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公开(公告)号：US20210189430A1
公开(公告)日：2021-06-24
申请号：US17194009
申请日：2021-03-05
申请人： Genzyme Corporation
IPC分类号： C12N15/86 , A61K48/00 , C07K14/71 , C07K14/015 , C12N7/00 , C12N15/113
摘要： Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and/or treating a CNS disorder.
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公开(公告)号：US20220288232A1
公开(公告)日：2022-09-15
申请号：US17681468
申请日：2022-02-25
申请人： Genzyme Corporation
发明人： Abraham SCARIA
摘要： Compositions and methods for treating macular degeneration are disclosed. The methods utilize gene delivery to human eyes of soluble Flt-1 receptors, as well fusion proteins including a soluble Flt-1 receptor.
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3.发明申请FUSION PROTEINS COMPRISING PDGF AND VEGF BINDING PORTIONS AND METHODS OF USING THEREOF 有权包含PDGF和VEGF结合部分的融合蛋白及其使用方法公开(公告)号：US20140315804A1
公开(公告)日：2014-10-23
申请号：US14210354
申请日：2014-03-13
申请人： GENZYME CORPORATION
IPC分类号： C07K14/71
CPC分类号： C07K14/71 , A61K38/00 , C07K2319/30 , C07K2319/70 , C12N2510/02 , C12N2750/14143
摘要： The present provides fusion proteins comprising PDGF and VEGF binding portions, and recombinant viral particles encoding the fusion proteins. Compositions comprising the fusion proteins and viral particles as well as methods of using the same are also provided.
摘要翻译： 本发明提供了包含PDGF和VEGF结合部分的融合蛋白，以及编码融合蛋白的重组病毒颗粒。 还提供了包含融合蛋白和病毒颗粒的组合物以及其使用方法。
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公开(公告)号：US20220340883A1
公开(公告)日：2022-10-27
申请号：US17686245
申请日：2022-03-03
申请人： Genzyme Corporation
IPC分类号： C12N7/00
摘要： The use of iNOS inhibitors, including aurintricarboxylic acid, dexamethasone and valproic acid, to increase the yield of a variety of viruses in culture, including recombinant herpesviruses is described.
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公开(公告)号：US20220064262A1
公开(公告)日：2022-03-03
申请号：US17367027
申请日：2021-07-02
申请人： Genzyme Corporation
摘要： The present provides fusion proteins comprising PDGF and VEGF binding portions, and recombinant viral particles encoding the fusion proteins. Compositions comprising the fusion proteins and viral particles as well as methods of using the same are also provided.
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公开(公告)号：US20240197837A1
公开(公告)日：2024-06-20
申请号：US18399364
申请日：2023-12-28
申请人： Genzyme Corporation
发明人： Guoxiang RUAN , Abraham SCARIA
IPC分类号： A61K38/46 , A61K31/7088 , A61K31/7105 , A61P27/02 , C12N9/22 , C12N15/11 , C12N15/90
CPC分类号： A61K38/465 , A61K31/7088 , A61K31/7105 , A61P27/02 , C12N9/22 , C12N15/11 , C12N15/907 , C12N2310/20 , C12N2750/14143 , C12N2800/80
摘要： Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)—CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.
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公开(公告)号：US20190022192A1
公开(公告)日：2019-01-24
申请号：US15574420
申请日：2016-04-15
申请人： Genzyme Corporation
发明人： Guoxiang RUAN , Abraham SCARIA
IPC分类号： A61K38/46 , C12N9/22 , C12N15/11 , C12N15/90 , A61K31/7105 , A61K35/761
摘要： Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.
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8.发明申请公开(公告)号：US20160068844A1
公开(公告)日：2016-03-10
申请号：US14785159
申请日：2014-04-17
申请人： GENZYME CORPORATION , THE UNITED STATES OF AMERICA,AS REPRESENTED BY THE SECRETARY,DEPARTMENT OF HEALTH AND HUMAN SERVICE
发明人： Samuel WADSWORTH , Abraham SCARIA , Chi-Chao CHAN
IPC分类号： C12N15/113
CPC分类号： C12N15/1136 , A61K38/1793 , C07K14/7155 , C07K2319/30 , C12N2750/14143
摘要： Compositions and methods for treating macular degeneration are disclosed. The methods utilize IL17 inhibitors, such as IL17 receptors, as well as fusion proteins including an IL17 receptor fused with a multimerization domain, and recombinant viral vectors encoding such fusions.
摘要翻译： 公开了治疗黄斑变性的组合物和方法。 所述方法利用IL17抑制剂，例如IL17受体，以及融合蛋白，包括与多聚化结构域融合的IL17受体，以及编码这种融合物的重组病毒载体。
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公开(公告)号：US20210040484A1
公开(公告)日：2021-02-11
申请号：US16835913
申请日：2020-03-31
申请人： Genzyme Corporation , The United States of America, as Represented by the Secretary, Department of Health and Human Servic
发明人： Samuel WADSWORTH , Abraham SCARIA , Chi-Chao CHAN
IPC分类号： C12N15/113 , A61K38/17 , C07K14/715
摘要： Compositions and methods for treating macular degeneration are disclosed. The methods utilize IL17 inhibitors, such as IL17 receptors, as well as fusion proteins including an IL17 receptor fused with a multimerization domain, and recombinant viral vectors encoding such fusions.
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10.发明申请公开(公告)号：US20170342127A1
公开(公告)日：2017-11-30
申请号：US15471725
申请日：2017-03-28
申请人： Genzyme Corporation
CPC分类号： C07K14/71 , A61K38/00 , C07K2319/30 , C07K2319/70 , C12N2510/02 , C12N2750/14143
摘要： The present provides fusion proteins comprising PDGF and VEGF binding portions, and recombinant viral particles encoding the fusion proteins. Compositions comprising the fusion proteins and viral particles as well as methods of using the same are also provided.
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