JPWO2021142227A5 - - Google Patents
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- JPWO2021142227A5 JPWO2021142227A5 JP2022542475A JP2022542475A JPWO2021142227A5 JP WO2021142227 A5 JPWO2021142227 A5 JP WO2021142227A5 JP 2022542475 A JP2022542475 A JP 2022542475A JP 2022542475 A JP2022542475 A JP 2022542475A JP WO2021142227 A5 JPWO2021142227 A5 JP WO2021142227A5
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- JP
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- Prior art keywords
- seq
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- antisense strand
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- 230000000692 anti-sense effect Effects 0.000 claims 15
- 125000003729 nucleotide group Chemical group 0.000 claims 14
- 239000002773 nucleotide Substances 0.000 claims 12
- 108091034117 Oligonucleotide Proteins 0.000 claims 9
- 239000002777 nucleoside Substances 0.000 claims 9
- 102100034134 Activin receptor type-1B Human genes 0.000 claims 8
- 101710173011 Activin receptor type-1B Proteins 0.000 claims 8
- 102100039939 Growth/differentiation factor 8 Human genes 0.000 claims 8
- 108010056852 Myostatin Proteins 0.000 claims 8
- 108010019691 inhibin beta A subunit Proteins 0.000 claims 8
- 108010047041 Complementarity Determining Regions Proteins 0.000 claims 7
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 6
- 125000003835 nucleoside group Chemical group 0.000 claims 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 5
- 108020004459 Small interfering RNA Proteins 0.000 claims 4
- 125000003275 alpha amino acid group Chemical group 0.000 claims 4
- 239000003814 drug Substances 0.000 claims 4
- 108091081021 Sense strand Proteins 0.000 claims 3
- 210000000663 muscle cell Anatomy 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 108010054477 Immunoglobulin Fab Fragments Proteins 0.000 claims 2
- 102000001706 Immunoglobulin Fab Fragments Human genes 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 150000004713 phosphodiesters Chemical class 0.000 claims 2
- YIMATHOGWXZHFX-WCTZXXKLSA-N (2r,3r,4r,5r)-5-(hydroxymethyl)-3-(2-methoxyethoxy)oxolane-2,4-diol Chemical compound COCCO[C@H]1[C@H](O)O[C@H](CO)[C@H]1O YIMATHOGWXZHFX-WCTZXXKLSA-N 0.000 claims 1
- 229960000549 4-dimethylaminophenol Drugs 0.000 claims 1
- VHYFNPMBLIVWCW-UHFFFAOYSA-N 4-dimethylaminopyridine Substances CN(C)C1=CC=NC=C1 VHYFNPMBLIVWCW-UHFFFAOYSA-N 0.000 claims 1
- 108010016626 Dipeptides Proteins 0.000 claims 1
- VGGSQFUCUMXWEO-UHFFFAOYSA-N Ethene Chemical compound C=C VGGSQFUCUMXWEO-UHFFFAOYSA-N 0.000 claims 1
- 239000005977 Ethylene Substances 0.000 claims 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 1
- 239000004472 Lysine Substances 0.000 claims 1
- 206010028289 Muscle atrophy Diseases 0.000 claims 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 1
- 230000001668 ameliorated effect Effects 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- 229960002173 citrulline Drugs 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000021615 conjugation Effects 0.000 claims 1
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 210000003205 muscle Anatomy 0.000 claims 1
- 230000020763 muscle atrophy Effects 0.000 claims 1
- 201000000585 muscular atrophy Diseases 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062959469P | 2020-01-10 | 2020-01-10 | |
| US202062959398P | 2020-01-10 | 2020-01-10 | |
| US202062959590P | 2020-01-10 | 2020-01-10 | |
| US62/959,469 | 2020-01-10 | ||
| US62/959,590 | 2020-01-10 | ||
| US62/959,398 | 2020-01-10 | ||
| PCT/US2021/012650 WO2021142227A1 (en) | 2020-01-10 | 2021-01-08 | Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023512441A JP2023512441A (ja) | 2023-03-27 |
| JPWO2021142227A5 true JPWO2021142227A5 (he) | 2024-01-17 |
Family
ID=76788282
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022542475A Pending JP2023512441A (ja) | 2020-01-10 | 2021-01-08 | 筋肉標的複合体および筋肉の健康に関連する遺伝子のモジュレーションのためのその使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230111147A1 (he) |
| EP (1) | EP4087586A4 (he) |
| JP (1) | JP2023512441A (he) |
| CN (1) | CN115335062A (he) |
| CA (1) | CA3163283A1 (he) |
| WO (1) | WO2021142227A1 (he) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | 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 |
| KR20240035825A (ko) | 2021-07-09 | 2024-03-18 | 다인 세라퓨틱스, 인크. | 디스트로핀병증을 치료하기 위한 근육 표적화 복합체 및 제제 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11648318B2 (en) | 2021-07-09 | 2023-05-16 | Dyne Therapeutics, Inc. | Anti-transferrin receptor (TFR) antibody and uses thereof |
| US11633498B2 (en) | 2021-07-09 | 2023-04-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| WO2023121445A1 (en) | 2021-12-22 | 2023-06-29 | Sapreme Technologies B.V. | Compositions comprising therapeutic nucleic acid and saponin for the treatment of muscle-wasting disorders |
| EP4452324A1 (en) | 2021-12-22 | 2024-10-30 | Sapreme Technologies B.V. | Compositions comprising therapeutic nucleic acid and targeted saponin for the treatment of muscle-wasting disorders |
| WO2023121446A1 (en) | 2021-12-22 | 2023-06-29 | Sapreme Technologies B.V. | Targeted saponin-nucleic acid conjugates for treatment of muscle wasting disorders |
| AU2023254846A1 (en) | 2022-04-15 | 2024-10-10 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| WO2023201318A1 (en) * | 2022-04-15 | 2023-10-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| WO2023230562A2 (en) * | 2022-05-26 | 2023-11-30 | Board Of Regents Of The University Ofnebraska | Rna compositions and therapeutic methods thereof |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2007104971A (ja) * | 2005-10-14 | 2007-04-26 | New Industry Research Organization | ミオスタチンのスプライシング制御アンチセンスオリゴヌクレオチド |
| WO2009126913A1 (en) * | 2008-04-11 | 2009-10-15 | Cedars-Sinai Medical Center | Poly(beta malic acid) with pendant leu-leu-leu tripeptide for effective cytoplasmic drug delivery |
| GB0904214D0 (en) * | 2009-03-11 | 2009-04-22 | Ucb Pharma Sa | Biological products |
| KR102188544B1 (ko) * | 2010-11-30 | 2020-12-08 | 제넨테크, 인크. | 저친화도 혈액-뇌 장벽 수용체 항체 및 그의 용도 |
| US10004814B2 (en) * | 2013-11-11 | 2018-06-26 | Sirna Therapeutics, Inc. | Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety |
| US10508151B2 (en) * | 2014-11-19 | 2019-12-17 | Genentech, Inc. | Anti-transferrin receptor antibodies and methods of use |
| US20190240346A1 (en) * | 2016-06-20 | 2019-08-08 | Genahead Bio, Inc. | Antibody-drug conjugate |
| EP3516062A1 (en) * | 2016-09-21 | 2019-07-31 | Alnylam Pharmaceuticals, Inc. | Myostatin irna compositions and methods of use thereof |
| CA3041990A1 (en) * | 2016-12-26 | 2018-07-05 | Jcr Pharmaceuticals Co., Ltd. | Novel anti-human transferrin receptor antibody capable of penetrating blood-brain barrier |
| KR102623786B1 (ko) * | 2017-12-06 | 2024-01-11 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
| AU2019215782A1 (en) * | 2018-02-05 | 2020-07-16 | Jcr Pharmaceuticals Co., Ltd. | Method for delivering drug to muscle |
| US20220378934A1 (en) * | 2018-08-02 | 2022-12-01 | Dyne Therapeutics, Inc. | Muscle-targeting complexes and uses thereof in treating muscle atrophy |
-
2021
- 2021-01-08 CN CN202180018131.0A patent/CN115335062A/zh active Pending
- 2021-01-08 CA CA3163283A patent/CA3163283A1/en active Pending
- 2021-01-08 EP EP21738475.9A patent/EP4087586A4/en active Pending
- 2021-01-08 US US17/791,670 patent/US20230111147A1/en active Pending
- 2021-01-08 WO PCT/US2021/012650 patent/WO2021142227A1/en unknown
- 2021-01-08 JP JP2022542475A patent/JP2023512441A/ja active Pending
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