric antigen receptor T cells; cost; design; efficacy study; improved; in vitro Assay; in vivo; instrument; instrumentation; manufacturing process; meetings; prototype; scale up; therapy development

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.

该合同提案的目标是改进和商业化Hydropore，这是一种低成本、高效率的方法，用于支持基于细胞的癌症免疫疗法的制造。实现这一目标将产生一种技术，使细胞治疗从早期发展到临床研究现代化，从而加快向有需要的患者提供治疗。在第一阶段，Hydropore技术证明了改善CAR-T细胞制造工艺的可行性。在第二阶段，该提案计划(1)扩大Hydropore性能，以优化构建基因编辑CAR-T细胞的效率；(2)扩大Hydropore容量，开发大型Hydropore消耗品设备，具有bb50 × 10^8的细胞处理能力；(3)验证Hydropore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、体外试验和体内（小鼠）功效研究。还将建立质量控制参数。最终交付的产品定义为：(1)能够稳健地处理bbbb5 × 10^8 T细胞的大容量细胞治疗应用的Hydropore仪器和设备；(2)制造Hydropore仪器和耗材的标准操作程序。