JPWO2020247818A5 - - Google Patents
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- JPWO2020247818A5 JPWO2020247818A5 JP2021572519A JP2021572519A JPWO2020247818A5 JP WO2020247818 A5 JPWO2020247818 A5 JP WO2020247818A5 JP 2021572519 A JP2021572519 A JP 2021572519A JP 2021572519 A JP2021572519 A JP 2021572519A JP WO2020247818 A5 JPWO2020247818 A5 JP WO2020247818A5
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- JP
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- Prior art keywords
- substituted
- unsubstituted
- alkyl
- compound
- formula
- 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.)
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- 229910052739 hydrogen Inorganic materials 0.000 claims 17
- 239000001257 hydrogen Substances 0.000 claims 17
- 150000002431 hydrogen Chemical class 0.000 claims 15
- 125000000217 alkyl group Chemical group 0.000 claims 13
- 150000001875 compounds Chemical class 0.000 claims 13
- 108091034117 Oligonucleotide Proteins 0.000 claims 12
- 150000003839 salts Chemical class 0.000 claims 9
- 239000012453 solvate Substances 0.000 claims 9
- 125000003118 aryl group Chemical group 0.000 claims 8
- 125000000592 heterocycloalkyl group Chemical group 0.000 claims 7
- 125000000753 cycloalkyl group Chemical group 0.000 claims 6
- YZCKVEUIGOORGS-OUBTZVSYSA-N Deuterium Chemical compound [2H] YZCKVEUIGOORGS-OUBTZVSYSA-N 0.000 claims 5
- 229910052805 deuterium Inorganic materials 0.000 claims 5
- 125000001072 heteroaryl group Chemical group 0.000 claims 5
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims 4
- 125000002733 (C1-C6) fluoroalkyl group Chemical group 0.000 claims 3
- 125000002103 4,4'-dimethoxytriphenylmethyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)(C1=C([H])C([H])=C(OC([H])([H])[H])C([H])=C1[H])C1=C([H])C([H])=C(OC([H])([H])[H])C([H])=C1[H] 0.000 claims 3
- -1 acetylisobutyl Chemical group 0.000 claims 3
- 229910052757 nitrogen Inorganic materials 0.000 claims 3
- 125000004433 nitrogen atom Chemical group N* 0.000 claims 3
- 125000006735 (C1-C20) heteroalkyl group Chemical group 0.000 claims 2
- 125000006374 C2-C10 alkenyl group Chemical group 0.000 claims 2
- 125000005865 C2-C10alkynyl group Chemical group 0.000 claims 2
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 2
- 208000026350 Inborn Genetic disease Diseases 0.000 claims 2
- 206010028289 Muscle atrophy Diseases 0.000 claims 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 125000003709 fluoroalkyl group Chemical group 0.000 claims 2
- 208000016361 genetic disease Diseases 0.000 claims 2
- 229910052736 halogen Inorganic materials 0.000 claims 2
- 150000002367 halogens Chemical class 0.000 claims 2
- 201000000585 muscular atrophy Diseases 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 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
- 208000024172 Cardiovascular disease Diseases 0.000 claims 1
- 239000005977 Ethylene Substances 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 150000001408 amides Chemical class 0.000 claims 1
- 125000001797 benzyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])* 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- NAGJZTKCGNOGPW-UHFFFAOYSA-K dioxido-sulfanylidene-sulfido-$l^{5}-phosphane Chemical compound [O-]P([O-])([S-])=S NAGJZTKCGNOGPW-UHFFFAOYSA-K 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 claims 1
- 125000005647 linker group Chemical group 0.000 claims 1
- YACKEPLHDIMKIO-UHFFFAOYSA-N methylphosphonic acid Chemical compound CP(O)(O)=O YACKEPLHDIMKIO-UHFFFAOYSA-N 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 208000018360 neuromuscular disease Diseases 0.000 claims 1
- 150000007523 nucleic acids Chemical class 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024190019A JP2025028839A (ja) | 2019-06-06 | 2024-10-29 | Ｕｎａアミダイトおよびその使用 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962858289P | 2019-06-06 | 2019-06-06 | |
| US62/858,289 | 2019-06-06 | ||
| PCT/US2020/036420 WO2020247818A1 (en) | 2019-06-06 | 2020-06-05 | Una amidites and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024190019A Division JP2025028839A (ja) | 2019-06-06 | 2024-10-29 | Ｕｎａアミダイトおよびその使用 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022535911A JP2022535911A (ja) | 2022-08-10 |
| JP2022535911A5 JP2022535911A5 (https=) | 2023-05-08 |
| JPWO2020247818A5 true JPWO2020247818A5 (https=) | 2023-05-08 |
| JP7581252B2 JP7581252B2 (ja) | 2024-11-12 |
Family
ID=73651180
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021572519A Active JP7581252B2 (ja) | 2019-06-06 | 2020-06-05 | Ｕｎａアミダイトおよびその使用 |
| JP2024190019A Pending JP2025028839A (ja) | 2019-06-06 | 2024-10-29 | Ｕｎａアミダイトおよびその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024190019A Pending JP2025028839A (ja) | 2019-06-06 | 2024-10-29 | Ｕｎａアミダイトおよびその使用 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US12006499B2 (https=) |
| EP (1) | EP3980437A4 (https=) |
| JP (2) | JP7581252B2 (https=) |
| KR (1) | KR20220019259A (https=) |
| CN (1) | CN114375297A (https=) |
| AU (1) | AU2020287880B2 (https=) |
| CA (1) | CA3142337A1 (https=) |
| WO (1) | WO2020247818A1 (https=) |
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| WO2019071028A1 (en) | 2017-10-04 | 2019-04-11 | Avidity Biosciences Llc | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | 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 |
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF 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 |
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| CN109310765A (zh) | 2016-06-20 | 2019-02-05 | 领先基因生物技术股份有限公司 | 抗体-药物偶联物 |
| 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. |
| KR20200019127A (ko) * | 2017-06-16 | 2020-02-21 | 에자이 알앤드디 매니지먼트 가부시키가이샤 | 변형 핵산 단량체 화합물 및 올리고핵산 유사체 |
| 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 |
| KR20220019259A (ko) | 2019-06-06 | 2022-02-16 | 어비디티 바이오사이언시스 인크. | Una 아미다이트 및 이의 용도 |
| 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 |
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2020
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- 2020-06-05 CN CN202080055883.XA patent/CN114375297A/zh active Pending
- 2020-06-05 JP JP2021572519A patent/JP7581252B2/ja active Active
- 2020-06-05 WO PCT/US2020/036420 patent/WO2020247818A1/en not_active Ceased
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- 2024-10-29 JP JP2024190019A patent/JP2025028839A/ja active Pending
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