JP2018183178A5 - - Google Patents
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- Publication number
- JP2018183178A5 JP2018183178A5 JP2018144099A JP2018144099A JP2018183178A5 JP 2018183178 A5 JP2018183178 A5 JP 2018183178A5 JP 2018144099 A JP2018144099 A JP 2018144099A JP 2018144099 A JP2018144099 A JP 2018144099A JP 2018183178 A5 JP2018183178 A5 JP 2018183178A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutically acceptable
- hydrate
- acceptable salt
- antisense oligomer
- oligomer
- 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
- 230000000692 anti-sense effect Effects 0.000 claims 16
- 150000003839 salts Chemical class 0.000 claims 15
- 239000002773 nucleotide Substances 0.000 claims 13
- 125000003729 nucleotide group Chemical group 0.000 claims 13
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 5
- 230000000295 complement effect Effects 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 108091034117 Oligonucleotide Proteins 0.000 claims 4
- 201000006938 muscular dystrophy Diseases 0.000 claims 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 108010069091 Dystrophin Proteins 0.000 claims 2
- 108010058514 Phosphate-Binding Proteins Proteins 0.000 claims 2
- 102000006335 Phosphate-Binding Proteins Human genes 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims 1
- 239000004480 active ingredient Substances 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000002947 alkylene group Chemical group 0.000 claims 1
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 150000004677 hydrates Chemical class 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021179353A JP2022033738A (ja) | 2015-09-15 | 2021-11-02 | アンチセンス核酸 |
| JP2023218764A JP2024038104A (ja) | 2015-09-15 | 2023-12-26 | アンチセンス核酸 |
| JP2025197087A JP2026032058A (ja) | 2015-09-15 | 2025-11-18 | アンチセンス核酸 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2015182145 | 2015-09-15 | ||
| JP2015182145 | 2015-09-15 | ||
| JP2017539975A JP6384845B2 (ja) | 2015-09-15 | 2016-09-15 | アンチセンス核酸 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017539975A Division JP6384845B2 (ja) | 2015-09-15 | 2016-09-15 | アンチセンス核酸 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021179353A Division JP2022033738A (ja) | 2015-09-15 | 2021-11-02 | アンチセンス核酸 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018183178A JP2018183178A (ja) | 2018-11-22 |
| JP2018183178A5 true JP2018183178A5 (enExample) | 2019-10-31 |
| JP6977998B2 JP6977998B2 (ja) | 2021-12-08 |
Family
ID=58289325
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017539975A Active JP6384845B2 (ja) | 2015-09-15 | 2016-09-15 | アンチセンス核酸 |
| JP2018144099A Active JP6977998B2 (ja) | 2015-09-15 | 2018-07-31 | アンチセンス核酸 |
| JP2021179353A Withdrawn JP2022033738A (ja) | 2015-09-15 | 2021-11-02 | アンチセンス核酸 |
| JP2023218764A Pending JP2024038104A (ja) | 2015-09-15 | 2023-12-26 | アンチセンス核酸 |
| JP2025197087A Pending JP2026032058A (ja) | 2015-09-15 | 2025-11-18 | アンチセンス核酸 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017539975A Active JP6384845B2 (ja) | 2015-09-15 | 2016-09-15 | アンチセンス核酸 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021179353A Withdrawn JP2022033738A (ja) | 2015-09-15 | 2021-11-02 | アンチセンス核酸 |
| JP2023218764A Pending JP2024038104A (ja) | 2015-09-15 | 2023-12-26 | アンチセンス核酸 |
| JP2025197087A Pending JP2026032058A (ja) | 2015-09-15 | 2025-11-18 | アンチセンス核酸 |
Country Status (29)
| Country | Link |
|---|---|
| US (4) | US10144931B2 (enExample) |
| EP (2) | EP3778895A1 (enExample) |
| JP (5) | JP6384845B2 (enExample) |
| KR (3) | KR101968880B1 (enExample) |
| CN (3) | CN108026531B (enExample) |
| AU (1) | AU2016324800B2 (enExample) |
| CA (1) | CA2996280C (enExample) |
| CO (1) | CO2018002557A2 (enExample) |
| CY (1) | CY1123119T1 (enExample) |
| DK (1) | DK3351633T3 (enExample) |
| ES (1) | ES2808049T3 (enExample) |
| HR (1) | HRP20201125T1 (enExample) |
| HU (1) | HUE050061T2 (enExample) |
| IL (1) | IL258065B (enExample) |
| LT (1) | LT3351633T (enExample) |
| MX (1) | MX391304B (enExample) |
| MY (1) | MY185390A (enExample) |
| PH (1) | PH12018500568B1 (enExample) |
| PL (1) | PL3351633T3 (enExample) |
| PT (1) | PT3351633T (enExample) |
| RS (1) | RS60493B1 (enExample) |
| RU (1) | RU2724554C2 (enExample) |
| SG (1) | SG11201802138TA (enExample) |
| SI (1) | SI3351633T1 (enExample) |
| SM (1) | SMT202000379T1 (enExample) |
| TW (1) | TWI725990B (enExample) |
| UA (1) | UA123359C2 (enExample) |
| WO (1) | WO2017047707A1 (enExample) |
| ZA (1) | ZA201801682B (enExample) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HRP20201125T1 (hr) * | 2015-09-15 | 2020-10-30 | Nippon Shinyaku Co., Ltd. | Antisens nukleinska kiselina |
| EP3359523A4 (en) | 2015-10-09 | 2019-07-24 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| CA3137715A1 (en) | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| EP4079329A4 (en) * | 2019-12-19 | 2024-04-17 | Nippon Shinyaku Co., Ltd. | ANTISENSE NUCLEIC ACID ENABLING EXON SKIPPING |
| AU2021226089A1 (en) * | 2020-02-28 | 2022-09-15 | National Center Of Neurology And Psychiatry | Antisense nucleic acid inducing skipping of exon 51 |
| CA3216839A1 (en) | 2021-04-30 | 2022-11-03 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| AU2022298028A1 (en) * | 2021-06-23 | 2023-12-21 | National Center Of Neurology And Psychiatry | Combination of antisense oligomers |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CN117980006A (zh) | 2021-07-09 | 2024-05-03 | 达因疗法公司 | 用于治疗肌养蛋白病的肌肉靶向复合物和制剂 |
| 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 |
| KR20240129006A (ko) | 2021-12-27 | 2024-08-27 | 니뽄 신야쿠 가부시키가이샤 | 올리고 핵산 화합물의 제조 방법 |
| US20250170259A1 (en) | 2022-03-03 | 2025-05-29 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| JP2025509438A (ja) | 2022-03-17 | 2025-04-11 | サレプタ セラピューティクス， インコーポレイテッド | ホスホロジアミデートモルホリノオリゴマーコンジュゲート |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US2567664A (en) | 1948-04-22 | 1951-09-11 | Willis G Ewell | Chicken culling device |
| EP0962463B1 (en) | 1989-12-20 | 2002-07-10 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorus-containing chiral intersubunit linkages |
| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
| JP2000325085A (ja) * | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
| JP4836366B2 (ja) * | 2000-08-25 | 2011-12-14 | 雅文 松尾 | デュシェンヌ型筋ジストロフィー治療剤 |
| US6727355B2 (en) * | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| US7902160B2 (en) | 2002-11-25 | 2011-03-08 | Masafumi Matsuo | ENA nucleic acid drugs modifying splicing in mRNA precursor |
| AU2003225410A1 (en) | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| PL1766010T3 (pl) | 2004-06-28 | 2011-07-29 | Univ Western Australia | Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania |
| JPWO2006038608A1 (ja) | 2004-10-05 | 2008-05-15 | 日本新薬株式会社 | オリゴ二本鎖ｒｎａ及び医薬組成物 |
| JP5056413B2 (ja) | 2005-05-30 | 2012-10-24 | 日本新薬株式会社 | 核酸含有複合体製剤の製造方法 |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| WO2008096690A1 (ja) | 2007-02-05 | 2008-08-14 | Nippon Shinyaku Co., Ltd. | ポリエチレングリコール誘導体 |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| EP2203173B1 (en) * | 2007-10-26 | 2015-12-23 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| DK2207779T3 (da) | 2007-11-15 | 2014-07-14 | Sarepta Therapeutics Inc | Fremgangsmåde til syntese af morpholinooligomerer |
| 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 |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| CN105779453A (zh) * | 2008-10-24 | 2016-07-20 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| PT2344637E (pt) | 2008-10-27 | 2015-03-23 | Academisch Ziekenhuis Leiden | Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne |
| EP2421971B1 (en) * | 2009-04-24 | 2016-07-06 | BioMarin Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| ITTO20090487A1 (it) * | 2009-06-26 | 2010-12-27 | Univ Ferrara | Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd) |
| US20120270930A1 (en) * | 2009-10-29 | 2012-10-25 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Methods and compositions for dysferlin exon-skipping |
| BR122023023194A2 (pt) | 2009-11-12 | 2023-12-26 | The University Of Western Australia | Oligonucleotídeo antissentido, composição e respectivo uso |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CA2826836A1 (en) * | 2011-02-08 | 2012-08-16 | The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center | Antisense oligonucleotides |
| CA3132111A1 (en) * | 2011-12-28 | 2013-07-04 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids for skipping of exon 55 of human dystrophin gene |
| CN118581086A (zh) * | 2012-01-27 | 2024-09-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| EP2870246B1 (en) * | 2012-07-03 | 2019-09-11 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| NZ631289A (en) | 2013-03-15 | 2017-08-25 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| RU2730681C2 (ru) | 2014-03-12 | 2020-08-24 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
| JP2015182145A (ja) | 2014-03-20 | 2015-10-22 | キヤノン株式会社 | ロボットシステムの制御方法、およびロボットシステム |
| AU2015277924B2 (en) | 2014-06-17 | 2021-02-25 | National Center Of Neurology And Psychiatry | Antisense nucleic acids |
| HRP20201125T1 (hr) * | 2015-09-15 | 2020-10-30 | Nippon Shinyaku Co., Ltd. | Antisens nukleinska kiselina |
-
2016
- 2016-09-15 HR HRP20201125TT patent/HRP20201125T1/hr unknown
- 2016-09-15 HU HUE16846578A patent/HUE050061T2/hu unknown
- 2016-09-15 CA CA2996280A patent/CA2996280C/en active Active
- 2016-09-15 LT LTEP16846578.9T patent/LT3351633T/lt unknown
- 2016-09-15 CN CN201680053726.9A patent/CN108026531B/zh active Active
- 2016-09-15 PH PH1/2018/500568A patent/PH12018500568B1/en unknown
- 2016-09-15 EP EP20181465.4A patent/EP3778895A1/en active Pending
- 2016-09-15 UA UAA201802093A patent/UA123359C2/uk unknown
- 2016-09-15 RS RS20200811A patent/RS60493B1/sr unknown
- 2016-09-15 SG SG11201802138TA patent/SG11201802138TA/en unknown
- 2016-09-15 KR KR1020187001380A patent/KR101968880B1/ko active Active
- 2016-09-15 SM SM20200379T patent/SMT202000379T1/it unknown
- 2016-09-15 PT PT168465789T patent/PT3351633T/pt unknown
- 2016-09-15 US US15/759,267 patent/US10144931B2/en active Active
- 2016-09-15 JP JP2017539975A patent/JP6384845B2/ja active Active
- 2016-09-15 KR KR1020227012681A patent/KR20220053048A/ko not_active Ceased
- 2016-09-15 MX MX2018002955A patent/MX391304B/es unknown
- 2016-09-15 CN CN202111073060.2A patent/CN113930426A/zh active Pending
- 2016-09-15 AU AU2016324800A patent/AU2016324800B2/en active Active
- 2016-09-15 EP EP16846578.9A patent/EP3351633B1/en active Active
- 2016-09-15 KR KR1020197010069A patent/KR102473431B1/ko active Active
- 2016-09-15 WO PCT/JP2016/077305 patent/WO2017047707A1/ja not_active Ceased
- 2016-09-15 CN CN202111073029.9A patent/CN113913426B/zh active Active
- 2016-09-15 DK DK16846578.9T patent/DK3351633T3/da active
- 2016-09-15 MY MYPI2018000333A patent/MY185390A/en unknown
- 2016-09-15 ES ES16846578T patent/ES2808049T3/es active Active
- 2016-09-15 SI SI201630848T patent/SI3351633T1/sl unknown
- 2016-09-15 RU RU2018113276A patent/RU2724554C2/ru active
- 2016-09-15 PL PL16846578T patent/PL3351633T3/pl unknown
- 2016-09-19 TW TW105130156A patent/TWI725990B/zh active
-
2018
- 2018-03-09 CO CONC2018/0002557A patent/CO2018002557A2/es unknown
- 2018-03-12 ZA ZA2018/01682A patent/ZA201801682B/en unknown
- 2018-03-13 IL IL258065A patent/IL258065B/en unknown
- 2018-07-31 JP JP2018144099A patent/JP6977998B2/ja active Active
- 2018-10-16 US US16/161,946 patent/US10851373B2/en active Active
-
2020
- 2020-07-10 CY CY20201100640T patent/CY1123119T1/el unknown
- 2020-11-17 US US16/950,449 patent/US11981894B2/en active Active
-
2021
- 2021-11-02 JP JP2021179353A patent/JP2022033738A/ja not_active Withdrawn
-
2023
- 2023-12-26 JP JP2023218764A patent/JP2024038104A/ja active Pending
-
2024
- 2024-05-13 US US18/662,046 patent/US20240368597A1/en not_active Abandoned
-
2025
- 2025-11-18 JP JP2025197087A patent/JP2026032058A/ja active Pending
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