当前位置：
X-MOL 学术
›
J. Med. Chem.
›
论文详情
Our official English website, www.x-mol.net, welcomes your
feedback! (Note: you will need to create a separate account there.)
CC-90009: A Cereblon E3 Ligase Modulating Drug That Promotes Selective Degradation of GSPT1 for the Treatment of Acute Myeloid Leukemia
Journal of Medicinal Chemistry ( IF 6.8 ) Pub Date : 2021-02-16 , DOI: 10.1021/acs.jmedchem.0c01489 Joshua D. Hansen 1 , Matthew Correa 1 , Matt Alexander 1 , Mark Nagy 1 , Dehua Huang 1 , John Sapienza 1 , Gang Lu 1 , Laurie A. LeBrun 1 , Brian E. Cathers 1 , Weihong Zhang 2 , Yang Tang 1 , Massimo Ammirante 1 , Rama K. Narla 1 , Joseph R. Piccotti 1 , Michael Pourdehnad 3 , Antonia Lopez-Girona 1
Journal of Medicinal Chemistry ( IF 6.8 ) Pub Date : 2021-02-16 , DOI: 10.1021/acs.jmedchem.0c01489 Joshua D. Hansen 1 , Matthew Correa 1 , Matt Alexander 1 , Mark Nagy 1 , Dehua Huang 1 , John Sapienza 1 , Gang Lu 1 , Laurie A. LeBrun 1 , Brian E. Cathers 1 , Weihong Zhang 2 , Yang Tang 1 , Massimo Ammirante 1 , Rama K. Narla 1 , Joseph R. Piccotti 1 , Michael Pourdehnad 3 , Antonia Lopez-Girona 1
Affiliation
|
Acute myeloid leukemia (AML) is marked by significant unmet clinical need due to both poor survival and high relapse rates where long-term disease control for most patients with relapsed or refractory AML remain dismal. Inspired to bring novel therapeutic options to these patients, we envisioned protein degradation as a potential therapeutic approach for the treatment of AML. Following this course, we discovered and pioneered a novel mechanism of action which culminated in the discovery of CC-90009. CC-90009 represents a novel protein degrader and the first cereblon E3 ligase modulating drug to enter clinical development that specifically targets GSPT1 (G1 to S phase transition 1) for proteasomal degradation. This manuscript briefly summarizes the mechanism of action, scientific rationale, medicinal chemistry, pharmacokinetic properties, and efficacy data for CC-90009, which is currently in phase 1 clinical development.
中文翻译：
CC-90009：促进GSPT1选择性降解的Cereblon E3连接酶调节药物，用于治疗急性髓细胞性白血病
急性骨髓性白血病（AML）的特点是生存率低和复发率高，因此尚未满足临床上的巨大需求，而大多数复发或难治性AML患者的长期疾病控制仍然不佳。启发给这些患者带来新的治疗选择，我们设想蛋白质降解是治疗AML的潜在治疗方法。遵循这一过程，我们发现并开创了一种新颖的作用机制，最终发现了CC-90009。CC-90009代表一种新型蛋白降解剂，也是首个进入临床开发的脑神经E3连接酶调节药物，该药物专门针对蛋白酶体降解靶向GSPT1（G1到S相转变1）。本手稿简要总结了作用机理，科学原理，药物化学，药代动力学特性，
更新日期：2021-02-25
中文翻译：
CC-90009：促进GSPT1选择性降解的Cereblon E3连接酶调节药物，用于治疗急性髓细胞性白血病
急性骨髓性白血病（AML）的特点是生存率低和复发率高，因此尚未满足临床上的巨大需求，而大多数复发或难治性AML患者的长期疾病控制仍然不佳。启发给这些患者带来新的治疗选择，我们设想蛋白质降解是治疗AML的潜在治疗方法。遵循这一过程，我们发现并开创了一种新颖的作用机制，最终发现了CC-90009。CC-90009代表一种新型蛋白降解剂，也是首个进入临床开发的脑神经E3连接酶调节药物，该药物专门针对蛋白酶体降解靶向GSPT1（G1到S相转变1）。本手稿简要总结了作用机理，科学原理，药物化学，药代动力学特性，
京公网安备 11010802027423号