JP7506601B2 - 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 - Google Patents
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 Download PDFInfo
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- JP7506601B2 JP7506601B2 JP2020528393A JP2020528393A JP7506601B2 JP 7506601 B2 JP7506601 B2 JP 7506601B2 JP 2020528393 A JP2020528393 A JP 2020528393A JP 2020528393 A JP2020528393 A JP 2020528393A JP 7506601 B2 JP7506601 B2 JP 7506601B2
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—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
- 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
- A61K47/6835—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
- A61K47/6849—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 the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- 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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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
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- A—HUMAN NECESSITIES
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- A61K47/00—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
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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
- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/11—Protein-serine/threonine kinases (2.7.11)
- C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
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- Biomedical Technology (AREA)
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- Plant Pathology (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
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- Optics & Photonics (AREA)
- Virology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022162646A JP7564852B2 (ja) | 2017-12-06 | 2022-10-07 | 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 |
| JP2024169131A JP2025000818A (ja) | 2017-12-06 | 2024-09-27 | 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 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762595545P | 2017-12-06 | 2017-12-06 | |
| US62/595,545 | 2017-12-06 | ||
| US201862725883P | 2018-08-31 | 2018-08-31 | |
| US62/725,883 | 2018-08-31 | ||
| PCT/US2018/064359 WO2019113393A1 (en) | 2017-12-06 | 2018-12-06 | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022162646A Division JP7564852B2 (ja) | 2017-12-06 | 2022-10-07 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021507686A JP2021507686A (ja) | 2021-02-25 |
| JP2021507686A5 JP2021507686A5 (https=) | 2022-01-11 |
| JP7506601B2 true JP7506601B2 (ja) | 2024-06-26 |
Family
ID=66751772
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020528393A Active JP7506601B2 (ja) | 2017-12-06 | 2018-12-06 | 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 |
| JP2022162646A Active JP7564852B2 (ja) | 2017-12-06 | 2022-10-07 | 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 |
| JP2024169131A Pending JP2025000818A (ja) | 2017-12-06 | 2024-09-27 | 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 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022162646A Active JP7564852B2 (ja) | 2017-12-06 | 2022-10-07 | 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 |
| JP2024169131A Pending JP2025000818A (ja) | 2017-12-06 | 2024-09-27 | 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 |
Country Status (12)
| Country | Link |
|---|---|
| US (14) | US10881743B2 (https=) |
| EP (1) | EP3720448A4 (https=) |
| JP (3) | JP7506601B2 (https=) |
| KR (5) | KR102867099B1 (https=) |
| CN (4) | CN118638788A (https=) |
| AU (2) | AU2018378812B2 (https=) |
| CA (1) | CA3083526A1 (https=) |
| IL (2) | IL319835A (https=) |
| MA (1) | MA51103A (https=) |
| MX (3) | MX2020005860A (https=) |
| SG (1) | SG11202005022YA (https=) |
| WO (1) | WO2019113393A1 (https=) |
Families Citing this family (65)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2019513371A (ja) | 2016-04-01 | 2019-05-30 | アビディティー バイオサイエンシーズ エルエルシー | 核酸ポリペプチド組成物とその使用 |
| MX2019008199A (es) | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| WO2019071028A1 (en) | 2017-10-04 | 2019-04-11 | Avidity Biosciences Llc | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF |
| 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 |
| MA51583A (fr) * | 2018-01-04 | 2020-11-11 | Avidity Biosciences Inc | Molécules d'acide nucléique hétéroduplex et leurs utilisations |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular 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 |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| 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 |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| CN112930193A (zh) * | 2018-08-02 | 2021-06-08 | 达因疗法公司 | 肌肉靶向复合物及其在治疗肌肉萎缩中的用途 |
| IL312067B2 (en) | 2018-12-21 | 2025-08-01 | Avidity Biosciences Inc | Anti-transferrin receptor antibodies and uses thereof |
| JP7592034B2 (ja) | 2019-06-06 | 2024-11-29 | アビディティー バイオサイエンシーズ,インク. | 核酸ポリペプチド組成物およびその使用 |
| KR20220019259A (ko) | 2019-06-06 | 2022-02-16 | 어비디티 바이오사이언시스 인크. | Una 아미다이트 및 이의 용도 |
| EP4578503A3 (en) * | 2019-07-10 | 2025-09-03 | University of Washington | Non-viral immuno-targeting |
| WO2021020412A1 (ja) * | 2019-07-30 | 2021-02-04 | 塩野義製薬株式会社 | Murf1を標的とする核酸医薬 |
| US20220287347A1 (en) * | 2019-08-20 | 2022-09-15 | Nuritas Limited | Peptides for treating muscle atrophy |
| US20240148891A1 (en) * | 2019-10-18 | 2024-05-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| WO2021142269A1 (en) * | 2020-01-10 | 2021-07-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of genes associated with muscle atrophy |
| KR20220125801A (ko) * | 2020-01-10 | 2022-09-14 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| WO2021142313A1 (en) * | 2020-01-10 | 2021-07-15 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof |
| WO2021142331A1 (en) * | 2020-01-10 | 2021-07-15 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for modulation of genes associated with cardiac muscle disease |
| CA3163283A1 (en) * | 2020-01-10 | 2021-07-15 | Romesh R. Subramanian | Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health |
| JP7780691B2 (ja) * | 2020-01-10 | 2025-12-05 | ダイン セラピューティクス,インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用 |
| AU2021206234A1 (en) * | 2020-01-10 | 2022-09-01 | 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 |
| WO2021150382A1 (en) * | 2020-01-24 | 2021-07-29 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof in treating muscle atrophy |
| CN115427059A (zh) * | 2020-01-31 | 2022-12-02 | 达因疗法公司 | 抗转铁蛋白受体(tfr)抗体及其用途 |
| KR20220163960A (ko) * | 2020-03-06 | 2022-12-12 | 알리고스 테라퓨틱스 인코포레이티드 | 변경된 짧은 개재성 핵산(siNA) 분자 및 이의 용도 |
| US11555190B2 (en) | 2020-03-19 | 2023-01-17 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
| AU2021241682B2 (en) * | 2020-03-27 | 2026-02-12 | Avidity Biosciences, Inc. | Compositions and methods of treating muscle dystrophy |
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