-
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.
-
公开(公告)号：US20240309107A1
公开(公告)日：2024-09-19
申请号：US18656654
申请日：2024-05-07
IPC分类号： C07K16/28 , A61K47/68 , A61P21/00 , C12N15/113
CPC分类号： C07K16/2881 , A61K47/6807 , A61K47/6849 , A61K47/6889 , A61P21/00 , C12N15/113 , C07K2317/55 , C07K2317/92
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.
-
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.
-
公开(公告)号：US20240197901A1
公开(公告)日：2024-06-20
申请号：US18416981
申请日：2024-01-19
IPC分类号： A61K47/68 , A61K47/60 , A61P21/00 , C07K16/28 , 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.
-
公开(公告)号：US20240148891A1
公开(公告)日：2024-05-09
申请号：US17769467
申请日：2020-10-16
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.
-
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.
-
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.
-
公开(公告)号：US11795233B2
公开(公告)日：2023-10-24
申请号：US18184741
申请日：2023-03-16
IPC分类号： C07K16/28 , C12N15/113 , A61K47/68 , A61P21/00
CPC分类号： C07K16/2881 , A61K47/6807 , A61K47/6849 , A61K47/6889 , A61P21/00 , C12N15/113 , C07K2317/55 , C07K2317/92
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.
-
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).
-
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.
-
-
-
-
-
-
-
-
-
搜索结果
106 条记录 (耗时 0.026 秒)
