CN112955154A - 肌肉靶向复合物及其用于治疗强直性肌营养不良的用途 - Google Patents
肌肉靶向复合物及其用于治疗强直性肌营养不良的用途 Download PDFInfo
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- CN112955154A CN112955154A CN201980062906.7A CN201980062906A CN112955154A CN 112955154 A CN112955154 A CN 112955154A CN 201980062906 A CN201980062906 A CN 201980062906A CN 112955154 A CN112955154 A CN 112955154A
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
- A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
- A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
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- A61K47/6889—Conjugates wherein the antibody being the modifying agent and wherein the linker, binder or spacer confers particular properties to the conjugates, e.g. peptidic enzyme-labile linkers or acid-labile linkers, providing for an acid-labile immuno conjugate wherein the drug may be released from its antibody conjugated part in an acidic, e.g. tumoural or environment
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- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
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- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
- C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
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- C07K2317/622—Single chain antibody (scFv)
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- C07K2317/77—Internalization into the cell
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- C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
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Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862713914P | 2018-08-02 | 2018-08-02 | |
| US62/713,914 | 2018-08-02 | ||
| US201862779161P | 2018-12-13 | 2018-12-13 | |
| US62/779,161 | 2018-12-13 | ||
| US201962855761P | 2019-05-31 | 2019-05-31 | |
| US62/855,761 | 2019-05-31 | ||
| US201962858888P | 2019-06-07 | 2019-06-07 | |
| US62/858,888 | 2019-06-07 | ||
| US201962859672P | 2019-06-10 | 2019-06-10 | |
| US62/859,672 | 2019-06-10 | ||
| PCT/US2019/044987 WO2020028861A1 (en) | 2018-08-02 | 2019-08-02 | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN112955154A true CN112955154A (zh) | 2021-06-11 |
Family
ID=69230803
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980062906.7A Pending CN112955154A (zh) | 2018-08-02 | 2019-08-02 | 肌肉靶向复合物及其用于治疗强直性肌营养不良的用途 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210308272A1 (enExample) |
| EP (1) | EP3829596A4 (enExample) |
| JP (2) | JP7705793B2 (enExample) |
| KR (1) | KR20210081323A (enExample) |
| CN (1) | CN112955154A (enExample) |
| AU (2) | AU2019314538B2 (enExample) |
| CA (1) | CA3108287A1 (enExample) |
| IL (2) | IL321905A (enExample) |
| MX (1) | MX2021001283A (enExample) |
| SG (1) | SG11202100925VA (enExample) |
| WO (1) | WO2020028861A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7506601B2 (ja) | 2017-12-06 | 2024-06-26 | アビディティー バイオサイエンシーズ,インク. | 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法 |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| WO2021142217A1 (en) * | 2020-01-10 | 2021-07-15 | Dyne Thereapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of milck1 |
| EP4656242A2 (en) * | 2020-03-27 | 2025-12-03 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle dystrophy |
| KR20230046297A (ko) * | 2020-07-23 | 2023-04-05 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| MX2023000958A (es) * | 2020-07-23 | 2023-04-14 | Dyne Therapeutics Inc | Complejos dirigidos al musculo y usos de estos para el tratamiento de la distrofia miotonica. |
| WO2022056266A2 (en) * | 2020-09-11 | 2022-03-17 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use |
| WO2022120132A1 (en) * | 2020-12-04 | 2022-06-09 | Dyne Therapeutics, Inc. | Antibody-oligonucleotide complexes and uses thereof |
| EP4271816A4 (en) * | 2020-12-31 | 2025-04-02 | Dyne Therapeutics, Inc. | MUSCLE TARGETING COMPLEXES AND RELATED USES FOR THE TREATMENT OF MYOTONIC DYSTROPHY |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| MX2024000489A (es) | 2021-07-09 | 2024-04-09 | Dyne Therapeutics Inc | Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías. |
| US11672872B2 (en) | 2021-07-09 | 2023-06-13 | Dyne Therapeutics, Inc. | Anti-transferrin receptor antibody and uses thereof |
| EP4124345A1 (en) * | 2021-07-30 | 2023-02-01 | 4basebio UK Ltd | Nanoparticles for muscle delivery |
| CA3226457A1 (en) * | 2021-07-30 | 2023-02-02 | Amy WALKER | Nanoparticles and peptides for the delivery of cargos to muscle cells |
| KR20240107202A (ko) | 2021-09-01 | 2024-07-08 | 바이오젠 엠에이 인코포레이티드 | 항-트랜스페린 수용체 항체 및 이의 용도 |
| WO2023034870A2 (en) | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
| CA3255934A1 (en) | 2022-04-15 | 2023-10-19 | Dyne Therapeutics, Inc. | MUSCLE TARGETTING COMPLEXES AND FORMULATIONS FOR THE TREATMENT OF MYOTONIC DYSTROPHY |
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