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.
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页码：157 / 158
页数：2
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