The Ben May Department for Cancer Research , The University of Chicago , Chicago , Illinois 60637 , United States.
Department of Biochemistry and Molecular Biology , The University of Chicago , Chicago , Illinois 60637 , United States.
Biochemistry. 2019 Jul 9;58(27):2996-3004. doi: 10.1021/acs.biochem.9b00184. Epub 2019 Jun 19.
Anthrax, a lethal, weaponizable disease caused by Bacillus anthracis, acts through exotoxins that are primary mediators of systemic toxicity and also targets for neutralization by passive immunotherapy. The ease of engineering B. anthracis strains resistant to established therapy and the historic use of the microbe in bioterrorism present a compelling test case for platforms that permit the rapid and modular development of neutralizing agents. In vitro antigen-binding fragment (Fab) selection offers the advantages of speed, sequence level molecular control, and engineering flexibility compared to traditional monoclonal antibody pipelines. By screening an unbiased, chemically synthetic phage Fab library and characterizing hits in cell-based assays, we identified two high-affinity neutralizing Fabs, A4 and B7, against anthrax edema factor (EF), a key mediator of anthrax pathogenesis. Engineered homodimers of these Fabs exhibited potency comparable to that of the best reported neutralizing monoclonal antibody against EF at preventing EF-induced cyclic AMP production. Using internalization assays in COS cells, B7 was found to block steps prior to EF internalization. This work demonstrates the efficacy of synthetic alternatives to traditional antibody therapeutics against anthrax while also demonstrating a broadly generalizable, rapid, and modular screening pipeline for neutralizing antibody generation.
炭疽是由炭疽芽孢杆菌引起的一种致命的、可武器化的疾病,其作用机制是通过外毒素,这些外毒素是全身毒性的主要介质,也是被动免疫疗法中和的靶点。工程化对抗生素治疗有抗药性的炭疽菌株的容易性以及该微生物在生物恐怖主义中的历史应用,为能够快速和模块化开发中和剂的平台提供了一个极具说服力的案例。与传统的单克隆抗体管道相比,体外抗原结合片段 (Fab) 选择具有速度快、序列水平分子控制和工程灵活性的优势。通过筛选无偏的、化学合成的噬菌体 Fab 文库,并在基于细胞的测定中对命中物进行表征,我们鉴定了两种针对炭疽水肿因子 (EF) 的高亲和力中和 Fab,A4 和 B7,EF 是炭疽病发病机制的关键介质。这些 Fab 的工程同源二聚体在预防 EF 诱导的环 AMP 产生方面表现出与针对 EF 的最佳报道中和单克隆抗体相当的效力。使用 COS 细胞中的内化测定,发现 B7 阻断 EF 内化之前的步骤。这项工作证明了针对炭疽的合成替代传统抗体疗法的有效性,同时还展示了一种广泛适用于中和抗体生成的快速和模块化筛选管道。