罕见病细胞与基因疗法临床开发洞察——达舒创新设计科学工作组（IDSWG）著
Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases-by DahShu Innovative Design Scientific Working Group (IDSWG).
作者信息
Wang Chenkun, Di Junrui, Ghadessi Mercedeh, Tang Rui, Mulatya Caroline, Shi Daoyuan, Xu Tu, Wang Wenquan, Mei Chaoqun, Wang Susan, McComb Bryan, Beckman Robert A, McMillan Gianna
机构信息
Vertex Pharmaceuticals, Inc., 50 Northern Avenue, Boston, MA, 02210, USA.
Dexcom Inc., San Diego, CA, 02139, USA.
出版信息
Ther Innov Regul Sci. 2025 Aug 8. doi: 10.1007/s43441-025-00853-4.
The rapid advancement of cell and gene therapies (CGT) in the past ten years has inspired biopharmaceutical companies, biotechnologies, and nonprofits to tackle diseases that have traditionally been challenging to treat. Rare diseases, where roughly 80% have a genetic basis, have enjoyed this scrutiny, but the complexity of CGT trial design and implementation have proven challenging. This manuscript offers general guidance for CGT clinical development, current regulatory requirements and guidelines governed by FDA and EMA, considerations around preclinical development, safety monitoring and the need for long-term monitoring and follow up.
在过去十年中，细胞与基因疗法（CGT）的迅速发展激发了生物制药公司、生物技术公司和非营利组织攻克传统上难以治疗的疾病。罕见病（其中约80%有遗传基础）受到了这种审视，但CGT试验设计和实施的复杂性已被证明具有挑战性。本手稿提供了CGT临床开发的一般指南、美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）制定的现行监管要求和指南、临床前开发的考量因素、安全监测以及长期监测和随访的必要性。
Zotero 插件