-
公开(公告)号：US20230111212A1
公开(公告)日：2023-04-13
申请号：US17791701
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
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：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.
-
公开(公告)号：US20230287108A1
公开(公告)日：2023-09-14
申请号：US18017180
申请日：2021-07-09
发明人： Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Jason P. Rhodes , Cody A Desjardins , Brendan Quinn , John Najim
CPC分类号： C07K16/28 , A61K47/6807 , A61K47/6849 , C12N15/111 , C07K2317/55 , C07K2317/77 , C12N2310/11 , 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 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.
-
4.发明申请公开(公告)号：US20230088865A1
公开(公告)日：2023-03-23
申请号：US17791697
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
IPC分类号： C07K16/28 , A61P21/00 , 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.
-
公开(公告)号：US20210261680A1
公开(公告)日：2021-08-26
申请号：US17264998
申请日：2019-08-02
IPC分类号： C07K16/28 , A61K47/68 , A61P21/00 , 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 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.
-
公开(公告)号：US20210228730A1
公开(公告)日：2021-07-29
申请号：US17205139
申请日：2021-03-18
IPC分类号： A61K47/68 , A61K47/54 , A61K9/00 , C12N15/113 , 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.
-
-
-
-
-
搜索结果
6 条记录 (耗时 0.014 秒)
