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公开(公告)号：US20160184394A1
公开(公告)日：2016-06-30
申请号：US14938154
申请日：2015-11-11
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C.T. Byrne
IPC分类号： A61K38/17 , C07K14/005 , A61K9/00 , C12N7/00 , A61K48/00
CPC分类号： C12Q1/701 , A61K9/0019 , A61K9/0048 , A61K38/1709 , A61K48/0008 , A61K48/0025 , A61K48/0075 , C07K14/005 , C07K2319/33 , C12N7/00 , C12N15/113 , C12N15/115 , C12N15/86 , C12N2310/14 , C12N2310/16 , C12N2320/32 , C12N2750/14021 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152 , C12N2810/40 , C12Q1/70 , C12Q2600/158 , C12Q2600/16 , G01N33/5008 , G01N33/5058
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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2.发明申请ADENO-ASSOCIATED VIRUS VIRIONS WITH VARIANT CAPSID AND METHODS OF USE THEREOF 有权具有不同CAPSID的ADENO相关病毒血症及其使用方法公开(公告)号：US20150225702A1
公开(公告)日：2015-08-13
申请号：US14606543
申请日：2015-01-27
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C.T. Byrne
CPC分类号： C12Q1/701 , A61K9/0019 , A61K9/0048 , A61K38/1709 , A61K48/0008 , A61K48/0025 , A61K48/0075 , C07K14/005 , C07K2319/33 , C12N7/00 , C12N15/113 , C12N15/115 , C12N15/86 , C12N2310/14 , C12N2310/16 , C12N2320/32 , C12N2750/14021 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152 , C12N2810/40 , C12Q1/70 , C12Q2600/158 , C12Q2600/16 , G01N33/5008 , G01N33/5058
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
摘要翻译： 本公开提供具有改变的衣壳蛋白的腺相关病毒（AAV）病毒颗粒，其中当与野生型AAV相比时，通过玻璃体内注射施用时，AAV病毒体显示较大的视网膜细胞感染性。 本公开进一步提供将基因产物递送至个体中的视网膜细胞的方法以及治疗眼部疾病的方法。
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公开(公告)号：US11236402B2
公开(公告)日：2022-02-01
申请号：US16230080
申请日：2018-12-21
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C. T. Byrne
IPC分类号： C07K14/005 , C12Q1/70 , A61K48/00 , C12N15/86 , C12N7/00 , G01N33/50 , A61K9/00 , A61K38/17 , C12N15/113 , C12N15/115
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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公开(公告)号：US10202657B2
公开(公告)日：2019-02-12
申请号：US15244884
申请日：2016-08-23
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C. T. Byrne
IPC分类号： A61K48/00 , C12Q1/70 , C07K14/005 , C12N15/86 , C12N7/00 , G01N33/50 , A61K9/00 , A61K38/17 , C12N15/113 , C12N15/115
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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公开(公告)号：US20160376323A1
公开(公告)日：2016-12-29
申请号：US15244884
申请日：2016-08-23
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C.T. Byrne
IPC分类号： C07K14/005 , A61K38/17 , A61K9/00 , C12N15/115 , C12N15/113 , C12N7/00 , A61K48/00
CPC分类号： C12Q1/701 , A61K9/0019 , A61K9/0048 , A61K38/1709 , A61K48/0008 , A61K48/0025 , A61K48/0075 , C07K14/005 , C07K2319/33 , C12N7/00 , C12N15/113 , C12N15/115 , C12N15/86 , C12N2310/14 , C12N2310/16 , C12N2320/32 , C12N2750/14021 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152 , C12N2810/40 , C12Q1/70 , C12Q2600/158 , C12Q2600/16 , G01N33/5008 , G01N33/5058
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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公开(公告)号：US20190218627A1
公开(公告)日：2019-07-18
申请号：US16230080
申请日：2018-12-21
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C.T. Byrne
IPC分类号： C12Q1/70 , C12N15/113 , A61K48/00 , C07K14/005 , C12N15/86 , C12N7/00 , G01N33/50 , A61K9/00 , A61K38/17 , C12N15/115
CPC分类号： C12Q1/701 , A61K9/0019 , A61K9/0048 , A61K38/1709 , A61K48/0008 , A61K48/0025 , A61K48/0075 , C07K14/005 , C07K2319/33 , C12N7/00 , C12N15/113 , C12N15/115 , C12N15/86 , C12N2310/14 , C12N2310/16 , C12N2320/32 , C12N2750/14021 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152 , C12N2810/40 , C12Q1/70 , C12Q2600/158 , C12Q2600/16 , G01N33/5008 , G01N33/5058
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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7.发明授权Adeno-associated virus virions with variant capsid and methods of use thereof 有权具有变体衣壳的腺相关病毒颗粒及其使用方法公开(公告)号：US09587282B2
公开(公告)日：2017-03-07
申请号：US14938154
申请日：2015-11-11
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C. T. Byrne
IPC分类号： A61K48/00 , C07H21/04 , C12Q1/70 , C07K14/005 , C12N15/86 , C12N7/00 , G01N33/50 , A61K9/00 , A61K38/17
CPC分类号： C12Q1/701 , A61K9/0019 , A61K9/0048 , A61K38/1709 , A61K48/0008 , A61K48/0025 , A61K48/0075 , C07K14/005 , C07K2319/33 , C12N7/00 , C12N15/113 , C12N15/115 , C12N15/86 , C12N2310/14 , C12N2310/16 , C12N2320/32 , C12N2750/14021 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152 , C12N2810/40 , C12Q1/70 , C12Q2600/158 , C12Q2600/16 , G01N33/5008 , G01N33/5058
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
摘要翻译： 本公开提供具有改变的衣壳蛋白的腺相关病毒（AAV）病毒颗粒，其中当与野生型AAV相比时，通过玻璃体内注射施用时，AAV病毒体显示较大的视网膜细胞感染性。 本公开进一步提供将基因产物递送至个体中的视网膜细胞的方法以及治疗眼部疾病的方法。
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公开(公告)号：US20150232953A1
公开(公告)日：2015-08-20
申请号：US14701063
申请日：2015-04-30
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C.T. Byrne
CPC分类号： C12Q1/701 , A61K9/0019 , A61K9/0048 , A61K38/1709 , A61K48/0008 , A61K48/0025 , A61K48/0075 , C07K14/005 , C07K2319/33 , C12N7/00 , C12N15/113 , C12N15/115 , C12N15/86 , C12N2310/14 , C12N2310/16 , C12N2320/32 , C12N2750/14021 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152 , C12N2810/40 , C12Q1/70 , C12Q2600/158 , C12Q2600/16 , G01N33/5008 , G01N33/5058
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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公开(公告)号：US10214785B2
公开(公告)日：2019-02-26
申请号：US15244892
申请日：2016-08-23
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C. T. Byrne
IPC分类号： C12Q1/70 , A61K48/00 , C07K14/005 , C12N15/86 , C12N7/00 , G01N33/50 , A61K9/00 , A61K38/17 , C12N15/113 , C12N15/115
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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公开(公告)号：US09856539B2
公开(公告)日：2018-01-02
申请号：US14606543
申请日：2015-01-27
发明人： David V. Schaffer , Ryan R. Klimczak , James T. Koerber , John G. Flannery , Deniz Dalkara Mourot , Meike Visel , Leah C. T. Byrne
IPC分类号： A01N63/00 , C12Q1/70 , A61K48/00 , C07K14/005 , C12N15/86 , C12N7/00 , G01N33/50 , A61K9/00 , A61K38/17 , C12N15/113 , C12N15/115
CPC分类号： C12Q1/701 , A61K9/0019 , A61K9/0048 , A61K38/1709 , A61K48/0008 , A61K48/0025 , A61K48/0075 , C07K14/005 , C07K2319/33 , C12N7/00 , C12N15/113 , C12N15/115 , C12N15/86 , C12N2310/14 , C12N2310/16 , C12N2320/32 , C12N2750/14021 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152 , C12N2810/40 , C12Q1/70 , C12Q2600/158 , C12Q2600/16 , G01N33/5008 , G01N33/5058
摘要： The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
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