Xu Yan, Anirudhan Varada, Gaisina Irina N, Du Haijuan, Alqarni Saad, Moore Terry W, Caffrey Michael, Manicassamy Balaji, Zhou Tongqing, Rong Lijun, Xu Kai
Department of Veterinary Biosciences, The Ohio State University, Columbus, OH 43210.
Department of Microbiology and Immunology, University of Illinois, Chicago, IL 60612.
Proc Natl Acad Sci U S A. 2025 Aug 19;122(33):e2503899122. doi: 10.1073/pnas.2503899122. Epub 2025 Aug 13.
Influenza A virus (IAV) is a zoonotic pathogen responsible for seasonal and pandemic flu. The extensive genetic and antigenic diversity within and between IAV phylogenetic groups presents major challenges for developing universal vaccines and broad-spectrum antiviral therapies. Current interventions provide limited protection due to the virus's high mutation rate and capacity for immune evasion. Recent advancements in viral hemagglutinin (HA)-targeting small-molecule entry inhibitors offer a promising avenue to overcome these limitations. Here, we present structural and functional analyses of two group 2 HA-specific small-molecule inhibitors recently identified by our team. Cryogenic electron microscopy (cryo-EM) structures revealed that these inhibitors bind a conserved pocket within the HA stalk, likely interfering with the conformational rearrangements necessary for membrane fusion and viral entry. Structure-guided mutagenesis confirmed the critical roles of key interacting residues and uncovered distinct resistance profiles between the two compounds, as well as in comparison to Arbidol, a previously reported HA inhibitor. Notably, our structural analysis highlights intrinsic barriers to achieving cross-group inhibition with current small-molecule designs. To address this, we propose an alternative strategy for broadening antiviral coverage. Together, these findings provide mechanistic insights into IAV entry inhibition and a foundation for the rational design of next-generation anti-influenza therapeutics.
甲型流感病毒(IAV)是一种人畜共患病原体,可引发季节性流感和大流行性流感。IAV系统发育组内部和之间广泛的遗传和抗原多样性,给开发通用疫苗和广谱抗病毒疗法带来了重大挑战。由于病毒的高突变率和免疫逃逸能力,目前的干预措施提供的保护有限。针对病毒血凝素(HA)的小分子进入抑制剂的最新进展,为克服这些限制提供了一条有前景的途径。在此,我们展示了我们团队最近鉴定的两种2组HA特异性小分子抑制剂的结构和功能分析。低温电子显微镜(cryo-EM)结构表明,这些抑制剂结合在HA茎干内的一个保守口袋中,可能会干扰膜融合和病毒进入所需的构象重排。结构导向诱变证实了关键相互作用残基的关键作用,并揭示了这两种化合物之间以及与先前报道的HA抑制剂阿比朵尔相比的不同耐药谱。值得注意的是,我们的结构分析突出了当前小分子设计在实现跨组抑制方面的内在障碍。为了解决这个问题,我们提出了一种扩大抗病毒覆盖范围的替代策略。总之,这些发现为IAV进入抑制提供了机制见解,并为下一代抗流感疗法的合理设计奠定了基础。