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1.发明公开ADENO-ASSOCIATED VIRUS VIRIONS WITH VARIANT CAPSID AND METHODS OF USE THEREOF 有权具有变体荚膜的腺伴随病毒病毒及其使用方法
公开(公告)号：EP2699270A2
公开(公告)日：2014-02-26
申请号：EP12774323.5
申请日：2012-04-20
发明人： SCHAFFER, David V. , KLIMCZAK, Ryan R. , KOERBER, James T. , FLANNERY, John G. , DALKARA MOUROT, Deniz , VISEL, Meike , BYRNE, Leah C.T.
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： 本公开内容提供了具有改变的衣壳蛋白的腺伴随病毒（AAV）病毒体，其中与野生型AAV相比，当通过玻璃体内注射施用时，AAV毒粒表现出更大的视网膜细胞感染性。 本公开还提供了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：EP4005603A1
公开(公告)日：2022-06-01
申请号：EP21201945.9
申请日：2012-04-20
发明人： SCHAFFER, David V. , KLIMCZAK, Ryan R. , KOERBER, James, T. , FLANNERY, John G. , DALKARA MOUROT, Deniz , VISEL, Meike , BYRNE, Leah C.T.
IPC分类号： A61K48/00 , C12N15/63 , A61P9/00 , A61P3/10 , C07K14/005 , A61P27/00 , A61P43/00 , A61P27/02 , A61P27/06 , A61P9/10 , C12N15/86 , A61K38/17 , A61K9/00 , C12N7/00 , C12Q1/70 , C12N15/113 , C12N15/115 , G01N33/50
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：EP3254703B1
公开(公告)日：2020-02-19
申请号：EP17168534.0
申请日：2012-04-20
发明人： SCHAFFER, David V. , KLIMCZAK, Ryan R. , KOERBER, James T. , FLANNERY, John G. , DALKARA MOUROT, Deniz , VISEL, Meike , BYRNE, Leah C.T.
IPC分类号： A61K48/00 , C12N15/63 , C07K14/005 , C12N15/86 , A61K38/17 , A61K9/00 , C12N7/00 , C12Q1/70 , C12N15/113 , C12N15/115 , G01N33/50
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公开(公告)号：EP3693025A1
公开(公告)日：2020-08-12
申请号：EP20151479.1
申请日：2012-04-20
发明人： SCHAFFER, David V. , KLIMCZAK, Ryan R. , KOERBER, James, T. , FLANNERY, John G. , DALKARA MOUROT, Deniz , VISEL, Meike , BYRNE, Leah C.T.
IPC分类号： A61K48/00 , C12N15/63 , C07K14/005 , C12N15/86 , A61P3/10 , A61P9/00 , A61P9/10 , A61P27/00 , A61P27/02 , A61P27/06 , A61P43/00
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：EP3254703A1
公开(公告)日：2017-12-13
申请号：EP17168534.0
申请日：2012-04-20
发明人： SCHAFFER, David V. , KLIMCZAK, Ryan R. , KOERBER, James T. , FLANNERY, John G. , DALKARA MOUROT, Deniz , VISEL, Meike , BYRNE, Leah C.T.
IPC分类号： A61K48/00 , C12N15/63 , C07K14/005 , C12N15/86 , A61K38/17 , A61K9/00 , C12N7/00 , C12Q1/70 , C12N15/113 , C12N15/115 , G01N33/50
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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