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Kymriah®(tisagenlecleucel):首个获批 CAR-T 疗法临床开发历程概述

英文原题:Kymriah® (tisagenlecleucel) - An overview of the clinical development journey of the first approved CAR-T therapy.

PubMed 2023/05/15(内容时间) Hum Vaccin Immunother Q2 · IF 4.2(JCR 2025)

研究概要

细胞与基因疗法的出现极大地改变了肿瘤学及其他治疗领域的治疗格局。

中文摘要

细胞和基因疗法的出现显著改变了肿瘤学及其他治疗领域的治疗范式。Kymriah(tisagenlecleucel)是一种靶向 CD19 的基因修饰自体 T 细胞免疫疗法,目前已在主要市场获批,用于治疗复发/难治性(r/r)儿童和青年成人急性淋巴细胞白血病、r/r 弥漫大 B 细胞淋巴瘤及 r/r 滤泡性淋巴瘤。本文概述 tisagenlecleucel 的临床开发历程,包括疗效结局和安全性考量。

展开英文摘要原文

The emergence of cell and gene therapies has dramatically changed the treatment paradigm in oncology and other therapeutic areas. Kymriah (tisagenlecleucel), a CD19-directed genetically modified autologous T-cell immunotherapy, is currently approved in major markets for the treatment of relapsed/refractory (r/r) pediatric and young adult acute lymphoblastic leukemia, r/r diffuse large B-cell lymphoma, and r/r follicular lymphoma. This article presents a high-level overview of the clinical development journey of tisagenlecleucel, including its efficacy outcomes and safety considerations.

论文信息

作者
Awasthi R、Maier HJ、Zhang J、Lim S
第一作者单位
Oncology & Hematology, Novartis Institutes for BioMedical Research, East Hanover, NJ, USA.United States
通讯作者单位
US Medical Affairs, Oncology, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.United States
期刊
Human vaccines & immunotherapeutics2023 Dec 31
原文标识
PubMed 37185251 · DOI 10.1080/21645515.2023.2210046