基因和细胞疗法可持续性的挑战和解决方案

IF 51.4 1区生物学 Q1 GENETICS & HEREDITY Nature Reviews Genetics Pub Date : 2025-03-10 DOI:10.1038/s41576-025-00827-0

Celeste Scotti, Alessandro Aiuti, Luigi Naldini

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引用次数: 0

摘要

基因和细胞疗法的前景已经成为几种毁灭性疾病的临床现实。然而，重大障碍限制了它们的经济可持续性，并危及它们在药品市场上的生存，特别是罕见疾病的药品，这需要创新的解决办法。尽管针对罕见疾病的基因和细胞疗法潜力巨大，但由于若干挑战，它们面临着退出市场的风险。这组作者描述了这些障碍，并呼吁在监管机构批准后采取创新措施来提高基因和细胞疗法的经济可持续性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Challenges and solutions to the sustainability of gene and cell therapies

The promise of gene and cell therapy has become a clinical reality for several devastating diseases. However, major hurdles constrain their economic sustainability and endanger their survival on the drug market, especially those for rare diseases, which calls for innovative solutions. Despite their immense potential, gene and cell therapies that target rare diseases are at risk of market withdrawal, owing to several challenges. The authors describe these hurdles and call for innovative measures to improve the economic sustainability of gene and cell therapies after regulatory approval.

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来源期刊

Nature Reviews Genetics 生物-遗传学

CiteScore

57.40

自引率

0.50%

发文量

113

审稿时长

6-12 weeks

期刊介绍： At Nature Reviews Genetics, our goal is to be the leading source of reviews and commentaries for the scientific communities we serve. We are dedicated to publishing authoritative articles that are easily accessible to our readers. We believe in enhancing our articles with clear and understandable figures, tables, and other display items. Our aim is to provide an unparalleled service to authors, referees, and readers, and we are committed to maximizing the usefulness and impact of each article we publish. Within our journal, we publish a range of content including Research Highlights, Comments, Reviews, and Perspectives that are relevant to geneticists and genomicists. With our broad scope, we ensure that the articles we publish reach the widest possible audience. As part of the Nature Reviews portfolio of journals, we strive to uphold the high standards and reputation associated with this esteemed collection of publications.

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