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公开(公告)号：US20190127424A1
公开(公告)日：2019-05-02
申请号：US16179818
申请日：2018-11-02
发明人： Arun Srivastava , George Vladimirovich Aslanidi , Sergei Zolotukhin , Mavis Agbandje-McKenna , Kim M. Van Vliet , Li Zhong , Lakshmanan Govindasamy
IPC分类号： C07K14/005 , C07K14/805 , A61K38/42 , C12N15/86
摘要： Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.
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公开(公告)号：US10011640B2
公开(公告)日：2018-07-03
申请号：US14214011
申请日：2014-03-14
发明人： Arun Srivastava , George Vladimirovich Aslanidi , Sergei Zolotukhin , Mavis Agbandje-McKenna , Kim M. Van Vliet , Li Zhong , Lakshmanan Govindasamy
IPC分类号： C12N15/86 , C07K14/005 , A61K38/42 , C07K14/805
CPC分类号： C07K14/005 , A61K38/42 , C07K14/805 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2810/6027
摘要： Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.
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公开(公告)号：US20230340526A1
公开(公告)日：2023-10-26
申请号：US17779510
申请日：2020-11-24
CPC分类号： C12N15/86 , C12N15/1058 , C12N2750/14143
摘要： Disclosed herein are recombinant AAV variant (e.g., variant serotype 3B (AAV3B)) capsid proteins and variant capsid protein-containing viral particles with enhanced ability to transduce hepatic cells. Viral particles containing these capsid variants are capable of evading neutralization by the host humoral immune response. The recombinant AAV3B variant proteins and viral particles disclosed herein were identified from a variant AAV3B capsid library that was engineered by making substitutions in only the variable regions of the capsid. Some embodiments of the AAV3B capsid variants disclosed herein comprise the AAV3B-DE5 variant, which contains 24 mutations relative to wild-type. Compositions of these variant AAV particles are provided that are useful for transducing and delivering therapeutic transgenes to cells, such as liver cells, and thus treat diseases and disorders pertaining to these cells.
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公开(公告)号：US20230049066A1
公开(公告)日：2023-02-16
申请号：US17779505
申请日：2020-11-24
申请人： University of Florida Research Foundation, Incorporated , University of Massachusetts One Beacon Street
摘要： Disclosed herein are recombinant AAV variant (e.g., variant serotype 3B (AAV3B)) capsid proteins and variant capsid protein-containing viral particles with enhanced ability to transduce hepatic cells. Viral particles containing these capsid variants are capable of evading neutralization by the host humoral immune response. The recombinant AAV3B variant proteins and viral particles disclosed herein were identified from a variant AAV3B capsid library that was engineered by making substitutions in only the variable regions of the capsid. Some embodiments of the AAV3B capsid variants disclosed herein comprise the AAV3B-G3 variant and the AAV3B-E12 variant. Compositions of these variant AAV particles are provided that are useful for transducing and delivering therapeutic transgenes to cells, such as liver cells, and thus treat diseases and disorders pertaining to these cells.
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15.发明授权公开(公告)号：US11384364B2
公开(公告)日：2022-07-12
申请号：US16093586
申请日：2017-04-16
IPC分类号： C12N15/86
摘要： Provided herein are methods and compositions useful in the production of recombinant AAV (rAAV) in insect cells. In some embodiments, methods and compositions include the use of modified Kozak sequences to express AAV VP1 proteins in amounts that are useful for producing infective rAAV particles.
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公开(公告)号：US11091777B2
公开(公告)日：2021-08-17
申请号：US16208127
申请日：2018-12-03
IPC分类号： C12N15/86 , C07K14/005 , C12N7/00 , A61K48/00
摘要： Disclosed are compositions and methods for producing modified adeno-associated virus (AAV) cap genes and combinatorial libraries of chimeric AAV vectors and virions; selecting for virions displaying cell-specific tropisms; and, in certain embodiments, producing helper vectors containing one or more modified AAV cap genes. The synthetic combinatorial AAV capsid libraries of the invention are useful in introducing into selected target host cells one or more nucleic acid molecules. The viral vectors and genetic constructs disclosed herein are also useful in a variety of diagnostic and/or therapeutic gene-therapy regimens.
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公开(公告)号：US20210061863A1
公开(公告)日：2021-03-04
申请号：US17009536
申请日：2020-09-01
发明人： Sergei Zolotukhin , Sanford L. Boye , Shannon E. Boye , Damien Marsic , Nicholas Muzyczka , Hector Ruben Mendez-Gomez , Paul E. Gamlin
IPC分类号： C07K14/005 , C12N15/10 , C12N15/86
摘要： Adeno associated viral (AAV) particles are emerging as a useful vehicle for gene delivery to various organs and tissues, one of them being the retina. Provided here are variant AAV (e.g., variant serotype 2 (AAV2)) capsid proteins and variant capsid protein containing particles with enhanced ability to transduce retinal cells.
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公开(公告)号：US20210040156A1
公开(公告)日：2021-02-11
申请号：US16933780
申请日：2020-07-20
发明人： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC分类号： C07K14/005 , A61K48/00 , A61K39/00 , C12N15/86 , C12N15/864 , A61K35/76 , C12N7/00
摘要： Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
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公开(公告)号：US10815279B2
公开(公告)日：2020-10-27
申请号：US15987993
申请日：2018-05-24
发明人： Arun Srivastava , George Vladimirovich Aslanidi , Sergei Zolotukhin , Mavis Agbandje-McKenna , Kim M. Van Vliet , Li Zhong , Lakshmanan Govindasamy
IPC分类号： C07K14/005 , C12N15/86 , A61K38/42 , C07K14/805
摘要： Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.
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公开(公告)号：US20180036428A1
公开(公告)日：2018-02-08
申请号：US15680668
申请日：2017-08-18
发明人： Li Zhong , Sergei Zolotukhin , Lakshmanan Govindasamy , Mavis Agbandje-McKenna , Arun Srivastava
IPC分类号： A61K48/00 , C12N15/86 , A61K35/76 , C12N15/864 , C12N7/00
摘要： Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.
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