大树创新设计科学工作组对罕见病细胞和基因治疗临床发展的见解

IF 1.9 4区医学 Q4 MEDICAL INFORMATICS

Therapeutic innovation & regulatory science Pub Date : 2025-08-08 DOI:10.1007/s43441-025-00853-4

Chenkun Wang, Junrui Di, Mercedeh Ghadessi, Rui Tang, Caroline Mulatya, Daoyuan Shi, Tu Xu, Wenquan Wang, Chaoqun Mei, Susan Wang, Bryan McComb, Robert A Beckman, Gianna McMillan

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引用次数: 0

摘要

在过去的十年里，细胞和基因疗法（CGT）的快速发展激发了生物制药公司、生物技术和非营利组织来解决传统上难以治疗的疾病。大约80%的罕见疾病具有遗传基础，它们受到了这种审查，但事实证明，CGT试验设计和实施的复杂性具有挑战性。本文提供了CGT临床开发的一般指导，FDA和EMA管理的当前监管要求和指南，临床前开发的考虑，安全监测以及长期监测和随访的需要。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases-by DahShu Innovative Design Scientific Working Group (IDSWG).

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.

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来源期刊

Therapeutic innovation & regulatory science MEDICAL INFORMATICS-PHARMACOLOGY & PHARMACY

CiteScore

3.40

自引率

13.30%

发文量

127

期刊介绍： Therapeutic Innovation & Regulatory Science (TIRS) is the official scientific journal of DIA that strives to advance medical product discovery, development, regulation, and use through the publication of peer-reviewed original and review articles, commentaries, and letters to the editor across the spectrum of converting biomedical science into practical solutions to advance human health. The focus areas of the journal are as follows: Biostatistics Clinical Trials Product Development and Innovation Global Perspectives Policy Regulatory Science Product Safety Special Populations