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公开(公告)号：US12102687B2
公开(公告)日：2024-10-01
申请号：US18609032
申请日：2024-03-19
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
IPC分类号： A61K39/395 , A61K47/68 , C07K14/47 , C07K16/28 , C12N15/113 , A61K38/00 , A61K39/00
CPC分类号： A61K47/6807 , A61K47/6849 , C07K14/4707 , C07K16/2881 , C12N15/113 , A61K38/00 , A61K2039/505 , C07K2317/24 , C07K2317/33 , C07K2317/55 , C07K2317/77 , C07K2317/92 , C07K2317/94 , C12N2310/11 , C12N2310/14 , C12N2310/3513
摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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公开(公告)号：US12097263B2
公开(公告)日：2024-09-24
申请号：US18416981
申请日：2024-01-19
IPC分类号： C07K16/28 , A61K47/60 , A61K47/68 , A61P21/00 , C12N15/113
CPC分类号： A61K47/6807 , A61K47/60 , A61K47/6849 , A61P21/00 , C07K16/2881 , C12N15/1137 , C07K2317/55 , C07K2317/92 , C12N2310/11 , C12N2310/315 , C12N2310/322 , C12N2310/3513
摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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23.发明公开公开(公告)号：US20240110184A1
公开(公告)日：2024-04-04
申请号：US18270284
申请日：2021-12-30
发明人： Duncan Brown , Nelson Hsia , Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
IPC分类号： C12N15/113 , A61K31/712 , A61K31/7125 , A61K47/68 , A61P21/00 , C07K16/28
CPC分类号： C12N15/113 , A61K31/712 , A61K31/7125 , A61K47/6807 , A61K47/6849 , A61P21/00 , C07K16/2881
摘要： Aspects of the disclosure relate to oligonucleotides (e.g., RNAi oligonucleotides such as siRNAs) designed to target DUX4 RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of disease (e.g., FSHD).
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公开(公告)号：US20240100177A1
公开(公告)日：2024-03-28
申请号：US18265065
申请日：2021-12-03
发明人： Scott Hilderbrand , John Najim , Qifeng Qiu , Benjamin Vieira , Timothy Weeden , Sean Spring
IPC分类号： A61K47/68
CPC分类号： A61K47/6807 , A61K47/6889 , A61K47/6849
摘要： Provided herein are oligonucleotide-antibody complexes, methods of preparing the complexes, and methods of using the complexes (e.g., treating muscle diseases). In particular, provided are compounds of Formula (I) and various methods for the preparation of the compounds. Also provided are pharmaceutical compositions comprising compounds of Formula (I) and methods of treating muscle disease in a subject by administering a compound or composition described herein.
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公开(公告)号：US11931421B2
公开(公告)日：2024-03-19
申请号：US18303506
申请日：2023-04-19
发明人： Scott Hilderbrand , Timothy Weeden , John Najim , Stefano Zanotti , Romesh R. Subramanian , Mohammed T. Qatanani , Cody A. Desjardins , Kim Tang , Brendan Quinn
CPC分类号： A61K47/6807 , A61K47/545 , A61K47/548 , A61K47/6849 , A61P21/00
摘要： Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes comprising an oligonucleotide (e.g., useful for targeting DMPK) covalently linked to an antibody (e.g., anti-TfR1 antibody).
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公开(公告)号：US11911484B2
公开(公告)日：2024-02-27
申请号：US17205102
申请日：2021-03-18
IPC分类号： A61K47/68 , C07K16/28 , C12N15/113 , A61K47/60 , A61P21/00
CPC分类号： A61K47/6807 , A61K47/60 , A61K47/6849 , A61P21/00 , C07K16/2881 , C12N15/1137 , C07K2317/55 , C07K2317/92 , C12N2310/11 , C12N2310/315 , C12N2310/322 , C12N2310/3513
摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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公开(公告)号：US20240016952A1
公开(公告)日：2024-01-18
申请号：US18017170
申请日：2021-07-09
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
CPC分类号： A61K47/6849 , A61P21/00
摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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公开(公告)号：US20230346966A1
公开(公告)日：2023-11-02
申请号：US18017182
申请日：2021-07-09
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
CPC分类号： A61K47/6849 , A61K47/6807 , A61K47/6889 , A61P21/00 , A61K2039/505
摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits activity of a pro-atrophy gene. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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29.发明公开公开(公告)号：US20230321264A1
公开(公告)日：2023-10-12
申请号：US18329781
申请日：2023-06-06
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
IPC分类号： A61K47/68 , C07K14/47 , C07K16/28 , C12N15/113
CPC分类号： A61K47/6807 , C07K14/4707 , C07K16/2881 , C12N15/113 , A61K47/6849 , C12N2310/3513 , A61K2039/505
摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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公开(公告)号：US11771776B2
公开(公告)日：2023-10-03
申请号：US17811370
申请日：2022-07-08
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
CPC分类号： A61K47/6849 , A61K47/6807 , A61K47/6889 , A61P21/00
摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.
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