Mieczkowski Carl, Cheng Alan, Fischmann Thierry, Hsieh Mark, Baker Jeanne, Uchida Makiko, Raghunathan Gopalan, Strickland Corey, Fayadat-Dilman Laurence
Discovery Biologics, Protein Sciences, Merck & Co., Inc., South San Francisco, CA 94080, USA.
Discovery Chemistry, Modeling and Informatics, Merck & Co., Inc., South San Francisco, CA 94080, USA.
Antibodies (Basel). 2021 Feb 15;10(1):8. doi: 10.3390/antib10010008.
Reversible antibody self-association, while having major developability and therapeutic implications, is not fully understood or readily predictable and correctable. For a strongly self-associating humanized mAb variant, resulting in unacceptable viscosity, the monovalent affinity of self-interaction was measured in the low μM range, typical of many specific and biologically relevant protein-protein interactions. A face-to-face interaction model extending across both the heavy-chain (HC) and light-chain (LC) Complementary Determining Regions (CDRs) was apparent from biochemical and mutagenesis approaches as well as computational modeling. Light scattering experiments involving individual mAb, Fc, Fab, and Fab'2 domains revealed that Fabs self-interact to form dimers, while bivalent mAb/Fab'2 forms lead to significant oligomerization. Site-directed mutagenesis of aromatic residues identified by homology model patch analysis and self-docking dramatically affected self-association, demonstrating the utility of these predictive approaches, while revealing a highly specific and tunable nature of self-binding modulated by single point mutations. Mutagenesis at these same key HC/LC CDR positions that affect self-interaction also typically abolished target binding with notable exceptions, clearly demonstrating the difficulties yet possibility of correcting self-association through engineering. Clear correlations were also observed between different methods used to assess self-interaction, such as Dynamic Light Scattering (DLS) and Affinity-Capture Self-Interaction Nanoparticle Spectroscopy (AC-SINS). Our findings advance our understanding of therapeutic protein and antibody self-association and offer insights into its prediction, evaluation and corrective mitigation to aid therapeutic development.
可逆抗体自缔合虽然具有重大的可开发性和治疗意义,但尚未得到充分理解,也难以轻易预测和校正。对于一种强烈自缔合的人源化单克隆抗体变体,其导致了不可接受的粘度,自相互作用的单价亲和力在低微摩尔范围内测得,这是许多特异性且与生物学相关的蛋白质-蛋白质相互作用的典型情况。从生化和诱变方法以及计算建模中可以明显看出,一种跨越重链(HC)和轻链(LC)互补决定区(CDR)的面对面相互作用模型。涉及单个单克隆抗体、Fc、Fab和Fab'2结构域的光散射实验表明,Fab会自相互作用形成二聚体,而二价单克隆抗体/Fab'2形式会导致显著的寡聚化。通过同源模型补丁分析和自对接鉴定出的芳香族残基的定点诱变极大地影响了自缔合,证明了这些预测方法的实用性,同时揭示了由单点突变调节的自结合具有高度特异性和可调节性。在这些影响自相互作用的相同关键HC/LC CDR位置进行诱变通常也会消除靶标结合,但有明显例外,这清楚地证明了通过工程校正自缔合的困难和可能性。在用于评估自相互作用的不同方法之间也观察到了明显的相关性,例如动态光散射(DLS)和亲和捕获自相互作用纳米颗粒光谱(AC-SINS)。我们的研究结果推进了我们对治疗性蛋白质和抗体自缔合的理解,并为其预测、评估和校正缓解提供了见解,以帮助治疗性开发。