First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathway

Cyclin-dependent kinase 9 (CDK9) is a member of the transcription CDK subfamily and plays a role in transcriptional regulation. Selective CDK9 degraders possess potent clinical advantages over reversible CDK9 inhibitors. Herein, we report the first ATG101-recruiting selective CDK9 degrader, AZ-9, ba...

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Main Authors: Ye Zhong, Jing Xu, Huiying Cao, Jie Gao, Shaoyue Ding, Zhaohui Ren, Huali Yang, Yili Sun, Maosheng Cheng, Jia Li, Yang Liu
Format: Article
Language:English
Published: Elsevier 2025-05-01
Series:Acta Pharmaceutica Sinica B
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Online Access:http://www.sciencedirect.com/science/article/pii/S2211383525002151
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author Ye Zhong
Jing Xu
Huiying Cao
Jie Gao
Shaoyue Ding
Zhaohui Ren
Huali Yang
Yili Sun
Maosheng Cheng
Jia Li
Yang Liu
author_facet Ye Zhong
Jing Xu
Huiying Cao
Jie Gao
Shaoyue Ding
Zhaohui Ren
Huali Yang
Yili Sun
Maosheng Cheng
Jia Li
Yang Liu
author_sort Ye Zhong
collection DOAJ
description Cyclin-dependent kinase 9 (CDK9) is a member of the transcription CDK subfamily and plays a role in transcriptional regulation. Selective CDK9 degraders possess potent clinical advantages over reversible CDK9 inhibitors. Herein, we report the first ATG101-recruiting selective CDK9 degrader, AZ-9, based on the hydrophobic tag kinesin degradation technology. AZ-9 showed significant degradation effects and selectivity toward other homologous cell cycle CDKs in vitro and in vivo, which could also affect downstream related phenotypes. Mechanism research revealed that AZ-9 recruits ATG101 to initiate the autophagy–lysosome pathway, and forms autophagosomes through the recruitment of LC3, which then fuses with lysosomes to degrade CDK9 and the partner protein Cyclin T1. These dates validated the existence of non-proteasomal degradation pathway of hydrophobic driven protein degradation strategy for the first time, which might provide research ideas for chemical induction intervention on other types of pathogenic proteins.
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institution DOAJ
issn 2211-3835
language English
publishDate 2025-05-01
publisher Elsevier
record_format Article
series Acta Pharmaceutica Sinica B
spelling doaj-art-c816b57d82f745088bdcc01775d5871c2025-08-20T03:09:59ZengElsevierActa Pharmaceutica Sinica B2211-38352025-05-011552612262410.1016/j.apsb.2025.03.047First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathwayYe Zhong0Jing Xu1Huiying Cao2Jie Gao3Shaoyue Ding4Zhaohui Ren5Huali Yang6Yili Sun7Maosheng Cheng8Jia Li9Yang Liu10Key Laboratory of Structure-Based Drug Design & Discovery, Ministry of Education, Shenyang Pharmaceutical University, Shenyang 110016, ChinaDepartment of Pharmacology, Shenyang Pharmaceutical University, Shenyang 110016, China; Shandong Laboratory of Yantai Drug Discovery, Bohai Rim Advanced Research Institute for Drug Discovery, Yantai 264117, ChinaShandong Laboratory of Yantai Drug Discovery, Bohai Rim Advanced Research Institute for Drug Discovery, Yantai 264117, ChinaShandong Laboratory of Yantai Drug Discovery, Bohai Rim Advanced Research Institute for Drug Discovery, Yantai 264117, China; School of Pharmaceutical Science and Technology, Hangzhou Institute for Advanced Study, University of Chinese Academy of Sciences, Hangzhou 310024, ChinaKey Laboratory of Structure-Based Drug Design & Discovery, Ministry of Education, Shenyang Pharmaceutical University, Shenyang 110016, ChinaShandong Laboratory of Yantai Drug Discovery, Bohai Rim Advanced Research Institute for Drug Discovery, Yantai 264117, ChinaKey Laboratory of Structure-Based Drug Design & Discovery, Ministry of Education, Shenyang Pharmaceutical University, Shenyang 110016, ChinaShandong Laboratory of Yantai Drug Discovery, Bohai Rim Advanced Research Institute for Drug Discovery, Yantai 264117, China; State Key Laboratory of Chemical Biology, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China; Corresponding authors.Key Laboratory of Structure-Based Drug Design & Discovery, Ministry of Education, Shenyang Pharmaceutical University, Shenyang 110016, China; Corresponding authors.Department of Pharmacology, Shenyang Pharmaceutical University, Shenyang 110016, China; Shandong Laboratory of Yantai Drug Discovery, Bohai Rim Advanced Research Institute for Drug Discovery, Yantai 264117, China; State Key Laboratory of Chemical Biology, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China; Corresponding authors.Key Laboratory of Structure-Based Drug Design & Discovery, Ministry of Education, Shenyang Pharmaceutical University, Shenyang 110016, China; Corresponding authors.Cyclin-dependent kinase 9 (CDK9) is a member of the transcription CDK subfamily and plays a role in transcriptional regulation. Selective CDK9 degraders possess potent clinical advantages over reversible CDK9 inhibitors. Herein, we report the first ATG101-recruiting selective CDK9 degrader, AZ-9, based on the hydrophobic tag kinesin degradation technology. AZ-9 showed significant degradation effects and selectivity toward other homologous cell cycle CDKs in vitro and in vivo, which could also affect downstream related phenotypes. Mechanism research revealed that AZ-9 recruits ATG101 to initiate the autophagy–lysosome pathway, and forms autophagosomes through the recruitment of LC3, which then fuses with lysosomes to degrade CDK9 and the partner protein Cyclin T1. These dates validated the existence of non-proteasomal degradation pathway of hydrophobic driven protein degradation strategy for the first time, which might provide research ideas for chemical induction intervention on other types of pathogenic proteins.http://www.sciencedirect.com/science/article/pii/S2211383525002151Protein degradationHydrophobic tagsATG101Autophagy–lysosome pathwayCDK9Cyclin T1
spellingShingle Ye Zhong
Jing Xu
Huiying Cao
Jie Gao
Shaoyue Ding
Zhaohui Ren
Huali Yang
Yili Sun
Maosheng Cheng
Jia Li
Yang Liu
First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathway
Acta Pharmaceutica Sinica B
Protein degradation
Hydrophobic tags
ATG101
Autophagy–lysosome pathway
CDK9
Cyclin T1
title First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathway
title_full First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathway
title_fullStr First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathway
title_full_unstemmed First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathway
title_short First ATG101-recruiting small molecule degrader for selective CDK9 degradation via autophagy–lysosome pathway
title_sort first atg101 recruiting small molecule degrader for selective cdk9 degradation via autophagy lysosome pathway
topic Protein degradation
Hydrophobic tags
ATG101
Autophagy–lysosome pathway
CDK9
Cyclin T1
url http://www.sciencedirect.com/science/article/pii/S2211383525002151
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