CAS Key Laboratory of Special Pathogens and Biosafety, Wuhan Institute of Virology, Center for Biosafety Mega-Science, Chinese Academy of Sciences, Wuhan, Hubei, China.
University of Chinese Academy of Sciences, Beijing, China.
MAbs. 2020 Jan-Dec;12(1):1689027. doi: 10.1080/19420862.2019.1689027.
The immunoglobulin (Ig) CH2 domain is a promising scaffold for the development of candidate therapeutics. We have previously shown that the stability of isolated CH2 could be increased by the introduction of an additional disulfide bond and removal of seven N-terminal residues (m01s). However, both isolated CH2 and m01s aggregate, likely due to the existence of aggregation-prone regions (APRs) that we identified by using computational methods. This knowledge was used to generate a phage display library of mutants. The library was incubated at high temperature to remove aggregating CH2 domains, and then panned against a mouse anti-human CH2 monoclonal antibody targeting a conformational epitope to remove misfolded CH2s. After two rounds of panning, one clone, m01s5, with smaller APRs, was identified. After additional mutagenesis one clone, m01s5.4, which aggregated much less than m01s as measured by a turbidity assay and dynamic light scattering, was identified. m01s5.4 also exhibited much lower nonspecific binding than m01s. Engineering of a previously identified m01s-based tumor antigen-specific binder led to a dramatic reduction of its aggregation without affecting its binding. In summary, we describe a new approach for reducing aggregation based on a combination of computational and phage display methodologies, and show that aggregation of CH2-based scaffolds can be significantly reduced by the newly identified mutants, which can improve the developability of potential CH2-based therapeutics.
免疫球蛋白 (Ig) CH2 结构域是开发候选治疗药物的有前途的支架。我们之前已经表明,通过引入额外的二硫键并去除七个 N 端残基 (m01s),可以提高分离的 CH2 的稳定性。然而,分离的 CH2 和 m01s 都容易聚集,这可能是由于我们使用计算方法确定的存在聚集倾向区域 (APR)。这一知识被用于生成突变体噬菌体展示文库。该文库在高温下孵育以去除聚集的 CH2 结构域,然后针对靶向构象表位的抗人 CH2 单克隆抗体进行淘选,以去除错误折叠的 CH2。经过两轮淘选,发现了一个 APR 较小的克隆 m01s5。经过进一步的诱变,发现了一个聚集性比 m01s 低得多的克隆 m01s5.4,这可以通过浊度测定法和动态光散射来衡量。m01s5.4 的非特异性结合也比 m01s 低得多。对之前鉴定的基于 m01s 的肿瘤抗原特异性结合物进行工程改造,在不影响其结合的情况下,大大降低了其聚集性。总之,我们描述了一种基于计算和噬菌体展示方法组合的减少聚集的新方法,并表明基于 CH2 的支架的聚集可以通过新鉴定的突变体显著降低,这可以提高潜在的基于 CH2 的治疗药物的开发性。