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1.发明申请
公开(公告)号：WO2019050948A1
公开(公告)日：2019-03-14
申请号：PCT/US2018/049547
申请日：2018-09-05
Applicant: REGENERON PHARMACEUTICALS, INC.
Inventor： KYRATSOUS, Christos , MURPHY, Andrew, J. , WANG, Cheng
CPC classification number: C12N9/22 , C07K2319/00 , C12N15/86 , C12N2310/20 , C12N2740/13022 , C12N2740/13043
Abstract: The invention provides a recombinant RNA molecule comprising (i) a sequence of a gene-editing molecule mRNA, or a sequence of a functional fragment or derivative thereof, and (ii) at least one sequence of a coding or non-coding enrichment RNA, or a sequence of a functional fragment or derivative thereof, wherein the enrichment RNA, or functional fragment or derivative thereof, is capable of enhancing inclusion of the gene-editing molecule mRNA, or functional fragment or derivative thereof, into a retroviral particle. The invention provides a method of producing the retroviral particles of the invention, the method comprising culturing a packaging cell in conditions sufficient for the production of a plurality of retroviral particles.
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2.发明申请
公开(公告)号：WO2019006043A1
公开(公告)日：2019-01-03
申请号：PCT/US2018/039874
申请日：2018-06-27
Applicant: REGENERON PHARMACEUTICALS, INC.
Inventor： KYRATSOUS, Christos , MURPHY, Andrew J. , WANG, Cheng , SABIN, Leah
IPC: C07K14/015 , C12N15/35 , C12N15/864 , A61K35/76 , A61K48/00 , C12N5/10 , C07K16/18 , C07K16/28
Abstract: Provided herein are compositions and methods for retargeting recombinant viral capsid proteins/capsids/vectors, e.g., in vivo , with a multispecific binding molecule, such as a bispecific antibody, that specifically binds a heterologous epitope displayed by the capsid protein and a protein expressed on the cell of interest for the targeted delivery of a nucleotide of interest.
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公开(公告)号：WO2019006034A1
公开(公告)日：2019-01-03
申请号：PCT/US2018/039864
申请日：2018-06-27
Applicant: REGENERON PHARMACEUTICALS, INC.
Inventor： MUJICA, Alexander O. , GUSAROVA, Viktoria , WANG, Cheng , KYRATSOUS, Christos , POTOCKY, Terra , CYGNAR, Katherine , MARTIN, Joel
IPC: A01K67/027 , C07K14/705 , C12N15/86
Abstract: Non-human animal cells and non-human animals comprising a humanized Asgr1 locus and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized Asgr1 locus express a human ASGR1 protein or an Asgr1 protein, fragments of which are from human ASGR1. Methods are provided for using such non-human animals comprising a humanized Asgr1 locus to assess in vivo efficacy of human-ASGR1-mediated delivery of therapeutic molecules or therapeutic complexes to the liver and to assess the efficacy of therapeutic molecules or therapeutic complexes acting via human-ASGR1-mediated mechanisms.
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4.发明申请
公开(公告)号：WO2020206162A1
公开(公告)日：2020-10-08
申请号：PCT/US2020/026445
申请日：2020-04-02
Applicant: REGENERON PHARMACEUTICALS, INC.
Inventor： WANG, Cheng , HARTFORD, Suzanne , GONG, Guochun , KYRATSOUS, Christos , ZAMBROWICZ, Brian , YANCOPOULOS, George D.
IPC: A01K67/027 , C12N15/90 , C07K16/10
Abstract: Methods and compositions are provided for integrating coding sequences for antigenbinding proteins such as broadly neutralizing antibodies into a safe harbor locus such as an albumin locus in an animal in vivo .
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公开(公告)号：WO2020082042A3
公开(公告)日：2020-04-23
申请号：PCT/US2019/057086
申请日：2019-10-18
Inventor： FINN, John , HUANG, Hon-Ren , ROY, Moitri , LAI, KehDih , SATTLER, Rachel , KYRATSOUS, Christos , WANG, Cheng
IPC: C12N15/113 , A61K48/00 , C12N15/90
Abstract: Methods for editing, e.g ., introducing a heterologous transgene, within the human albumin gene ( e.g ., at intron 1) are provided.
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Patent Agency Ranking
公开(公告)号：WO2020082042A2
公开(公告)日：2020-04-23
申请号：PCT/US2019/057086
申请日：2019-10-18
Inventor： FINN, John , HUANG, Hon-Ren , ROY, Moitri , LAI, KehDih , SATTLER, Rachel , KYRATSOUS, Christos , WANG, Cheng
IPC: C12N15/113 , A61K48/00 , C12N15/90
Abstract: Methods for editing, e.g ., introducing a heterologous transgene, within the human albumin gene ( e.g ., at intron 1) are provided.
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公开(公告)号：WO2020082046A3
公开(公告)日：2020-04-23
申请号：PCT/US2019/057090
申请日：2019-10-18
Inventor： FINN, Jonathan Douglas , HUANG, Hon-Ren , ROY, Moitri , LAI, KehDih , SATTLER, Rachel , KYRATSOUS, Christos , WANG, Cheng
IPC: C12N15/113 , A61K48/00 , C12N15/90
Abstract: Compositions and methods for expressing Factor IX in a host cell or a population of host cells are provided. Also provided are engineered host cells expressing Factor IX.
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公开(公告)号：WO2018226861A4
公开(公告)日：2018-12-13
申请号：PCT/US2018/036306
申请日：2018-06-06
Applicant: REGENERON PHARMACEUTICALS, INC.
Inventor： BAIK, Andrew , CYGNAR, Katherine , SCHOENHERR, Christopher , KYRATSOUS, Christos , WANG, Cheng
Abstract: Compositions and methods for treating enzyme-deficiency diseases are disclosed. Multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.
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公开(公告)号：WO2020082046A2
公开(公告)日：2020-04-23
申请号：PCT/US2019/057090
申请日：2019-10-18
Inventor： FINN, John , HUANG, Hon-Ren , ROY, Moitri , LAI, KehDih , SATTLER, Rachel , KYRATSOUS, Christos , WANG, Cheng
IPC: C12N15/113 , A61K48/00 , C12N15/90
Abstract: Compositions and methods for expressing Factor IX in a host cell or a population of host cells are provided. Also provided are engineered host cells expressing Factor IX.
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公开(公告)号：WO2019006034A9
公开(公告)日：2019-01-03
申请号：PCT/US2018/039864
申请日：2018-06-27
Applicant: REGENERON PHARMACEUTICALS, INC.
Inventor： MUJICA, Alexander O. , GUSAROVA, Viktoria , WANG, Cheng , KYRATSOUS, Christos , POTOCKY, Terra , CYGNAR, Katherine , MARTIN, Joel
IPC: A01K67/027 , C07K14/705 , C12N15/86
Abstract: Non-human animal cells and non-human animals comprising a humanized Asgr1 locus and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized Asgr1 locus express a human ASGR1 protein or an Asgr1 protein, fragments of which are from human ASGR1. Methods are provided for using such non-human animals comprising a humanized Asgr1 locus to assess in vivo efficacy of human-ASGR1-mediated delivery of therapeutic molecules or therapeutic complexes to the liver and to assess the efficacy of therapeutic molecules or therapeutic complexes acting via human-ASGR1-mediated mechanisms.
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