Ravi Kant, Saurabh Mazumdar, Ananya Mahajan, Myron Christodoulides
{"title":"Artificial Intelligence-Integrated Proteome-Wide Target Prioritization for Drug Discovery against Multidrug-Resistant Neisseria gonorrhoeae","authors":"Ravi Kant, Saurabh Mazumdar, Ananya Mahajan, Myron Christodoulides","doi":"10.1021/acsinfecdis.6c00521","DOIUrl":null,"url":null,"abstract":"<p><em>Neisseria gonorrhoeae</em> causes ∼82.4 million\r\ninfections annually and is rapidly approaching an untreatable status.\r\nThe pathogen has developed resistance to every antibiotic class introduced\r\nsince the sulfonamide era. Ceftriaxone, the last WHO- and CDC-recommended\r\nempirical therapy, is now compromised by high-level resistance in\r\nextensively drug-resistant lineages reported across multiple continents.\r\nNo licensed vaccine exists, and no antibiotic with a novel mechanism\r\nhas been introduced for this pathogen in over 40 years. Conventional\r\ndrug discovery is too slow and insufficiently resistance-aware to\r\naddress this escalating threat. In this review, we examine in silico\r\nproteome-wide target fishing as a rapid, systematic approach. The\r\napproach applies six biologically informed filters to the <em>N. gonorrhoeae</em> core proteome: core-genome definition, host-homology\r\nexclusion, essentiality screening, metabolic chokepoint identification,\r\nsubcellular localization, and resistance-aware prioritization. Across\r\ndata sets spanning 12–69 clinical strains, this pipeline reduces\r\n2000–12,300 protein-coding sequences to 12–30 high-confidence\r\ntargets. An integrated artificial intelligence (AI)/machine learning\r\n(ML) scoring layer further ranks candidates using sequence features,\r\nAlphaFold2 structures, protein–protein interaction networks,\r\nand genotype–phenotype data from ∼20,000 clinical isolates.\r\nFive targets showed consistent cross-study prioritization: LpxC, MurA,\r\nFabI, DapD, and NGFG_RS03485. Of these, only LpxC has experimental\r\ninhibitor validation against multidrug-resistant gonococci; the remaining\r\ncandidates are supported computationally but lack biochemical confirmation.\r\nBridging this validation gap remains the critical bottleneck. However,\r\nadvances in pan-genome analysis, structural prediction, and generative\r\nAI for compound design provide a more tractable route to novel therapeutics\r\nthan previously available.</p>","PeriodicalId":17,"journal":{"name":"ACS Infectious Diseases","volume":"12 10","pages":"3296–3308"},"PeriodicalIF":4.5000,"publicationDate":"2026-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"ACS Infectious Diseases","FirstCategoryId":"3","ListUrlMain":"https://pubs.acs.org/doi/10.1021/acsinfecdis.6c00521","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2026/9/9 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"CHEMISTRY, MEDICINAL","Score":null,"Total":0}
引用次数: 0
Abstract
Neisseria gonorrhoeae causes ∼82.4 million
infections annually and is rapidly approaching an untreatable status.
The pathogen has developed resistance to every antibiotic class introduced
since the sulfonamide era. Ceftriaxone, the last WHO- and CDC-recommended
empirical therapy, is now compromised by high-level resistance in
extensively drug-resistant lineages reported across multiple continents.
No licensed vaccine exists, and no antibiotic with a novel mechanism
has been introduced for this pathogen in over 40 years. Conventional
drug discovery is too slow and insufficiently resistance-aware to
address this escalating threat. In this review, we examine in silico
proteome-wide target fishing as a rapid, systematic approach. The
approach applies six biologically informed filters to the N. gonorrhoeae core proteome: core-genome definition, host-homology
exclusion, essentiality screening, metabolic chokepoint identification,
subcellular localization, and resistance-aware prioritization. Across
data sets spanning 12–69 clinical strains, this pipeline reduces
2000–12,300 protein-coding sequences to 12–30 high-confidence
targets. An integrated artificial intelligence (AI)/machine learning
(ML) scoring layer further ranks candidates using sequence features,
AlphaFold2 structures, protein–protein interaction networks,
and genotype–phenotype data from ∼20,000 clinical isolates.
Five targets showed consistent cross-study prioritization: LpxC, MurA,
FabI, DapD, and NGFG_RS03485. Of these, only LpxC has experimental
inhibitor validation against multidrug-resistant gonococci; the remaining
candidates are supported computationally but lack biochemical confirmation.
Bridging this validation gap remains the critical bottleneck. However,
advances in pan-genome analysis, structural prediction, and generative
AI for compound design provide a more tractable route to novel therapeutics
than previously available.
期刊介绍:
ACS Infectious Diseases will be the first journal to highlight chemistry and its role in this multidisciplinary and collaborative research area. The journal will cover a diverse array of topics including, but not limited to:
* Discovery and development of new antimicrobial agents — identified through target- or phenotypic-based approaches as well as compounds that induce synergy with antimicrobials.
* Characterization and validation of drug target or pathways — use of single target and genome-wide knockdown and knockouts, biochemical studies, structural biology, new technologies to facilitate characterization and prioritization of potential drug targets.
* Mechanism of drug resistance — fundamental research that advances our understanding of resistance; strategies to prevent resistance.
* Mechanisms of action — use of genetic, metabolomic, and activity- and affinity-based protein profiling to elucidate the mechanism of action of clinical and experimental antimicrobial agents.
* Host-pathogen interactions — tools for studying host-pathogen interactions, cellular biochemistry of hosts and pathogens, and molecular interactions of pathogens with host microbiota.
* Small molecule vaccine adjuvants for infectious disease.
* Viral and bacterial biochemistry and molecular biology.