JPWO2021142313A5 - - Google Patents
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- JPWO2021142313A5 JPWO2021142313A5 JP2022542337A JP2022542337A JPWO2021142313A5 JP WO2021142313 A5 JPWO2021142313 A5 JP WO2021142313A5 JP 2022542337 A JP2022542337 A JP 2022542337A JP 2022542337 A JP2022542337 A JP 2022542337A JP WO2021142313 A5 JPWO2021142313 A5 JP WO2021142313A5
- Authority
- JP
- Japan
- Prior art keywords
- cdr
- seq
- conjugate
- optionally
- antibody
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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- 208000029578 Muscle disease Diseases 0.000 claims 9
- 108091034117 Oligonucleotide Proteins 0.000 claims 9
- 108010047041 Complementarity Determining Regions Proteins 0.000 claims 7
- 238000000034 method Methods 0.000 claims 6
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 125000003275 alpha amino acid group Chemical group 0.000 claims 4
- 108020003175 receptors Proteins 0.000 claims 4
- 102000005962 receptors Human genes 0.000 claims 4
- 239000012581 transferrin Substances 0.000 claims 4
- 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 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
- 108091030071 RNAI Proteins 0.000 claims 2
- 238000003776 cleavage reaction Methods 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 230000009368 gene silencing by RNA Effects 0.000 claims 2
- 230000001404 mediated effect Effects 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 239000002777 nucleoside Substances 0.000 claims 2
- 125000003835 nucleoside group Chemical group 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 230000007017 scission Effects 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 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
- 206010013801 Duchenne Muscular Dystrophy Diseases 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
- 208000037149 Facioscapulohumeral dystrophy Diseases 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 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
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 1
- 230000001668 ameliorated 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
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 208000008570 facioscapulohumeral muscular dystrophy Diseases 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- 201000009340 myotonic dystrophy type 1 Diseases 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
Applications Claiming Priority (17)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062959804P | 2020-01-10 | 2020-01-10 | |
| US62/959,804 | 2020-01-10 | ||
| US202062965754P | 2020-01-24 | 2020-01-24 | |
| US62/965,754 | 2020-01-24 | ||
| US202062968411P | 2020-01-31 | 2020-01-31 | |
| US62/968,411 | 2020-01-31 | ||
| US202062980925P | 2020-02-24 | 2020-02-24 | |
| US62/980,925 | 2020-02-24 | ||
| US202063055521P | 2020-07-23 | 2020-07-23 | |
| US63/055,521 | 2020-07-23 | ||
| US202063061836P | 2020-08-06 | 2020-08-06 | |
| US63/061,836 | 2020-08-06 | ||
| US202063069067P | 2020-08-23 | 2020-08-23 | |
| US63/069,067 | 2020-08-23 | ||
| US202063132929P | 2020-12-31 | 2020-12-31 | |
| US63/132,929 | 2020-12-31 | ||
| PCT/US2021/012764 WO2021142313A1 (en) | 2020-01-10 | 2021-01-08 | Muscle-targeting complexes and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023510351A JP2023510351A (ja) | 2023-03-13 |
| JPWO2021142313A5 true JPWO2021142313A5 (he) | 2024-01-17 |
Family
ID=76788871
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022542337A Pending JP2023510351A (ja) | 2020-01-10 | 2021-01-08 | 筋標的化複合体およびそれらの使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230226212A1 (he) |
| EP (1) | EP4087876A4 (he) |
| JP (1) | JP2023510351A (he) |
| CN (1) | CN115427448A (he) |
| CA (1) | CA3163608A1 (he) |
| WO (1) | WO2021142313A1 (he) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3565577A4 (en) | 2017-01-06 | 2020-10-07 | Avidity Biosciences, Inc. | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCTION OF EXON SKIP |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | 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 |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | 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 |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| IL294479A (he) * | 2020-01-10 | 2022-09-01 | Dyne Therapeutics Inc | קומפלקסים המכוונים לשרירים ושימושים בהם לטיפול בדיסטרופיה מיוטונית |
| AU2022298028A1 (en) | 2021-06-23 | 2023-12-21 | National Center Of Neurology And Psychiatry | Combination of antisense oligomers |
| KR20240035825A (ko) | 2021-07-09 | 2024-03-18 | 다인 세라퓨틱스, 인크. | 디스트로핀병증을 치료하기 위한 근육 표적화 복합체 및 제제 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| 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 |
| KR20240107202A (ko) | 2021-09-01 | 2024-07-08 | 바이오젠 엠에이 인코포레이티드 | 항-트랜스페린 수용체 항체 및 이의 용도 |
| US12071621B2 (en) * | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
| AU2023254846A1 (en) | 2022-04-15 | 2024-10-10 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating myotonic dystrophy |
| US11912997B2 (en) | 2022-06-15 | 2024-02-27 | Arrowhead Pharmaceuticals, Inc. | RNAi agents for inhibiting expression of Superoxide Dismutase 1 (SOD1), compositions thereof, and methods of use |
| CN118516348A (zh) * | 2024-07-23 | 2024-08-20 | 深川生物科技(山东)股份公司 | 单链核酸或双链核酸及在制备治疗杜氏肌营养不良的药物中的应用 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7241444B2 (en) * | 2002-01-18 | 2007-07-10 | Pierre Fabre Medicament | Anti-IGF-IR antibodies and uses thereof |
| CA3050672A1 (en) * | 2012-02-24 | 2013-08-29 | Abbvie Stemcentrx Llc | Dll3-binding antibodies and drug conjugates thereof to treat cancer |
| EP3565577A4 (en) * | 2017-01-06 | 2020-10-07 | Avidity Biosciences, Inc. | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCTION OF EXON SKIP |
| KR102623786B1 (ko) * | 2017-12-06 | 2024-01-11 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
-
2021
- 2021-01-08 WO PCT/US2021/012764 patent/WO2021142313A1/en unknown
- 2021-01-08 CA CA3163608A patent/CA3163608A1/en active Pending
- 2021-01-08 US US17/791,667 patent/US20230226212A1/en active Pending
- 2021-01-08 CN CN202180022140.7A patent/CN115427448A/zh active Pending
- 2021-01-08 JP JP2022542337A patent/JP2023510351A/ja active Pending
- 2021-01-08 EP EP21738007.0A patent/EP4087876A4/en active Pending
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