JP2015514418A5 - - Google Patents
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- Publication number
- JP2015514418A5 JP2015514418A5 JP2015506930A JP2015506930A JP2015514418A5 JP 2015514418 A5 JP2015514418 A5 JP 2015514418A5 JP 2015506930 A JP2015506930 A JP 2015506930A JP 2015506930 A JP2015506930 A JP 2015506930A JP 2015514418 A5 JP2015514418 A5 JP 2015514418A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- oligonucleotide according
- methylcytosine
- methyluracil
- composition
- 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.)
- Granted
Links
- 108091034117 Oligonucleotide Proteins 0.000 claims 22
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 10
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 6
- 239000002773 nucleotide Substances 0.000 claims 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical group OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 4
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims 4
- 230000003252 repetitive effect Effects 0.000 claims 4
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims 3
- 208000026350 Inborn Genetic disease Diseases 0.000 claims 3
- 239000003814 drug Substances 0.000 claims 3
- 208000016361 genetic disease Diseases 0.000 claims 3
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 2
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical group NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- TWGNOYAGHYUFFR-UHFFFAOYSA-N 5-methylpyrimidine Chemical class CC1=CN=CN=C1 TWGNOYAGHYUFFR-UHFFFAOYSA-N 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 239000004480 active ingredient Substances 0.000 claims 1
- 229940104302 cytosine Drugs 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- 229940035893 uracil Drugs 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261636914P | 2012-04-23 | 2012-04-23 | |
| EP12165139.2 | 2012-04-23 | ||
| US61/636,914 | 2012-04-23 | ||
| EP12165139 | 2012-04-23 | ||
| PCT/NL2013/050306 WO2013162363A1 (en) | 2012-04-23 | 2013-04-23 | RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017133005A Division JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015514418A JP2015514418A (ja) | 2015-05-21 |
| JP2015514418A5 true JP2015514418A5 (enExample) | 2016-06-16 |
| JP6174678B2 JP6174678B2 (ja) | 2017-08-02 |
Family
ID=49483552
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015506930A Active JP6174678B2 (ja) | 2012-04-23 | 2013-04-23 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| JP2017133005A Pending JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| JP2020115868A Active JP7459691B2 (ja) | 2012-04-23 | 2020-07-03 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| JP2022093254A Pending JP2022126711A (ja) | 2012-04-23 | 2022-06-08 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| JP2025061303A Pending JP2025111478A (ja) | 2012-04-23 | 2025-04-02 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017133005A Pending JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| JP2020115868A Active JP7459691B2 (ja) | 2012-04-23 | 2020-07-03 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| JP2022093254A Pending JP2022126711A (ja) | 2012-04-23 | 2022-06-08 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| JP2025061303A Pending JP2025111478A (ja) | 2012-04-23 | 2025-04-02 | 神経筋障害の治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
Country Status (19)
| Country | Link |
|---|---|
| US (5) | US9745576B2 (enExample) |
| EP (4) | EP3228712A1 (enExample) |
| JP (5) | JP6174678B2 (enExample) |
| CN (2) | CN110025628B (enExample) |
| AU (1) | AU2013253074C1 (enExample) |
| BR (1) | BR112014026285B1 (enExample) |
| CA (2) | CA3103101C (enExample) |
| DK (1) | DK2841578T3 (enExample) |
| ES (2) | ES2639066T3 (enExample) |
| HR (1) | HRP20171254T1 (enExample) |
| HU (1) | HUE033431T2 (enExample) |
| LT (1) | LT2841578T (enExample) |
| NZ (1) | NZ700561A (enExample) |
| PL (2) | PL3604536T3 (enExample) |
| PT (1) | PT2841578T (enExample) |
| RS (1) | RS56319B1 (enExample) |
| SI (1) | SI2841578T1 (enExample) |
| SM (1) | SMT201700339T1 (enExample) |
| WO (1) | WO2013162363A1 (enExample) |
Families Citing this family (47)
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| AU2007282224B2 (en) | 2006-08-11 | 2013-08-29 | Vico Therapeutics B.V. | Methods and means for treating DNA repeat instability associated genetic disorders |
| NZ584793A (en) | 2007-10-26 | 2012-05-25 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| US9744183B2 (en) | 2009-07-06 | 2017-08-29 | Wave Life Sciences Ltd. | Nucleic acid prodrugs and methods of use thereof |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| EP2707486B1 (en) * | 2011-04-08 | 2020-06-10 | Zain-Luqman, Rula | Diagnosis and treatment of friedreich's ataxia |
| RU2014105311A (ru) | 2011-07-19 | 2015-08-27 | Уэйв Лайф Сайенсес Пте. Лтд. | Способы синтеза функционализованных нуклеиновых кислот |
| EP4043039A1 (en) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
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| PT2872485T (pt) | 2012-07-13 | 2021-03-05 | Wave Life Sciences Ltd | Grupo auxiliar assimétrico |
| EP2906696B2 (en) | 2012-10-15 | 2022-12-14 | Ionis Pharmaceuticals, Inc. | Methods for modulating c9orf72 expression |
| US10443052B2 (en) * | 2012-10-15 | 2019-10-15 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C9ORF72 expression |
| EP2906697A4 (en) * | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| US10221414B2 (en) | 2013-10-11 | 2019-03-05 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C9ORF72 expression |
| PT3094728T (pt) | 2014-01-16 | 2022-05-19 | Wave Life Sciences Ltd | Desenho quiral |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| EP3119888B1 (en) | 2014-03-19 | 2021-07-28 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| WO2016060919A1 (en) * | 2014-10-14 | 2016-04-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant c9orf72 foci expression by duplex rnas targeting the expanded hexanucleotide repeat |
| SG11201708468YA (en) * | 2015-04-16 | 2017-11-29 | Ionis Pharmaceuticals Inc | Compositions for modulating c9orf72 expression |
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| US20190002887A1 (en) | 2015-12-31 | 2019-01-03 | Ionis Pharmaceuticals, Inc. | Methods for reducing ataxin-2 expression |
| BR112019000061A2 (pt) * | 2016-07-05 | 2019-11-26 | Biomarin Technologies B.V. | comutação e modulação de splicing de pré-mrna compreendendo porções de arcabouço bicíclico, com características melhoradas para o tratamento de distúrbios genéticos |
| JP2020508685A (ja) | 2017-03-03 | 2020-03-26 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
| CA3083582A1 (en) * | 2017-10-23 | 2019-05-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| AU2019310097A1 (en) | 2018-07-25 | 2021-02-04 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing ATXN2 expression |
| 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 |
| 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | 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 |
| AR120341A1 (es) * | 2019-11-01 | 2022-02-09 | Alnylam Pharmaceuticals Inc | COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO |
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| EP4143321A4 (en) * | 2020-05-01 | 2025-02-19 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating atxn1 |
| JP2023542889A (ja) | 2020-09-17 | 2023-10-12 | キュー－ステート バイオサイエンシーズ， インコーポレイテッド | 複数の標的を介して疼痛を処置するための治療用組成物 |
| EP4329774A4 (en) * | 2021-04-28 | 2025-11-05 | Quiver Holdings Inc | THERAPEUTIC COMPOSITIONS FOR THE TREATMENT OF MULTI-TARGET PAIN |
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| WO2025068368A1 (en) * | 2023-09-26 | 2025-04-03 | Katholieke Universiteit Leuven | Inhibitors for treating cancer |
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-
2013
- 2013-04-23 ES ES13720617.3T patent/ES2639066T3/es active Active
- 2013-04-23 SM SM20170339T patent/SMT201700339T1/it unknown
- 2013-04-23 ES ES19194127T patent/ES2983421T3/es active Active
- 2013-04-23 PL PL19194127.7T patent/PL3604536T3/pl unknown
- 2013-04-23 EP EP17170330.9A patent/EP3228712A1/en not_active Ceased
- 2013-04-23 CN CN201811488693.8A patent/CN110025628B/zh active Active
- 2013-04-23 EP EP13720617.3A patent/EP2841578B1/en active Active
- 2013-04-23 CA CA3103101A patent/CA3103101C/en active Active
- 2013-04-23 HU HUE13720617A patent/HUE033431T2/en unknown
- 2013-04-23 PT PT137206173T patent/PT2841578T/pt unknown
- 2013-04-23 LT LTEP13720617.3T patent/LT2841578T/lt unknown
- 2013-04-23 AU AU2013253074A patent/AU2013253074C1/en active Active
- 2013-04-23 CA CA2870697A patent/CA2870697C/en active Active
- 2013-04-23 RS RS20170765A patent/RS56319B1/sr unknown
- 2013-04-23 EP EP19194127.7A patent/EP3604536B1/en active Active
- 2013-04-23 SI SI201330728T patent/SI2841578T1/sl unknown
- 2013-04-23 PL PL13720617T patent/PL2841578T3/pl unknown
- 2013-04-23 NZ NZ700561A patent/NZ700561A/en unknown
- 2013-04-23 WO PCT/NL2013/050306 patent/WO2013162363A1/en not_active Ceased
- 2013-04-23 HR HRP20171254TT patent/HRP20171254T1/hr unknown
- 2013-04-23 CN CN201380021505.XA patent/CN104271741A/zh active Pending
- 2013-04-23 BR BR112014026285-3A patent/BR112014026285B1/pt active IP Right Grant
- 2013-04-23 DK DK13720617.3T patent/DK2841578T3/en active
- 2013-04-23 EP EP24173123.1A patent/EP4400169A3/en active Pending
- 2013-04-23 JP JP2015506930A patent/JP6174678B2/ja active Active
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2014
- 2014-10-23 US US14/522,002 patent/US9745576B2/en active Active
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2017
- 2017-07-06 JP JP2017133005A patent/JP2018027080A/ja active Pending
- 2017-08-14 US US15/676,569 patent/US20180066256A1/en not_active Abandoned
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2019
- 2019-10-22 US US16/660,416 patent/US11345915B2/en active Active
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2020
- 2020-07-03 JP JP2020115868A patent/JP7459691B2/ja active Active
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2022
- 2022-04-25 US US17/728,336 patent/US20220389421A1/en active Pending
- 2022-06-08 JP JP2022093254A patent/JP2022126711A/ja active Pending
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2024
- 2024-06-24 US US18/752,689 patent/US20250011784A1/en active Pending
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2025
- 2025-04-02 JP JP2025061303A patent/JP2025111478A/ja active Pending
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