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.

项目总结/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的未满足需求， 药物开发时间表和制造成本。没有供应商提供的系统能够完全 在实验室规模的集成连续工艺。我们目前专注于开发一种 我们的Pilot PAKTM净化系统的实验室规模版本，第一个商用全连续系统 该市场允许多达4个单元操作同时运行。这种实验室规模的版本将允许 科学家们可以快速有效地开发一个连续的过程，使用一个单一的系统，能够运行所有 生物制造过程中常用的单元操作。批量生产是行业标准 在过去的几十年里几乎没有进展。这种制造模式需要资本密集型 不锈钢设施，导致生产率低（药物/时间/成本）。在实验室规模上，科学家必须使用 相同的批处理技术，由于其分段的性质， 制造风格。这些研究不仅耗时，而且不能准确反映复杂的过程 在一个连续的过程中发现的动力学。此外，许多连续单元操作在一个或多个连续单元中操作。 与它们的批处理对应物根本不同的方式（例如，流过病毒灭活和单程 渗滤）。这些操作需要按比例缩小的连续系统来适当地开发。我们的目标是通过 这个资金的机会是开发一个实验室规模的版本，我们现有的生产规模的系统， 用于连续工艺开发。“微型PAK”系统将为工艺开发科学家提供 使用独特的工具开发具有按比例缩小的连续单元操作的连续工艺。科学家可以 自信地优化工艺，并快速扩大到生产规模， 加工方式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，并增加了成功扩大连续工艺的可能性， 发展到生产。连续处理已经引起几乎所有主要生物制药公司的兴趣。 组织的微型PAK系统的预计成本为325，000美元，我们估计每年的市场价值 91 M/年。为了使我们走上商业化的道路，我们建议首先开发一个功能原型 能够在1 L细胞培养物/天上运行从初始捕获到最终原料药的连续工艺 规模在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 系统然后，我们将通过一系列实验设计研究进行广泛的beta测试， 与我们的工艺规模相比，使用小规模系统处理的物质的关键质量属性 所有单位的业务。