公开(公告)号：US20240100177A1

公开(公告)日：2024-03-28

申请号：US18265065

申请日：2021-12-03

申请人： Dyne Therapeutics, Inc.

发明人： Scott Hilderbrand ,  John Najim ,  Qifeng Qiu ,  Benjamin Vieira ,  Timothy Weeden ,  Sean Spring

IPC分类号： A61K47/68

CPC分类号： A61K47/6807 ,  A61K47/6889 ,  A61K47/6849

摘要： Provided herein are oligonucleotide-antibody complexes, methods of preparing the complexes, and methods of using the complexes (e.g., treating muscle diseases). In particular, provided are compounds of Formula (I) and various methods for the preparation of the compounds. Also provided are pharmaceutical compositions comprising compounds of Formula (I) and methods of treating muscle disease in a subject by administering a compound or composition described herein.

公开(公告)号：US12128109B2

公开(公告)日：2024-10-29

申请号：US18455351

申请日：2023-08-24

申请人： Dyne Therapeutics, Inc.

发明人： Timothy Weeden ,  Scott Hilderbrand ,  Sean Spring ,  Peiyi Shen ,  Cody A. Desjardins ,  Romesh R. Subramanian ,  Mohammed T. Qatanani ,  Brendan Quinn ,  John Najim

IPC分类号： A61K39/00 ,  A61K47/54 ,  A61K47/65 ,  A61K47/68 ,  A61P21/00

CPC分类号： A61K47/6807 ,  A61K47/545 ,  A61K47/548 ,  A61K47/65 ,  A61K47/6849 ,  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：US20240016950A1

公开(公告)日：2024-01-18

申请号：US18455351

申请日：2023-08-24

申请人： Dyne Therapeutics, Inc.

发明人： Timothy Weeden ,  Scott Hilderbrand ,  Sean Spring ,  Peiyi Shen ,  Cody A. Desjardins ,  Romesh R. Subramanian ,  Mohammed T. Qatanani ,  Brendan Quinn ,  John Najim

IPC分类号： A61K47/68 ,  A61K47/54 ,  A61K47/65 ,  A61P21/00

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：US20230330562A1

公开(公告)日：2023-10-19

申请号：US18024486

申请日：2021-09-03

申请人： Dyne Therapeutics, Inc.

发明人： Timothy Weeden ,  Scott Hilderbrand ,  Sean Spring ,  Peiyi Shen ,  Benjamin Vieira

IPC分类号： B01D15/38 ,  A61K47/68

CPC分类号： B01D15/3847 ,  A61K47/6807 ,  A61K47/6849

摘要： Aspects of the disclosure relate to methods of purifying complexes comprising a protein (e.g., antibody) covalently linked to a molecular payload (e.g., a charge-neutral oligonucleotide, a charged oligonucleotide, or a hydrophobic small molecule) using mixed-mode resin that comprises positively-charged metal sites and negatively charged ionic sites, e.g., hydroxyapatite resin. Methods of producing the complexes are also provided.

公开(公告)号：US20220306685A1

公开(公告)日：2022-09-29

申请号：US17616870

申请日：2020-06-05

申请人： Dyne Therapeutics, Inc.

发明人： Timothy Weeden ,  Sean Spring

IPC分类号： C07K1/20 ,  C07K16/28 ,  A61K47/68 ,  C12N15/113 ,  C07K1/16

摘要： Aspects of the disclosure relate to methods of purifying complexes comprising a protein (e.g., antibody) covalently linked to an oligonucleotide. In some embodiments, complexes comprising a protein covalently linked to an oligonucleotide are purified and isolated from unlinked oligonucleotide using an mixed-mode resin that comprises positively-charged metal sites and negatively charged ionic sites, e.g., hydroxyapatite resin. In some embodiments, complexes comprising a protein covalently linked to an oligonucleotide are purified from a mixture comprising the complexes, unlinked protein, and unlinked oligonucleotide using a purification step involving hydrophobic interaction chromatography resin followed by a purification step involving mixed-mode resin.