血液系统恶性肿瘤治疗的突破：2024年美国血液学会（ASH）关于分子胶、蛋白水解靶向嵌合体（PROTACs）和RNA降解剂的最新进展
Breakthroughs in treatment for hematological malignancies: latest updates on molecular glue, PROTACs and RNA degraders from ASH 2024.
作者信息
Li Na, Sheng Jianpeng, Zhu Hong-Hu
机构信息
Department of Hematology, Beijing Chao-Yang Hospital, Capital Medical University, No. 8 Gongren Tiyuchang Nanlu, Chaoyang District, Beijing, 100020, China.
Chinese Institutes for Medical Research, Capital Medical University, Beijing, 100020, China.
出版信息
J Hematol Oncol. 2025 Mar 5;18(1):26. doi: 10.1186/s13045-025-01674-6.
Degrader therapies have garnered significant attention for their innovative approach to targeting and eliminating malignancy-associated proteins, holding promise for improving outcomes for patients with relapsed or refractory (R/R) hematological malignancies, especially in cases of leukemia, non-Hodgkin lymphoma, and multiple myeloma. Currently, the main categories developed based on degraders include molecular glue (such as Cemsidomide, NX-5948), PROTACs (such as BGB-16673, AC-676, KT-333 ), and RNA degraders (such as SKY-1214). This correspondence summarizes the preclinical and clinical updates on degrader therapies presented at the ASH 2024 annual meeting.
降解剂疗法因其靶向和消除恶性肿瘤相关蛋白的创新方法而备受关注，有望改善复发或难治性（R/R）血液系统恶性肿瘤患者的预后，尤其是白血病、非霍奇金淋巴瘤和多发性骨髓瘤患者。目前，基于降解剂开发的主要类别包括分子胶（如Cemsidomide、NX-5948）、PROTACs（如BGB-16673、AC-676、KT-333）和RNA降解剂（如SKY-1214）。本通讯总结了在2024年ASH年会上展示的降解剂疗法的临床前和临床进展。
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