LABORATORY SCALE SYSTEM FOR CONTINUOUS PURIFICATION OF BIOPHARMACEUTICALS

项目摘要/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 这解决了工艺开发科学家和制造人员的未得到满足的需求，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支持 实验室规模的综合连续工艺。我们目前正专注于开发一种 我们的试验性PAKTM净化系统的实验室规模版本，是第一个商业完全连续的系统 允许同时运行最多4个单元操作的市场。这个实验室规模的版本将允许 科学家使用能够运行所有系统的单个系统快速高效地开发连续流程 通常在生物制造过程中使用的单元操作。批量生产是行业标准 在过去的几十年里进展甚微。这种制造模式需要资本密集型 不锈钢设施和低生产率(药品/时间/成本)。在台式规模上，科学家必须使用 相同的批处理技术由于其分段性质而减慢了过程开发时间 制造风格。这些研究不仅时间密集，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，许多连续的单元操作都是在 与批处理完全不同的方式(例如，流经病毒灭活和单次通过 透析过滤)。这些操作需要一个按比例缩小的连续系统才能正确开发。我们的目标是通过 这次融资机会是为了开发我们现有制造规模系统的实验室规模版本，以 在持续流程开发中使用。“微型PAK”系统将为工艺开发科学家提供 通过一个独特的工具来开发具有缩减的连续单元操作的连续流程。科学家们可以 信心十足地优化流程并快速扩展到生产规模，对 处理通道。Micro PAK节省的时间和资源将转化为更便宜的每克成本 制造成本，并增加连续流程成功放大的可能性 从开发到生产。几乎所有主要的生物制药都对连续加工感兴趣。 组织。一个微型PAK系统的预计成本为325,000美元，我们估计其年市场价值 9100万美元/年。为了让我们走上商业化的道路，我们建议首先开发一个功能原型 能够在每天1L的细胞培养中运行从最初捕获到最终药物物质的连续过程 比例。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来进行广泛的Beta测试 与我们的工艺规模相比，用小规模系统加工的物质的关键质量属性 所有单元操作的系统。

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.