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公开(公告)号:US20240052051A1
公开(公告)日:2024-02-15
申请号:US18361367
申请日:2023-07-28
发明人: Nicole Keating , Pascaline Aimé-Wilson , John Dugan , Min Gao , Robert Babb , Maria Praggastis , Katherine Cygnar , Bojie Zhang
CPC分类号: C07K16/2881 , A61P3/00 , C07K2317/565 , C07K2317/55 , C07K2317/622 , C07K2317/92 , C07K2317/14
摘要: Provided, in part, are an anti-human transferrin receptor antigen-binding proteins and fusion proteins comprising an anti-human transferrin receptor antigen-binding proteins (e.g., in scFv, Fab or antibody format) which may be fused to a payload for delivery of the payload to a targeted tissue (e.g., past the blood-brain barrier and to the brain). Payloads include, for example, alpha-glucosidase (GAA) polypeptide. Methods for treating various diseases with such molecules, e.g., glycogen storage diseases, such as Pompe Disease, with the fusions are provided.
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公开(公告)号:US20220195011A1
公开(公告)日:2022-06-23
申请号:US17528828
申请日:2021-11-17
摘要: 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:US11129903B2
公开(公告)日:2021-09-28
申请号:US15202822
申请日:2016-07-06
发明人: Julian Andreev , Nithya Thambi , Frank Delfino , Joel Martin , Gavin Thurston , Katherine Cygnar , Nicholas Papadopoulos
IPC分类号: A61K47/68 , C07K16/22 , C07K16/28 , C07K1/32 , C07K16/12 , C07K16/18 , C07K14/47 , C07K16/30 , A61P35/00 , C07K16/46 , A61K39/00 , C12N9/64 , C07K16/32
摘要: The present invention provides multispecific antigen-binding molecules and uses thereof. The multispecific antigen-binding molecules comprise a first antigen-binding domain that specifically binds a target molecule, and a second antigen-binding domain that specifically binds an internalizing effector protein. The multispecific antigen-binding molecules of the present invention can, in some embodiments, be bispecific antibodies that are capable of binding both a target molecule and an internalizing effector protein. In certain embodiments of the invention, the simultaneous binding of the target molecule and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention results in the attenuation of the activity of the target molecule to a greater extent than the binding of the target molecule alone. In other embodiments of the invention, the target molecule is a tumor associated antigen, and the simultaneous binding of the tumor associated antigen and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention causes or facilitates the targeted killing of tumor cells.
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公开(公告)号:US11696572B2
公开(公告)日:2023-07-11
申请号:US16625168
申请日:2018-06-27
发明人: Alexander O. Mujica , Viktoria Gusarova , Cheng Wang , Christos Kyratsous , Terra Potocky , Katherine Cygnar , Joel H. Martin
IPC分类号: A01K67/027 , A61K49/00 , C07K14/705 , C12N5/071 , C12N5/10
CPC分类号: A01K67/0278 , A61K49/0008 , C07K14/7056 , C12N5/0608 , C12N5/10 , A01K2207/15 , A01K2217/072 , A01K2227/105
摘要: 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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公开(公告)号:US20230149563A1
公开(公告)日:2023-05-18
申请号:US18050161
申请日:2022-10-27
发明人: Leah Sabin , Christos Kyratsous , Evangelos Pefanis , Sven Moller-Tank , Joseph Katakowski , Andrew Baik , Katherine Cygnar , Poulami Samai , Philip Calafati
IPC分类号: A61K48/00 , C12N15/11 , C12N9/22 , C12N15/86 , C12N15/90 , C12N5/071 , A61K38/36 , A61K38/46 , A61P7/04
CPC分类号: A61K48/0016 , C12N15/11 , C12N9/22 , C12N15/86 , C12N15/907 , C12N5/067 , A61K38/36 , A61K38/465 , A61P7/04 , C12N2750/14143 , C12N2310/20 , C12N2800/80
摘要: Nucleic acid constructs and compositions that allow insertion of a FIX coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the FIX coding sequence are provided. The nucleic acid constructs and compositions can be used in methods of introducing a F9 nucleic acid into a cell, methods of integration of a F9 nucleic acid into a target genomic locus, methods of expression of FIX in a cell, and in methods of treating hemophilia B or FIX deficiency in a subject.
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公开(公告)号:US20200095338A1
公开(公告)日:2020-03-26
申请号:US16097108
申请日:2017-04-28
发明人: Katherine Cygnar , Frank Delfino
摘要: The present invention provides multispecific antigen-binding molecules and methods of making or selecting same. The multispecific antigen-binding molecules comprise a first antigen-binding domain that specifically binds a target molecule, and a second antigen-binding domain that specifically binds an internalizing effector protein. The multispecific antigen-binding molecules of the present invention can, in some embodiments, be bispecific antibodies that are capable of binding both a target molecule and an internalizing effector protein. In certain embodiments of the invention, the simultaneous binding of the target molecule and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention results in the attenuation of the activity of the target molecule to a greater extent than the binding of the target molecule alone. In other embodiments of the invention, the target molecule is a tumor associated antigen, and the simultaneous binding of the tumor associated antigen and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention causes or facilitates the targeted killing of tumor cells.
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公开(公告)号:US20180355017A1
公开(公告)日:2018-12-13
申请号:US16001717
申请日:2018-06-06
IPC分类号: C07K14/705 , C12N9/14 , C12N9/24 , C07K16/28
CPC分类号: C07K14/70596 , A61K38/47 , A61K48/00 , A61P43/00 , C07K16/2896 , C07K2317/24 , C07K2317/31 , C07K2317/622 , C07K2317/77 , C07K2317/92 , C07K2319/00 , C07K2319/33 , C12N9/14 , C12N9/24 , C12N9/2402 , C12N9/2428 , C12N2710/10043 , C12N2750/14143
摘要: 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:US20230345919A1
公开(公告)日:2023-11-02
申请号:US18320789
申请日:2023-05-19
发明人: Alexander O. Mujica , Viktoria Gusarova , Cheng Wang , Christos Kyratsous , Terra Potocky , Katherine Cygnar , Joel H. Martin
IPC分类号: A01K67/027 , A61K49/00 , C07K14/705 , C12N5/071 , C12N5/10
CPC分类号: A01K67/0278 , A61K49/0008 , C07K14/7056 , C12N5/0608 , C12N5/10 , A01K2207/15 , A01K2217/072 , A01K2227/105
摘要: 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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公开(公告)号:US11352446B2
公开(公告)日:2022-06-07
申请号:US16097108
申请日:2017-04-28
发明人: Katherine Cygnar , Frank Delfino
摘要: The present invention provides multispecific antigen-binding molecules and methods of making or selecting same. The multispecific antigen-binding molecules comprise a first antigen-binding domain that specifically binds a target molecule, and a second antigen-binding domain that specifically binds an internalizing effector protein. The multispecific antigen-binding molecules of the present invention can, in some embodiments, be bispecific antibodies that are capable of binding both a target molecule and an internalizing effector protein. In certain embodiments of the invention, the simultaneous binding of the target molecule and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention results in the attenuation of the activity of the target molecule to a greater extent than the binding of the target molecule alone. In other embodiments of the invention, the target molecule is a tumor associated antigen, and the simultaneous binding of the tumor associated antigen and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention causes or facilitates the targeted killing of tumor cells.
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公开(公告)号:US20220008548A1
公开(公告)日:2022-01-13
申请号:US17483588
申请日:2021-09-23
发明人: Julian Andreev , Nithya Thambi , Frank Delfino , Joel Martin , Gavin Thurston , Katherine Cygnar , Nicholas Papadopoulos
IPC分类号: A61K47/68 , C07K16/22 , C07K16/28 , C07K16/32 , C12N9/64 , C07K16/12 , C07K16/18 , C07K14/47 , C07K16/30 , A61P35/00 , C07K16/46
摘要: The present invention provides multispecific antigen-binding molecules and uses thereof. The multispecific antigen-binding molecules comprise a first antigen-binding domain that specifically binds a target molecule, and a second antigen-binding domain that specifically binds an internalizing effector protein. The multispecific antigen-binding molecules of the present invention can, in some embodiments, be bispecific antibodies that are capable of binding both a target molecule and an internalizing effector protein. In certain embodiments of the invention, the simultaneous binding of the target molecule and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention results in the attenuation of the activity of the target molecule to a greater extent than the binding of the target molecule alone. In other embodiments of the invention, the target molecule is a tumor associated antigen, and the simultaneous binding of the tumor associated antigen and the internalizing effector protein by the multispecific antigen-binding molecule of the present invention causes or facilitates the targeted killing of tumor cells.
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