公开(公告)号：US20240318177A1

公开(公告)日：2024-09-26

申请号：US18577378

申请日：2022-07-08

申请人： Dyne Therapeutics, Inc.

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 ,  A61K31/7125 ,  A61K47/68 ,  C07K16/28

CPC分类号： C12N15/113 ,  A61K31/7125 ,  A61K47/6807 ,  A61K47/6849 ,  C07K16/2881 ,  C07K2317/55 ,  C07K2317/77 ,  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：US20240318176A1

公开(公告)日：2024-09-26

申请号：US18577374

申请日：2022-07-08

申请人： Dyne Therapeutics, Inc.

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：US20240209119A1

公开(公告)日：2024-06-27

申请号：US18577462

申请日：2022-07-08

申请人： DYNE THERAPEUTICS, INC.

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： C07K19/00 ,  A61K48/00 ,  C07K16/28 ,  C12N15/113

CPC分类号： C07K19/00 ,  A61K48/0058 ,  C07K16/2881 ,  C12N15/113 ,  C12N2310/314 ,  C12N2310/3233

摘要： Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular pay load. 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.