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公开(公告)号：WO2022187473A2
公开(公告)日：2022-09-09
申请号：PCT/US2022/018687
申请日：2022-03-03
IPC分类号： C07K14/005 , C12N15/86 , C12N2710/14044 , C12N2710/14144 , C12N2750/14122 , C12N2750/14143 , C12N2750/14151
摘要： The present disclosure describes methods and systems for use in the production of adeno- associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Spodopterafrugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs).
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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：WO2020150556A1
公开(公告)日：2020-07-23
申请号：PCT/US2020/014000
申请日：2020-01-17
发明人： MARANGA, Luis , MORRISON, Christopher J. , MATHUR, Krishanu , LUTHER, Matthew , HURWIT, Daniel S. , CARDINAL, Jacob J. , KARPES, Lori B. , ANSONDARIA, Aditya , FORSTER, James , DISMUKE, David , STEININGER, Robert
摘要： The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, comprising recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles.
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公开(公告)号：WO2019241486A1
公开(公告)日：2019-12-19
申请号：PCT/US2019/036922
申请日：2019-06-13
发明人： MARANGA, Luis , CECCHINI, Sylvain
IPC分类号： C12N7/00 , C12N15/86 , C07K14/005
摘要： The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system include engineered untranslated regions (UTR) which allow for the controlled expression of AAV capsid proteins, such as VP1, VP2 and VP3. In certain embodiments, the production process and system include engineered untranslated regions (UTR) which allow for the controlled expression of non-structural AAV replication proteins, such as Rep78 and Rep52.
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公开(公告)号：WO2019222444A2
公开(公告)日：2019-11-21
申请号：PCT/US2019/032566
申请日：2019-05-16
发明人： HOU, Jinzhao , WANG, Hongxing , NG, Robert , JOO, Kye Il
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：WO2019018342A1
公开(公告)日：2019-01-24
申请号：PCT/US2018/042391
申请日：2018-07-17
发明人： ROSENBLUTH, Kathryn , KELLS, Adrian Philip , SCHRECK, Stefan , LUTTICH, Edward H. , BLOCH, Kenneth A. , BANKIEWICZ, Krystof
摘要： The present disclosure presents a trajectory array guide system (19) for defining a trajectory to a target location in the brain of a subject and for guiding an elongated tool along the trajectory. The trajectory array guide system (19) can comprise a base (450), an array guide (320), an imaging unit (360), and an elongated handle (440) configured for connection with a stereotaxic navigation system. The present disclosure presents a method of using a trajectory array guide system (19) for defining a trajectory to a target location in the brain of a subject and for guiding an elongated tool along the trajectory.
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公开(公告)号：WO2018204786A1
公开(公告)日：2018-11-08
申请号：PCT/US2018/031089
申请日：2018-05-04
发明人： SAH, Dinah, Wen-Yee , CHEN, Qingmin , HOU, Jinzhao
IPC分类号： A61K31/713 , A61K48/00 , A61P25/28 , C12N15/11 , C12N15/113 , C12N15/86
摘要： The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS). The present invention relates to compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides encoding at least one small interfering RNA (siRNA) molecules which target the superoxide dismutase 1 (SOD1) gene. Methods for using the AAV particles to inhibit the expression of the SOD1 gene in a subject with a neurodegenerative disease (e.g., amyotrophic lateral sclerosis (ALS)) are also disclosed.
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公开(公告)号：WO2017189964A2
公开(公告)日：2017-11-02
申请号：PCT/US2017/030061
申请日：2017-04-28
发明人： PAUL, Steven , WARD, Donna T.
摘要： The invention 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：WO2017075338A3
公开(公告)日：2017-06-01
申请号：PCT/US2016059302
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
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：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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