Department of Chemical and Biological Engineering, Center for Pharmaceutical Biotechnology, University of Colorado Boulder, Boulder, Colorado.
Ursa Analytics, Denver, Colorado.
Biotechnol Bioeng. 2020 Nov;117(11):3322-3335. doi: 10.1002/bit.27501. Epub 2020 Jul 28.
Therapeutic proteins are exposed to numerous stresses during their manufacture, shipping, storage and administration to patients, causing them to aggregate and form particles through a variety of different mechanisms. These varied mechanisms generate particle populations with characteristic morphologies, creating "fingerprints" that are reflected in images recorded using flow imaging microscopy. Particle population fingerprints in test samples can be extracted and compared against those of particles produced under baseline conditions using an algorithm that combines machine learning tools such as convolutional neural networks with statistical tools such as nonparametric density estimation and Rosenblatt transform-based goodness-of-fit hypothesis testing. This analysis provides a quantitative method with user-specified type 1 error rates to determine whether the mechanisms that produce particles in test samples differ from particle formation mechanisms operative under baseline conditions. As a demonstration, this algorithm was used to compare particles within intravenous immunoglobulin formulations that were exposed to freeze-thawing and shaking stresses within a variety of different containers. This analysis revealed that seemingly subtle differences in containers (e.g., glass vials from different manufacturers) generated distinguishable particle populations after the stresses were applied. This algorithm can be used to assess the impact of process and formulation changes on aggregation-related product instabilities.
治疗性蛋白在制造、运输、储存和给患者施用的过程中会受到多种应激,导致它们通过各种不同的机制聚集并形成颗粒。这些不同的机制产生具有特征形态的颗粒群体,形成“指纹”,这些指纹反映在使用流动成像显微镜记录的图像中。可以从测试样品中提取颗粒群体指纹,并使用一种算法将其与在基线条件下产生的颗粒进行比较,该算法结合了机器学习工具(如卷积神经网络)和统计工具(如非参数密度估计和基于 Rosenblatt 变换的拟合优度假设检验)。这种分析提供了一种具有用户指定的第一类错误率的定量方法,以确定在测试样品中产生颗粒的机制是否与基线条件下起作用的颗粒形成机制不同。作为一个演示,该算法用于比较在各种不同容器中经历冷冻-解冻和摇晃应激的静脉内免疫球蛋白制剂中的颗粒。该分析表明,在施加应激后,容器(例如,来自不同制造商的玻璃小瓶)之间看似微小的差异会产生可区分的颗粒群体。该算法可用于评估工艺和配方变化对聚集相关产品不稳定性的影响。