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公开(公告)号：US20230226212A1
公开(公告)日：2023-07-20
申请号：US17791667
申请日：2021-01-08
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , Jason P. Rhodes
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 inhibits activity of a disease allele associated with muscle disease. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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12.发明申请公开(公告)号：US20230111147A1
公开(公告)日：2023-04-13
申请号：US17791670
申请日：2021-01-08
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Cody A. Desjardins , Duncan Brown , Victor Kotelianski , Timothy Weeden , Brendan Quinn
IPC分类号： C07K16/28
摘要： Aspects of the disclosure relate to molecular payloads that modulate the expression or activity of genes involved in muscle growth and maintenance (e.g., MSTN, INHBA, and/or ACVR1B), and complexes comprising a muscle-targeting agent covalently linked to such molecular payloads. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on a muscle cell (e.g., a cardiac muscle cell). 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：US20230044278A1
公开(公告)日：2023-02-09
申请号：US17811396
申请日：2022-07-08
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
摘要： Aspects of the disclosure relate to antibodies that bind to transferrin receptor (e.g., transferrin receptor 1) and complexes comprising the antibody covalently linked to a molecular payload. Methods of using the antibodies are also provided.
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14.发明申请公开(公告)号：US20220324992A1
公开(公告)日：2022-10-13
申请号：US17846738
申请日：2022-06-22
IPC分类号： C07K16/28 , C12N15/113
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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15.发明申请公开(公告)号：US20220169743A1
公开(公告)日：2022-06-02
申请号：US17671707
申请日：2022-02-15
IPC分类号： C07K16/28 , C12N15/113
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：US20220143206A1
公开(公告)日：2022-05-12
申请号：US17580844
申请日：2022-01-21
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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17.发明申请公开(公告)号：US20210380709A1
公开(公告)日：2021-12-09
申请号：US17400295
申请日：2021-08-12
IPC分类号： C07K16/28 , C12N15/113
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：US20210322562A1
公开(公告)日：2021-10-21
申请号：US17264966
申请日：2019-08-02
IPC分类号： A61K47/68 , C07K16/28 , C12N15/113 , C12N9/10 , C12N9/14
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 DNM2. 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：US20210317226A1
公开(公告)日：2021-10-14
申请号：US17265016
申请日：2019-08-02
IPC分类号： C07K16/28 , C12N15/113 , C12N9/24
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 reduces glycogen levels in a cell.
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公开(公告)号：US20210308272A1
公开(公告)日：2021-10-07
申请号：US17264905
申请日：2019-08-02
IPC分类号： A61K47/68 , C07K16/28 , C12N15/113
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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