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) | EP4400169A3 (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)
| Publication number | Priority date | Publication date | Assignee | Title |
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| PL2049664T3 (pl) | 2006-08-11 | 2012-02-29 | Biomarin Tech Bv | Jednoniciowe oligonukleotydy komplementarne do powtarzalnych elementów do leczenia chorób genetycznych związanych z niestabilnością powtórzeń DNA |
| CA2704049A1 (en) | 2007-10-26 | 2009-04-30 | 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 |
| ES2616051T3 (es) | 2008-12-02 | 2017-06-09 | Wave Life Sciences Japan, Inc. | Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| WO2012138289A1 (en) * | 2011-04-08 | 2012-10-11 | Zain-Luqman Rula | Diagnosis and treatment of friedreich's ataxia |
| JP6128529B2 (ja) | 2011-07-19 | 2017-05-17 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | 官能化核酸の合成のための方法 |
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| AU2013253074C1 (en) * | 2012-04-23 | 2018-06-14 | Biomarin Technologies B.V. | RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
| US9598458B2 (en) | 2012-07-13 | 2017-03-21 | Wave Life Sciences Japan, Inc. | Asymmetric auxiliary group |
| KR20150070278A (ko) * | 2012-10-15 | 2015-06-24 | 아이시스 파마수티컬즈 인코포레이티드 | C9orf72 발현 조절용 조성물 |
| EP2906697A4 (en) * | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| US9963699B2 (en) | 2012-10-15 | 2018-05-08 | Ionis Pharmaceuticals, Inc. | Methods for modulating C9ORF72 expression |
| EP3055414A4 (en) | 2013-10-11 | 2017-07-19 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| DK3094728T3 (da) | 2014-01-16 | 2022-05-16 | Wave Life Sciences Ltd | Kiralt design |
| SMT202100562T1 (it) | 2014-03-19 | 2021-11-12 | Ionis Pharmaceuticals Inc | Composizioni per modulare l'espressione di ataxina 2 |
| WO2015143245A1 (en) | 2014-03-19 | 2015-09-24 | Isis Pharmaceuticals, Inc. | Methods 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 |
| SMT202000165T1 (it) | 2015-04-16 | 2020-05-08 | Ionis Pharmaceuticals Inc | Composizioni per modulare l'espressione di c9orf72 |
| WO2017079291A1 (en) | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| WO2017117496A1 (en) | 2015-12-31 | 2017-07-06 | Ionis Pharmaceuticals, Inc. | Methods for reducing ataxin-2 expression |
| JP2019522972A (ja) * | 2016-07-05 | 2019-08-22 | バイオマリン テクノロジーズ ベー．フェー． | 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド |
| GB2574769A (en) | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
| KR20200075865A (ko) * | 2017-10-23 | 2020-06-26 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환에 대한 유전자 요법 |
| SG11202100077PA (en) | 2018-07-25 | 2021-02-25 | 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 |
| JP7611825B2 (ja) | 2018-08-02 | 2025-01-10 | 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 |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| 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 |
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-
2013
- 2013-04-23 AU AU2013253074A patent/AU2013253074C1/en active Active
- 2013-04-23 PL PL19194127.7T patent/PL3604536T3/pl unknown
- 2013-04-23 ES ES13720617.3T patent/ES2639066T3/es active Active
- 2013-04-23 NZ NZ700561A patent/NZ700561A/en unknown
- 2013-04-23 CA CA3103101A patent/CA3103101C/en active Active
- 2013-04-23 EP EP24173123.1A patent/EP4400169A3/en active Pending
- 2013-04-23 HU HUE13720617A patent/HUE033431T2/en unknown
- 2013-04-23 CN CN201811488693.8A patent/CN110025628B/zh active Active
- 2013-04-23 ES ES19194127T patent/ES2983421T3/es active Active
- 2013-04-23 EP EP17170330.9A patent/EP3228712A1/en not_active Ceased
- 2013-04-23 RS RS20170765A patent/RS56319B1/sr unknown
- 2013-04-23 DK DK13720617.3T patent/DK2841578T3/en active
- 2013-04-23 HR HRP20171254TT patent/HRP20171254T1/hr unknown
- 2013-04-23 EP EP13720617.3A patent/EP2841578B1/en active Active
- 2013-04-23 SM SM20170339T patent/SMT201700339T1/it unknown
- 2013-04-23 WO PCT/NL2013/050306 patent/WO2013162363A1/en not_active Ceased
- 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 JP JP2015506930A patent/JP6174678B2/ja active Active
- 2013-04-23 CA CA2870697A patent/CA2870697C/en active Active
- 2013-04-23 PT PT137206173T patent/PT2841578T/pt unknown
- 2013-04-23 SI SI201330728T patent/SI2841578T1/sl unknown
- 2013-04-23 LT LTEP13720617.3T patent/LT2841578T/lt unknown
- 2013-04-23 EP EP19194127.7A patent/EP3604536B1/en active Active
- 2013-04-23 PL PL13720617T patent/PL2841578T3/pl unknown
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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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