LABORATORY SCALE SYSTEM FOR CONTINUOUS PURIFICATION OF BIOPHARMACEUTICALS

用于生物制药连续纯化的实验室规模系统

• 批准号: 10480679
• 负责人: Joanna Frances Rucker Pezzini
• 金额: $ 32.5万
• 依托单位: PAK BIOSOLUTIONS, INC.
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-06-06 至 2024-06-05
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10480679
• 关键词: 3D Print; Adoption; Automation; Biological Products; Biomanufacturing; Bioreactors; Capital; Cell Culture Techniques; Cells; Characteristics; Chromatography; Clinical; Complex; Cost Savings; Development; Education and Outreach; Engineering; Ensure; Equipment; Excision; Feasibility Studies; Filtration; Funding Opportunities; Goals; Housing; Human Resources; Industry; Industry Standard; Infrastructure; International; Laboratories; Life; Liquid substance; Methods; Microfluidics; Modality; Nature; Outcome; Penetration; Performance; Pharmaceutical Preparations; Phase; Price; Process; Production; Productivity; Resources; Risk; Running; Sales; Scientist; Series; Site; Small Business Innovation Research Grant; Stainless Steel; System; Techniques; Technology; Test Result; Testing; Time; TimeLine; Translating; Vendor; Virus Inactivation; base; biopharmaceutical industry; bioprocess; commercial application; commercialization; cost; design; digital; drug development; experimental study; feasibility testing; improved; interest; manufacturing process; next generation; novel; operation; pressure; process optimization; prototype; scale up; tool

PROJECT SUMMARY/ABSTRACT PAK BioSolutions, Inc. (PAK Bio) is an early-stage company creating continuous bio-manufacturing equipment that solves the unmet needs of process development scientists and manufacturing personnel aiming to shorten drug development timelines and manufacturing costs. There are no vendor supplied systems that enable a fully integrated continuous process at the laboratory scale. We are currently focused on the development of a laboratory scale version of our Pilot PAKTM purification system, the first commercial fully continuous system on the market that enables up to 4 unit operations to be run simultaneously. This laboratory scale version will allow scientists to quickly and efficiently develop a continuous process using a single system capable of running all unit operations commonly used in a bio-manufacturing process. Batch manufacturing is the industry standard and has progressed little in the past few decades. This manufacturing modality requires capital-intensive stainless-steel facilities and results in low productivity (drug/time/cost). At benchtop scale, scientists must use the same batch techniques which slow down process development time due to the segmented nature of this manufacturing style. These studies are not only time intensive, but do not accurately reflect the complex process dynamics found in a continuous process. Additionally, many continuous unit operations operate in a fundamentally different way than their batch counterparts (e.g., flow through virus inactivation and single pass diafiltration). These operations require a scaled down continuous system to properly develop. Our aim through this funding opportunity is to develop a laboratory scale version of our existing manufacturing scale system for use in continuous process development. The “Micro PAK” system would provide process development scientists with a unique tool to develop a continuous process with scaled down continuous unit operations. Scientists could confidently optimize the process and quickly scale up to manufacturing scale with minimal to no changes in the processing modality. Time and resources saved by the Micro PAK would translate into cheaper $/gram manufacturing costs and increase the likelihood of successful scale up of a continuous process from development to production. Continuous processing has been of interest to nearly all major biopharmaceutical organizations. With a projected cost of a Micro PAK system to be $325,000, we estimate an annual market value of $91M/year. To set us on the path towards commercialization, we propose to first develop a functional prototype capable of running a continuous process from initial capture through final drug substance on a 1L cell culture/day scale. After successful completion of this Phase I feasibility study PAK Bio will further optimize and validate the system. We will then conduct extensive beta testing through a series of Design of Experiments studies comparing critical quality attributes of substances processed with the small-scale system compared to our process scale system on all unit operations.

项目概要/摘要 PAK BioSolutions, Inc. (PAK Bio) 是一家创建连续生物制造设备的早期公司 解决了工艺开发科学家和制造人员未满足的需求，旨在缩短 药物开发时间表和制造成本没有供应商提供的系统可以完全实现。 我们目前专注于实验室规模的集成连续工艺的开发。 我们的 Pilot PAKTM 纯化系统的实验室规模版本，是世界上第一个商业化的全连续系统 该市场允许同时运行多达 4 个单元操作。 科学家们使用能够运行所有功能的单一系统快速有效地开发连续流程 生物制造过程中常用的单元操作是批量制造的行业标准。 这种制造方式需要资本密集型，但在过去几十年里进展甚微。 不锈钢设施导致生产力低下（药物/时间/成本），科学家必须在实验室规模上使用。 由于其分段性质，相同的批次技术会减慢工艺开发时间 这些研究不仅耗时，而且不能准确反映复杂的过程。 此外，许多连续的单元操作都是在连续过程中进行的。 与批量复制完全不同的方式（例如，通过病毒灭活和单次传递 渗滤）。这些操作需要缩小规模的连续系统才能正确发展。 该资助机会是开发我们现有制造规模系统的实验室规模版本 “Micro PAK”系统将为工艺开发科学家提供连续工艺开发的使用。 科学家们可以通过一种独特的工具来开发连续过程，并缩小连续单元操作的规模。 自信地优化流程并快速扩大到制造规模，而对 Micro PAK 节省的时间和资源将转化为更便宜的美元/克。 制造成本并增加了连续工艺成功扩大规模的可能性 几乎所有主要生物制药公司都对连续加工感兴趣。 Micro PAK 系统的预计成本为 325,000 美元，我们估计年度市场价值。 为了让我们走上商业化之路，我们建议首先开发一个功能原型。 能够每天在 1L 细胞培养物上运行从初始捕获到最终药物的连续过程 成功完成第一阶段可行性研究后，PAK Bio 将进一步优化和验证该项目。 然后，我们将通过一系列实验设计研究进行广泛的测试比较。 与我们的工艺规模相比，使用小规模系统加工的物质的关键质量属性 所有单位操作的系统。