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公开(公告)号：US20240059742A1
公开(公告)日：2024-02-22
申请号：US18372823
申请日：2023-09-26
CPC分类号： C07K14/015 , C07K14/4707 , C12N7/00 , C12N9/22 , C12N15/11 , C12N15/86 , A61K38/00
摘要： The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the vims vectors and virus capsids of the disclosure to a cell or to a subject in vivo.
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公开(公告)号：US20230405014A1
公开(公告)日：2023-12-21
申请号：US18299640
申请日：2023-04-12
申请人： Genzyme Corporation
IPC分类号： A61K31/4162 , C12N15/86 , C07K14/015 , A61K48/00 , C12N5/0786
CPC分类号： A61K31/4162 , C12N15/86 , C07K14/015 , A61K48/005 , A61K48/0075 , C12N5/0645 , C12N2750/14143 , C12N2502/1121
摘要： Provided herein are methods for enhancing gene therapy in an individual by administering an IRAK degrader with the gene therapy to suppress innate immunity to the gene therapy. In some embodiments, the gene therapy uses an adeno-associated virus (AAV) vector, an adenovirus vector, a lentivirus vector, a Herpes simplex virus (HSV) vector or a lipid nanoparticle. Also provided herein are methods for selecting an individual for treatment with an IRAK degrader in combination with a gene therapy agent.
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公开(公告)号：US20230374540A1
公开(公告)日：2023-11-23
申请号：US18002585
申请日：2021-06-25
申请人： FERRING VENTURES SA
发明人： Lionel GALIBERT , Kari AIRENNE , Amira HYVÖNEN , Reetta ERIKSSON , Hanna Lesch , Justin Darius ALBERS
IPC分类号： C12N15/86 , C07K14/015
CPC分类号： C12N15/86 , C07K14/015 , C12N2750/14122 , C12N2750/14143 , C12N2750/14151
摘要： “MAAP” is a naturally-occurring, newly-discovered about 13 KDa adeno-associated vims protein. It is not homologous to known proteins. When AAV producer cells are cultured for more than 24 hours, we found that inactivating translation of the full-length MAAP improves the productivity of the transfected producer cells. The resulting AAV viruses are also of better quality and more stable. Our findings thus provide a way to improve the industrial manufacture of recombinant adeno-associated virus gene therapy vectors.
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公开(公告)号：US09636370B2
公开(公告)日：2017-05-02
申请号：US14431900
申请日：2013-09-27
发明人： Thomas McCown , Steven Gray
IPC分类号： A61K48/00 , A61K39/12 , A61K39/23 , C07K14/00 , C12N7/00 , C12N15/00 , A61K35/76 , C12N15/86 , C07K14/005 , A61K9/00 , C07K14/015 , C12N15/864
CPC分类号： A61K35/76 , A61K9/0085 , A61K39/23 , C07K14/005 , C07K14/015 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2810/6027
摘要： The invention relates to chimeric AAV capsids targeted to oligodendrocytes, nucleic acids encoding the capsids, virus vectors and particles comprising the same, methods of producing the vectors, and methods of using the vectors to target oligodendrocytes. The invention further relates to methods of treating a disorder associated with oligodendrocyte dysfunction using the vectors.
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公开(公告)号：US12116385B2
公开(公告)日：2024-10-15
申请号：US18323135
申请日：2023-05-24
发明人： Ina Chen , Jeff Gerold , Jerrah Holth , Sylvain Lapan , Kathy S. Lin , Samuel Wolock
IPC分类号： C12N7/00 , A61K48/00 , C07K14/015 , C12N15/86
CPC分类号： C07K14/015 , A61K48/0058 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145
摘要： The disclosure is directed in part to variant capsid polypeptides that can be used to deliver payloads.
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6.发明授权公开(公告)号：US11958884B2
公开(公告)日：2024-04-16
申请号：US17327380
申请日：2021-05-21
IPC分类号： C07K14/015 , C12N15/86
CPC分类号： C07K14/015 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14152
摘要： Methods and compositions are provided that can be used to modify the assembly activating protein (AAP)-dependence of an adeno-associated virus (AAV).
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公开(公告)号：US20240002882A1
公开(公告)日：2024-01-04
申请号：US18313661
申请日：2023-05-08
发明人： Ziying Yan , John F. Engelhardt , Jianming Qiu
IPC分类号： C12N15/86 , C07K14/015
CPC分类号： C12N15/86 , C07K14/015 , C12N2750/14143 , C12N2800/22 , C12N2810/6027
摘要： A method of preparing a chimeric virus comprising bocavirus capsid protein (VP) and a recombinant adeno-associated (AAV) viral genome, and isolated mutant bocavirus genomes, are provided.
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公开(公告)号：US20180236105A1
公开(公告)日：2018-08-23
申请号：US15769931
申请日：2016-10-24
CPC分类号： A61K48/0075 , A61K9/0085 , A61K31/5377 , A61K38/13 , A61K38/4813 , A61K48/005 , A61P25/28 , C07K14/015 , C12N7/00 , C12N9/485 , C12N2750/14143 , C12N2750/14171 , C12Y304/14009 , A61K2300/00
摘要： The present disclosure provides methods of treating a lysosomal storage disorder in a mammal which method comprises administering AAV particles encoding a polypeptide directly to the central nervous system of the mammal in conjunction with administering at least two immunosuppressive agents. AAV particles may be delivered by direct injection into the brain, spinal cord, cerebral spinal fluid or a portion thereof.
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公开(公告)号：US20170274024A1
公开(公告)日：2017-09-28
申请号：US15488966
申请日：2017-04-17
发明人： Thomas McCown , Steven Gray
IPC分类号： A61K35/76 , A61K9/00 , C07K14/005 , C12N15/86 , C12N7/00 , A61K39/23 , C07K14/015 , C12N15/864
CPC分类号： A61K35/76 , A61K9/0085 , A61K39/23 , C07K14/005 , C07K14/015 , C12N7/00 , C12N15/86 , C12N15/8645 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2750/14145 , C12N2810/6027
摘要： The invention relates to chimeric AAV capsids targeted to oligodendrocytes, virus vectors comprising the same, and methods of using the vectors to target oligodendrocytes.
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公开(公告)号：US11702672B2
公开(公告)日：2023-07-18
申请号：US16076219
申请日：2017-02-08
发明人： Ziying Yan , John F. Engelhardt , Jianming Qiu
IPC分类号： C12N15/63 , C12N15/85 , C07H21/04 , C12N15/86 , C07K14/015
CPC分类号： C12N15/86 , C07K14/015 , C12N2750/14143 , C12N2800/22 , C12N2810/6027
摘要： A method of preparing a chimeric virus comprising bocavirus capsid protein (VP) and a recombinant adeno-associated (AAV) viral genome, and isolated mutant bocavirus genomes, are provided.
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