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1.发明申请
公开(公告)号：WO2019067840A1
公开(公告)日：2019-04-04
申请号：PCT/US2018/053312
申请日：2018-09-28
申请人： VOYAGER THERAPEUTICS, INC. , INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , UNIVERSITÉ DE STRASBOURG
发明人： SAH, Dinah Wen-Yee , GOULET, Martin , PATZKE, Holger , SHU, Yanqun , HOU, Jinzhao , PUCCIO, Hélène
IPC分类号： A61K48/00 , C07K14/47 , C12N7/00 , C12N15/113 , C12N15/86 , C12N15/861
摘要： Provided herein is an adeno-associated virus (AAV) particle comprising a capsid and a viral genome, wherein said capsid delivers the AAV particle to a nervous system, and wherein said viral genome comprises a polynucleotide sequence encoding Frataxin and one or more microRNA binding sites. Also, provided herein is a method for treating, ameliorating, and/or preventing a disorder in a subject stemming from a loss or partial loss of frataxin protein in the subject, wherein the method comprises: administering to the subject a therapeutically effective amount of a composition comprising an AAV particle comprising a capsid and a viral genome, wherein said capsid delivers the viral genome to a nervous system, and wherein said viral genome comprises a polynucleotide sequence encoding Frataxin and one or more microRNA binding sites, as described herein.
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公开(公告)号：WO2019028306A3
公开(公告)日：2019-02-07
申请号：PCT/US2018/045088
申请日：2018-08-03
发明人： SAH, Dinah Wen-Yee , PATZKE, Holger , HOU, Jinzhao , NONNENMACHER, Mathieu E. , GOULET, Martin , CARTER, Todd
摘要： The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.
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公开(公告)号：WO2017075338A2
公开(公告)日：2017-05-04
申请号：PCT/US2016/059302
申请日：2016-10-28
发明人： SAH, Dinah Wen-Yee , HOU, Jinzhao , GOULET, Martin , KELLS, Adrian Philip , ZHOU, Pengcheng , STEWART, Gregory Robert
IPC分类号： A61K48/00 , C12N15/861
CPC分类号： C07K14/47 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： The invention relates to compositions and methods for the preparation, administration, manufacture and therapeutic use of viral particles for the treatment of CNS disorders.
摘要翻译： 本发明涉及用于治疗CNS病症的病毒颗粒的制备，给药，制造和治疗用途的组合物和方法。
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公开(公告)号：WO2016115503A1
公开(公告)日：2016-07-21
申请号：PCT/US2016/013671
申请日：2016-01-15
发明人： STEWART, Gregory Robert , SAH, Dinah Wen-Yee , HOU, Jinzhao , KELLS, Adrian Philip , GOULET, Martin , RAMAMOORTHI, Kartik , ZHOU, Pengcheng
IPC分类号： C07K14/005 , A61K31/7088
CPC分类号： A61K31/7088 , A61K48/005 , A61K48/0075 , A61K48/0083 , C07K14/47 , C12N2750/14143
摘要： The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding payloads for the treatment of CNS disorders.
摘要翻译： 本发明涉及用于制备，制造和治疗使用编码用于治疗CNS障碍的有效载荷的多核苷酸的组合物和方法。
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公开(公告)号：WO2020069461A1
公开(公告)日：2020-04-02
申请号：PCT/US2019/053681
申请日：2019-09-27
发明人： PATZKE, Holger , HOU, Jinzhao , WANG, Hongxing , SHU, Yanqun , GOULET, Martin , SAH, Dinah Wen-Yee
IPC分类号： C07K14/47 , A61K31/7088 , A61K48/00
摘要： The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.
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公开(公告)号：WO2019028306A2
公开(公告)日：2019-02-07
申请号：PCT/US2018/045088
申请日：2018-08-03
发明人： SAH, Dinah Wen-Yee , PATZKE, Holger , HOU, Jinzhao , NONNENMACHER, Mathieu E. , GOULET, Martin , CARTER, Todd
摘要： The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.
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