Wang Sijie, Faucher Franco F, Bertolini Matilde, Kim Heeyoung, Yu Bingchen, Cao Li, Roeltgen Katharina, Lovell Scott, Shanker Varun, Boyd Scott D, Wang Lei, Bartenschlager Ralf, Bogyo Matthew
Department of Pathology, School of Medicine, Stanford University, California 94305, United States.
Department of Chemistry, School of Humanities and Sciences, Stanford University, California 94305, United States.
bioRxiv. 2024 Nov 9:2024.11.08.622749. doi: 10.1101/2024.11.08.622749.
Peptide macrocycles are promising therapeutics for a variety of disease indications due to their overall metabolic stability and potential to make highly selective binding interactions with targets. Recent advances in covalent macrocycle peptide discovery, driven by phage and mRNA display methods, have enabled the rapid identification of highly potent and selective molecules from large libraires of diverse macrocycles. However, there are currently limited examples of macrocycles that can be used to disrupt protein-protein interactions and even fewer examples that function by formation of a covalent bond to a target protein. In this work, we describe a directed counter-selection method that enables identification of covalent macrocyclic ligands targeting a protein-protein interaction using a phage display screening platform. This method utilizes binary and ternary screenings of a chemically modified phage display library, employing the stable and weakly reactive aryl fluorosulfate electrophile. We demonstrate the utility of this approach using the SARS-CoV-2 Spike-ACE2 protein-protein interaction and identify multiple covalent macrocyclic inhibitors that disrupt this interaction. The resulting compounds displayed antiviral activity against live virus that was irreversible after washout due to the covalent binding mechanism. These results highlight the potential of this screening platform for developing covalent macrocyclic drugs that disrupt protein-protein interactions with long lasting effects.
由于其整体代谢稳定性以及与靶点进行高度选择性结合相互作用的潜力,肽大环化合物是多种疾病适应症的有前景的治疗药物。由噬菌体和mRNA展示方法驱动的共价大环肽发现的最新进展,使得能够从大量不同大环化合物库中快速鉴定出高效且选择性的分子。然而,目前可用于破坏蛋白质-蛋白质相互作用的大环化合物实例有限,通过与靶蛋白形成共价键发挥作用的实例更少。在这项工作中,我们描述了一种定向反选方法,该方法能够使用噬菌体展示筛选平台鉴定靶向蛋白质-蛋白质相互作用的共价大环配体。该方法利用化学修饰的噬菌体展示文库进行二元和三元筛选,采用稳定且反应性较弱的芳基氟硫酸盐亲电试剂。我们使用SARS-CoV-2刺突蛋白-血管紧张素转换酶2(Spike-ACE2)蛋白质-蛋白质相互作用证明了这种方法的实用性,并鉴定出多种破坏这种相互作用的共价大环抑制剂。由于共价结合机制,所得化合物对活病毒显示出抗病毒活性,洗脱后这种活性是不可逆的。这些结果突出了该筛选平台在开发具有持久作用的破坏蛋白质-蛋白质相互作用的共价大环药物方面的潜力。