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公开(公告)号：US20230044444A1
公开(公告)日：2023-02-09
申请号：US17931725
申请日：2022-09-13
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： Dinah Wen-Yee Sah , Martin Goulet , Holger Patzke , Yanqun Shu , Jinzhao Hou , Hélène Puccio
摘要： Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.
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2.发明申请公开(公告)号：US20200263199A1
公开(公告)日：2020-08-20
申请号：US16651617
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： Dinah Wen-Yee Sah , Martin Goulet , Holger Patzke , Yanqun Shu , Jinzhao Hou , Hélène Puccio
摘要： Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.
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公开(公告)号：US20240285667A1
公开(公告)日：2024-08-29
申请号：US18517820
申请日：2023-11-22
发明人： Gregory Robert Stewart , Dinah Wen-Yee Sah , Jinzhao Hou , Adrian Philip Kells , Martin Goulet , Kartik Ramamoorthi , Pengcheng Zhou , Yanqun Shu
IPC分类号： A61K31/7088 , A61K48/00 , C07K14/47
CPC分类号： A61K31/7088 , A61K48/005 , A61K48/0075 , A61K48/0083 , C07K14/47 , C12N2750/14143
摘要： The present disclosure relates to compositions, methods and processes for the formulation and for the administration of a gene therapy agent using parvovirus e.g., adeno-associated virus (AAV) to the CNS, CNS tissues, CNS structures or CNS cells.
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公开(公告)号：US20240131093A1
公开(公告)日：2024-04-25
申请号：US18351930
申请日：2023-07-12
发明人： Dinah Wen-Yee Sah , Fen Chen , Pengcheng Zhou , Xin Wang , Yanqun Shu , Jinzhao Hou , Jochen Deckert , Markus Hossbach
IPC分类号： A61K35/76 , A61P25/14 , C12N7/00 , C12N15/113 , C12N15/86
摘要： The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
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公开(公告)号：US20180021364A1
公开(公告)日：2018-01-25
申请号：US15543773
申请日：2016-01-15
发明人： Gregory Robert Stewart , Dinah Wen-Yee Sah , Jinzhao Hou , Adrian Philip Kells , Martin Goulet , Kartik Ramamoorthi , Pengcheng Zhou , Yanqun Shu
IPC分类号： A61K31/7088 , C07K14/47 , A61K48/00
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.
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公开(公告)号：US20240226203A9
公开(公告)日：2024-07-11
申请号：US18351930
申请日：2023-07-13
发明人： Dinah Wen-Yee Sah , Fen Chen , Pengcheng Zhou , Xin Wang , Yanqun Shu , Jinzhao Hou , Jochen Deckert , Markus Hossbach
IPC分类号： A61K35/76 , A61P25/14 , C12N7/00 , C12N15/113 , C12N15/86
摘要： The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
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公开(公告)号：US20230285596A1
公开(公告)日：2023-09-14
申请号：US18018089
申请日：2021-07-26
发明人： Heather Yonutas , Jeffrey Brown , Jinzhao Hou , Yanqun Shu , Elisabeth Knoll , Priyantha Herath , Brett Hoffman
IPC分类号： A61K48/00 , C07K14/705 , A61K45/06 , A61P3/06 , C12N15/86
CPC分类号： A61K48/0058 , C07K14/705 , A61K48/0041 , A61K45/06 , A61P3/06 , C12N15/86 , C12N2750/14143
摘要： The disclosure provides compositions and methods for altering, e.g., enhancing, the expression of NPC1 and/or NPC2 proteins, whether in vitro and/or in vivo. Such compositions include delivery 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 NPC1 disease or related condition resulting from a deficiency in the quantity and/or function of NPC1 and/or NPC2 proteins or associated with decreased expression or protein levels of NPC1 and/or NPC2 proteins.
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公开(公告)号：US11752181B2
公开(公告)日：2023-09-12
申请号：US16611041
申请日：2018-05-04
发明人： Dinah Wen-Yee Sah , Fen Chen , Pengcheng Zhou , Xin Wang , Yanqun Shu , Jinzhao Hou , Jochen Deckert , Markus Hossbach
CPC分类号： A61K35/76 , A61P25/14 , C12N7/00 , C12N15/113 , C12N15/86 , A61K48/00 , C12N2310/141 , C12N2320/32 , C12N2750/14121 , C12N2750/14123 , C12N2750/14132 , C12N2750/14143
摘要： The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
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公开(公告)号：US20230075314A1
公开(公告)日：2023-03-09
申请号：US17607213
申请日：2020-04-29
发明人： Jinzhao Hou , Yanqun Shu , Todd Carter , Dinah Wen-Yee Sah , Po-Jen Yen , Donna T. Ward , Johanna L. Crimins
IPC分类号： C12N15/86 , C12N15/113 , C07K16/00
摘要： The present disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
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公开(公告)号：US20220339270A1
公开(公告)日：2022-10-27
申请号：US17607141
申请日：2020-04-29
发明人： Wencheng Liu , Todd Carter , Jinzhao Hou , Yanqun Shu , Giridhar Murlidharan , Martin Goulet , Dinah Wen-Yee Sah , Steven Paul , Xiao-Qin Ren , Xin Wang , Hiu Yan Chung
摘要： The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
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