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公开(公告)号：WO2022098699A1
公开(公告)日：2022-05-12
申请号：PCT/US2021/057823
申请日：2021-11-03
发明人： CARTER, Todd , LIU, Wencheng , HOLTH, Jerrah , HOU, Jinzhao , SHU, Yanqun , MURLIDHARAN, Giridhar , REN, Xiao-Qin
IPC分类号： C07K16/18 , C12N15/861 , A61K39/00
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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公开(公告)号：WO2022132923A1
公开(公告)日：2022-06-23
申请号：PCT/US2021/063551
申请日：2021-12-15
发明人： KURELLA, Vinodhbabu , LIU, Li , KAVANAGH, Dillon , CAPILI, Allan, D. , HOU, Jinzhao , CARTER, Todd , LIU, Wencheng
摘要： The present disclosure provides anti-tau antibodies. Also provided are methods of using anti-tau antibodies for treatment and diagnosis of neurological indications.
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公开(公告)号：WO2020077165A1
公开(公告)日：2020-04-16
申请号：PCT/US2019/055756
申请日：2019-10-11
发明人： MURLIDHARAN, Giridhar , SAH, Dinah Wen-Yee , PATZKE, Holger , CARTER, Todd , HOU, Jinzhao , ADACHI, Kei , REN, Amy
IPC分类号： C07K14/005 , C12N15/86
摘要： The disclosure 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：WO2021016505A1
公开(公告)日：2021-01-28
申请号：PCT/US2020/043366
申请日：2020-07-24
发明人： SAH, Dinah Wen-Yee , ZHOU, Pengcheng , THOMPSON, Jeffrey S. , GAMBA-VITALO, Christina , CARROLL SOPER, Jenna , HERSCH, Steven M. , CARTER, Todd , CARDINAL, Jacob J. , KARPES, Lori B. , LIU, Bin
摘要： The present disclosure relates to pharmaceutical compositions of 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：WO2020023612A1
公开(公告)日：2020-01-30
申请号：PCT/US2019/043196
申请日：2019-07-24
发明人： CARDINAL, Jacob J. , STEININGER, Robert , KARPES, Lori B. , MORRISON, Christopher J. , HURWIT, Daniel S. , LUTHER, Matthew , WOOD, Andrew M. , SAH, Dinah Wen-Yee , ZHOU, Pengcheng , THOMPSON, Jeffrey S. , GAMBA-VITALO, Christina , CARROLL, Jenna , HERSCH, Steven M. , CARTER, Todd
摘要： The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles and AAV formulations, including recombinant adeno-associated virus (rAAV) particles and formulations. In certain embodiments, the present disclosure presents methods and systems for clarifying, purifying, formulating, filtering and processing AAV particles and AAV formulations. The present disclosure also describes compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides encoding small interfering RNA (siRNA) molecules which target the Huntingtin (HTT) gene (e.g., the wild-type or the mutated CAG-expanded HTT gene). Methods for using formulated AAV particles comprising modulatory polynucleotides to inhibit the HTT gene expression in a subject with a eurodegenerative disease (e.g., Huntington's Disease (HD)) are also disclosed.
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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：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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公开(公告)号：WO2021211753A1
公开(公告)日：2021-10-21
申请号：PCT/US2021/027346
申请日：2021-04-14
发明人： CARTER, Todd , HOU, Jinzhao , KURELLA, Vinodhbabu , CAPILI, Allan, D. , LIU, Wencheng , CHUNG, Hiu Yan , HOLTH, Jerrah , KAVANAGH, Dillon
摘要： The present disclosure provides anti-tau antibodies and vectorization thereof (e.g., into AAV particles). Also provided are methods of using anti-tau antibodies and/or AAV particles for prevention, treatment, and/or diagnosis of neurological indications.
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公开(公告)号：WO2019222329A1
公开(公告)日：2019-11-21
申请号：PCT/US2019/032387
申请日：2019-05-15
摘要： The disclosure 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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