The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

被引：6

作者：

Brooks, P. J. ^{[1
]}

Miller, Timothy M. ^{[2
]}

Revah, Frederic ^{[3
]}

Suh, Junghae ^{[4
]}

Garrison, Bradley R. ^{[5
]}

Starke, Lawrence C. ^{[6
]}

MacLachlan, Timothy K. ^{[7
]}

Neilan, Edward G. ^{[8
]}

Raychaudhuri, Gopa ^{[9
]}

Kassim, Sadik H. ^{[10
]}

Dehdashti, Jean ^{[1
]}

Rutter, Joni L. ^{[1
]}

机构：

[1] NIH, Natl Ctr Adv Translat Sci, Bethesda, MD 20892 USA

[2] Thermo Fisher Sci, Waltham, MA USA

[3] Genethon, Evry, France

[4] Biogen Inc, Cambridge, MA USA

[5] Fdn Natl Inst Hlth, North Bethesda, MD USA

[6] Novartis Pharmaceut, E Hanover, NJ USA

[7] Novartis, Biomed Res, Cambridge, MA USA

[8] Natl Org Rare Disorders, Quincy, MA USA

[9] US FDA, Ctr Biol Evaluat & Res, Silver Spring, MD USA

[10] Danaher Corp, Washington, DC USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2024年 / 23卷 / 03期

关键词：

D O I：

10.1038/d41573-024-00020-8

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

A public-private partnership managed by the FNIH aims to address biological, manufacturing and regulatory challenges to the development of gene therapies for rare diseases. A public-private partnership managed by the FNIH aims to address biological, manufacturing and regulatory challenges to the development of gene therapies for rare diseases.

引用

页码：157 / 158

页数：2

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