CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injury

Acute liver injury (ALI) serves as a critical precursor and major etiological factor in the progression and ultimate manifestation of various hepatic disorders. The prevention and treatment of ALI is still a serious global challenge. Given the limited therapeutic options for ALI, exploring novel tar...

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Main Authors: Yong Chen, Xue Yuan, Wei Yan, Yurong Zou, Haoche Wei, Yuhan Wei, Minghai Tang, Yulian Chen, Ziyan Ma, Tao Yang, Kongjun Liu, Baojian Xiong, Xiuying Hu, Jianhong Yang, Lijuan Chen
Format: Article
Language:English
Published: Elsevier 2025-07-01
Series:Acta Pharmaceutica Sinica B
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Online Access:http://www.sciencedirect.com/science/article/pii/S2211383525003090
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author Yong Chen
Xue Yuan
Wei Yan
Yurong Zou
Haoche Wei
Yuhan Wei
Minghai Tang
Yulian Chen
Ziyan Ma
Tao Yang
Kongjun Liu
Baojian Xiong
Xiuying Hu
Jianhong Yang
Lijuan Chen
author_facet Yong Chen
Xue Yuan
Wei Yan
Yurong Zou
Haoche Wei
Yuhan Wei
Minghai Tang
Yulian Chen
Ziyan Ma
Tao Yang
Kongjun Liu
Baojian Xiong
Xiuying Hu
Jianhong Yang
Lijuan Chen
author_sort Yong Chen
collection DOAJ
description Acute liver injury (ALI) serves as a critical precursor and major etiological factor in the progression and ultimate manifestation of various hepatic disorders. The prevention and treatment of ALI is still a serious global challenge. Given the limited therapeutic options for ALI, exploring novel targeted therapeutic agents becomes imperative. The potential therapeutic efficacy of inhibiting RIPK2 is highlighted, as it may provide significant benefits by attenuating the MAPK pathway and NF-κB signaling. Herein, we propose a CMD-OPT model, a two-stage molecular optimization tool for the rapid discovery of RIPK2 inhibitors with optimal properties. Compound RP20, which targets the ATP binding site, demonstrated excellent kinase specificity, ideal oral pharmacokinetics, and superior therapeutic effects in a model of APAP-induced ALI, positioning RP20 as a promising preclinical candidate. This marks the first application of RIPK2 inhibitors in ALI treatment, opening a novel therapeutic pathway for clinical applications. These results highlight the efficacy of the CMD-OPT model in producing lead compounds from known active molecules, showcasing its significant potential in drug discovery.
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issn 2211-3835
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publisher Elsevier
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series Acta Pharmaceutica Sinica B
spelling doaj-art-db9950af2c1047f2a93ca804bc2c734e2025-08-20T02:41:32ZengElsevierActa Pharmaceutica Sinica B2211-38352025-07-011573708372410.1016/j.apsb.2025.05.003CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injuryYong Chen0Xue Yuan1Wei Yan2Yurong Zou3Haoche Wei4Yuhan Wei5Minghai Tang6Yulian Chen7Ziyan Ma8Tao Yang9Kongjun Liu10Baojian Xiong11Xiuying Hu12Jianhong Yang13Lijuan Chen14State Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China; Innovation Center of Nursing Research and Nursing Key Laboratory of Sichuan Province, West China Hospital, Sichuan University/West China School of Nursing, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaState Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, ChinaInnovation Center of Nursing Research and Nursing Key Laboratory of Sichuan Province, West China Hospital, Sichuan University/West China School of Nursing, Sichuan University, Chengdu 610041, China; Corresponding authors.State Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China; Corresponding authors.State Key Laboratory of Biotherapy, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China; Chengdu Zenitar Biomedical Technology Co., Ltd., Chengdu 610212, China; Corresponding authors.Acute liver injury (ALI) serves as a critical precursor and major etiological factor in the progression and ultimate manifestation of various hepatic disorders. The prevention and treatment of ALI is still a serious global challenge. Given the limited therapeutic options for ALI, exploring novel targeted therapeutic agents becomes imperative. The potential therapeutic efficacy of inhibiting RIPK2 is highlighted, as it may provide significant benefits by attenuating the MAPK pathway and NF-κB signaling. Herein, we propose a CMD-OPT model, a two-stage molecular optimization tool for the rapid discovery of RIPK2 inhibitors with optimal properties. Compound RP20, which targets the ATP binding site, demonstrated excellent kinase specificity, ideal oral pharmacokinetics, and superior therapeutic effects in a model of APAP-induced ALI, positioning RP20 as a promising preclinical candidate. This marks the first application of RIPK2 inhibitors in ALI treatment, opening a novel therapeutic pathway for clinical applications. These results highlight the efficacy of the CMD-OPT model in producing lead compounds from known active molecules, showcasing its significant potential in drug discovery.http://www.sciencedirect.com/science/article/pii/S2211383525003090CMD-OPTAcute liver injuryRIPK2 inhibitorsAnti-inflammatoryDrug discoveryCo-crystal
spellingShingle Yong Chen
Xue Yuan
Wei Yan
Yurong Zou
Haoche Wei
Yuhan Wei
Minghai Tang
Yulian Chen
Ziyan Ma
Tao Yang
Kongjun Liu
Baojian Xiong
Xiuying Hu
Jianhong Yang
Lijuan Chen
CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injury
Acta Pharmaceutica Sinica B
CMD-OPT
Acute liver injury
RIPK2 inhibitors
Anti-inflammatory
Drug discovery
Co-crystal
title CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injury
title_full CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injury
title_fullStr CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injury
title_full_unstemmed CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injury
title_short CMD-OPT model enables the discovery of a potent and selective RIPK2 inhibitor as preclinical candidate for the treatment of acute liver injury
title_sort cmd opt model enables the discovery of a potent and selective ripk2 inhibitor as preclinical candidate for the treatment of acute liver injury
topic CMD-OPT
Acute liver injury
RIPK2 inhibitors
Anti-inflammatory
Drug discovery
Co-crystal
url http://www.sciencedirect.com/science/article/pii/S2211383525003090
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