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公开(公告)号：US20240318176A1
公开(公告)日：2024-09-26
申请号：US18577374
申请日：2022-07-08
发明人： Cody A. Desjardins , Kim Tang , James McSwiggen , Romesh R. Subramanian , Timothy Weeden , Mohammed T. Qatanani , Brendan Quinn , John Najim
IPC分类号： C12N15/113 , A61K39/00 , A61K47/68 , C07K16/28
CPC分类号： C12N15/113 , A61K47/6807 , A61K47/6849 , C07K16/2881 , A61K2039/505 , C07K2317/55 , C12N2310/11 , C12N2310/3233 , C12N2310/3513 , C12N2320/32 , C12N2320/33
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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公开(公告)号：US20240197905A1
公开(公告)日：2024-06-20
申请号：US18592695
申请日：2024-03-01
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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公开(公告)号：US20240016950A1
公开(公告)日：2024-01-18
申请号：US18455351
申请日：2023-08-24
发明人： Timothy Weeden , Scott Hilderbrand , Sean Spring , Peiyi Shen , Cody A. Desjardins , Romesh R. Subramanian , Mohammed T. Qatanani , Brendan Quinn , John Najim
CPC分类号： A61K47/6807 , A61K47/6849 , A61K47/545 , A61K47/65 , A61K47/548 , 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 (e.g., wherein each complex is of the exemplary formula shown below) comprising a phosphorodiamidate morpholino oligomer (e.g., useful for targeting DMD) covalently linked to an antibody (e.g., anti-TfR1 antibody). In some embodiments, the complexes are formulated with histidine (e.g., L-histidine) and sucrose at a specified pH (e.g., about 5.0 to 7.0). Also provided are uses of these formulations for treating a subject having a mutated DMD allele associated with Duchenne Muscular Dystrophy.
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公开(公告)号：US11839660B2
公开(公告)日：2023-12-12
申请号：US18181795
申请日：2023-03-10
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 , A61K47/6811 , A61K47/6889 , A61P21/00 , C07K16/2881 , A61K2039/505 , A61K2039/54 , A61K2039/545 , C07K2317/24 , C07K2317/33 , C07K2317/55 , C07K2317/92
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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公开(公告)号：US20230330247A1
公开(公告)日：2023-10-19
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 , A61K47/6849 , A61K47/548 , A61K47/545 , 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：US20230256113A1
公开(公告)日：2023-08-17
申请号：US18181795
申请日：2023-03-10
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 , A61K47/6811 , A61K47/6889 , C07K16/2881 , A61P21/00 , A61K2039/545
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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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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8.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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公开(公告)号：US20240294921A1
公开(公告)日：2024-09-05
申请号：US18572260
申请日：2022-06-17
发明人： Romesh R. Subramanian , Cody A. Desjardins , Oxana Beskrovnaya , Timothy Weeden , Mohammed T. Qatanani , Brendan Quinn , John Najim , Victor Kotelianski , Duncan Brown
IPC分类号： C12N15/113 , A61K47/68
CPC分类号： C12N15/1137 , A61K47/6807 , A61K47/6849 , C12N2310/11 , C12N2310/14 , C12N2310/313 , C12N2310/321 , C12N2310/322 , C12N2310/3513
摘要： Aspects of the disclosure relate to oligonucleotides (e.g., RNAi oligonucleotides such as siRNAs) designed to target GYSI 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., Pompe Disease).
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