应对商业挑战的平台解决方案，以扩大患者获取和使基因编辑可持续发展

IF 33.1 1区生物学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Nature biotechnology Pub Date : 2025-07-15 DOI:10.1038/s41587-025-02744-3

Sadik H. Kassim, Fyodor Urnov, Kiran Musunuru, Ann Lee, Luis Barrera, Pamela Stetkiewicz, Julianne Bruno, Matthew Hewitt, Troy Lister, Harry Malech, Lindsay Gasch, Matt Diver, Nicholas Gertler, Felix Grignon, Audrey Le, Michael Lehmicke, Edwin M. Horwitz, David R. Liu, Josephine Lembong, Vanessa Almendro-Navarro

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引用次数: 0

摘要

基于平台的基因编辑治疗方法可以显著提高开发效率，降低成本并增加罕见疾病患者的可及性。尽管基因编辑在少数孟德尔病适应症上取得了显著的临床成功，但更广泛的采用面临着巨大的障碍。我们提出了克服这些挑战的策略，包括非临床和化学、制造和控制（CMC）数据重用的模块化平台，基于风险的制造质量，以及简化的伞形临床试验，以提高监管效率和加速审批。

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Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable

Platform-based approaches for gene-editing therapies could markedly improve development efficiency, reduce costs and increase access for patients with rare diseases. Although gene editing has shown remarkable clinical success for a small number of Mendelian disease indications, broader adoption faces substantial hurdles. We propose strategies to overcome these challenges through modular platforms for nonclinical and chemistry, manufacturing and controls (CMC) data reuse, risk-based manufacturing quality, and streamlined umbrella clinical trials for regulatory efficiency and accelerated approval.

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来源期刊

Nature biotechnology 工程技术-生物工程与应用微生物

CiteScore

63.00

自引率

1.70%

发文量

382

审稿时长

3 months

期刊介绍： Nature Biotechnology is a monthly journal that focuses on the science and business of biotechnology. It covers a wide range of topics including technology/methodology advancements in the biological, biomedical, agricultural, and environmental sciences. The journal also explores the commercial, political, ethical, legal, and societal aspects of this research. The journal serves researchers by providing peer-reviewed research papers in the field of biotechnology. It also serves the business community by delivering news about research developments. This approach ensures that both the scientific and business communities are well-informed and able to stay up-to-date on the latest advancements and opportunities in the field. Some key areas of interest in which the journal actively seeks research papers include molecular engineering of nucleic acids and proteins, molecular therapy, large-scale biology, computational biology, regenerative medicine, imaging technology, analytical biotechnology, applied immunology, food and agricultural biotechnology, and environmental biotechnology. In summary, Nature Biotechnology is a comprehensive journal that covers both the scientific and business aspects of biotechnology. It strives to provide researchers with valuable research papers and news while also delivering important scientific advancements to the business community.