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Generative AI Uncovers Novel Chrebp/Txnip Axis Inhibitors with Potential Anti-inflammatory Activity
Naila Qayyum1,2, Abdul Waheed Khan1, Muhammad Haseeb2
1Department of Molecular Science and Technology, Ajou University, Suwon 16499, Republic of Korea.
Abstract:
Type 2 diabetes is driven in part by metabolic inflammation, where activation of the Chrebp/Txnip axis promotes NLRP3 inflammasome assembly, leading to pancreatic β-cell dysfunction and pro-inflammatory cytokine release. Despite the therapeutic relevance of this pathway, the Chrebp-14-3-3 (regulatory-protein client) protein-protein interaction (PPI) remains largely underexplored, with only a limited number of small-molecule modulators reported to date. To address this gap, we developed an artificial intelligence-driven generative design framework for de novo discovery of selective PPI-targeting compounds. A conditional recurrent neural network (cRNN), implemented as a quantitative structure-property relationship-guided generative network (QSPR-GEN), was pretrained on a large, chemically diverse corpus to learn general SMILES syntax and structural priors, and subsequently fine-tuned on a curated, target-focused data set of approximately 5900 compounds, achieving high scaffold uniqueness (94.6%). Selectivity-oriented physicochemical descriptors were incorporated as conditional inputs to bias generation away from promiscuous chemotypes, while maintaining anchoring to a known active seed. Structure-based refinement was further applied by focusing on the noncanonical α-helical epitope unique to the Chrebp regulatory-protein interface, establishing a dual-layered strategy for selective PPI modulation. The integrated pipeline, combining virtual screening, molecular dynamics simulations, and MM/PBSA free-energy calculations, prioritized lead candidates with favorable binding energetics and pharmacokinetic profiles. In THP-1 macrophages under metabolic stress, the top candidate T7 markedly suppressed Txnip and NLRP3 expression, reduced IL-1β secretion, and attenuated pyroptotic cell death, outperforming a reference inhibitor. Collectively, this study presents a robust computational framework for the inverse design of challenging PPIs and demonstrates its utility through the identification and experimental validation of mechanistically precise lead compounds, exemplified by T2 and T7.
Insights
Artificial intelligence identified novel compounds targeting the Chrebp-14-3-3 protein-protein interaction, crucial for metabolic inflammation in type 2 diabetes. These compounds effectively reduced inflammation markers and protected pancreatic cells.
Area of Science:
- Biochemistry
- Computational Chemistry
- Pharmacology
Background:
- Type 2 diabetes involves metabolic inflammation driven by the Chrebp/Txnip axis, promoting NLRP3 inflammasome assembly and pancreatic beta-cell dysfunction.
- The Chrebp-14-3-3 protein-protein interaction (PPI) is a relevant therapeutic target but remains underexplored, with few known modulators.
Purpose of the Study:
- To develop an AI-driven generative design framework for de novo discovery of selective PPI-targeting compounds against the Chrebp-14-3-3 interaction.
- To identify and validate novel small-molecule modulators of this PPI for potential type 2 diabetes therapeutics.
Main Methods:
- Utilized a conditional recurrent neural network (QSPR-GEN) pretrained on a large corpus and fine-tuned on target-specific data for de novo compound generation.
- Incorporated selectivity-oriented physicochemical descriptors and structure-based refinement focusing on a unique alpha-helical epitope.
- Employed virtual screening, molecular dynamics, and MM/PBSA calculations to prioritize lead candidates with favorable energetics and pharmacokinetics.
Main Results:
- Achieved high scaffold uniqueness (94.6%) in generated compounds, biased away from promiscuous chemotypes.
- Prioritized lead candidates T2 and T7 with favorable binding and pharmacokinetic profiles.
- Candidate T7 significantly suppressed Txnip and NLRP3 expression, reduced IL-1β secretion, and attenuated pyroptotic cell death in macrophages under metabolic stress, outperforming a reference inhibitor.
Conclusions:
- Presented a robust computational framework for the inverse design of challenging PPIs.
- Demonstrated successful identification and experimental validation of mechanistically precise lead compounds (T2, T7) targeting the Chrebp-14-3-3 interaction.
- Highlighted the potential of AI-driven approaches for discovering novel therapeutics for metabolic diseases like type 2 diabetes.
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