Nadofaragene firadenovec（nadofaragene firadenovec-vncg; Adstiladrin®）是一种非复制腺病毒载体基因治疗方法，由费林制药开发，用于治疗高危巴氏卡介苗（BCG）未响应的非肌层侵犯性膀胱癌（NMIBC）。Nadofaragene firadenovec由编码干扰素（IFN）-α2b的载体DNA组成，是膀胱癌中首个获批的基因治疗方法。转染的尿道上皮细胞产生的IFN-α2b与抗癌活性有关，包括免疫刺激、抗血管生成和凋亡效应。2022年12月，nadofaragene firadenovec在美国获得了首次全球批准，用于治疗成人高危BCG未响应的NMIBC（包括原位癌和有或无乳头肿瘤）。本文总结了nadofaragene firadenovec发展历程中的里程碑，导致了该适应症的首次批准。©2023.作者(s)在Springer Nature Switzerland AG独家许可下使用。

Nadofaragene firadenovec (nadofaragene firadenovec-vncg; Adstiladrin®) is a non-replicating adenoviral vector-based gene therapy developed by Ferring Pharmaceuticals for the treatment of high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC). Nadofaragene firadenovec constitutes vector DNA that encodes for interferon (IFN)-α2b and is the first approved gene therapy in bladder cancer. The production of IFN-α2b by transfected urothelial cells is associated with anticancer activity, including immunostimulatory, antiangiogenic and apoptotic effects. In December 2022, nadofaragene firadenovec received its first global approval in the USA for the treatment of high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours in adults. This article summarizes the milestones in the development of nadofaragene firadenovec leading to this first approval for this indication.© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.