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 (2)
| 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 |
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 (4)
| 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 |
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 (2)
| 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 |
Country Status (29)
| Country | Link |
|---|---|
| US (4) | US10144931B2 (enExample) |
| EP (2) | EP3778895A1 (enExample) |
| JP (4) | JP6384845B2 (enExample) |
| KR (3) | KR102473431B1 (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 (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3778895A1 (en) * | 2015-09-15 | 2021-02-17 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
| CN114533900A (zh) | 2015-10-09 | 2022-05-27 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| 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 |
| 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 |
| 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 |
| US12497615B2 (en) | 2019-04-25 | 2025-12-16 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | 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 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CA3226366A1 (en) | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CA3244382A1 (en) | 2021-12-27 | 2025-02-26 | Nippon Shinyaku Co., Ltd. | PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| AU2023235302A1 (en) | 2022-03-17 | 2024-10-24 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
| 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 |
| ATE312834T1 (de) | 1989-12-20 | 2005-12-15 | Avi Biopharma Inc | Ungeladene, auf morpholin basierende polymere mit chiralen, phosphor enthaltenden brücken zwischen den untereinheiten |
| 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 |
| ES2509140T3 (es) | 2002-11-25 | 2014-10-17 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
| 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 |
| HRP20110352T1 (hr) | 2004-06-28 | 2011-06-30 | The University Of Western Australia | Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe |
| EP1811024A1 (en) | 2004-10-05 | 2007-07-25 | Nippon Shinyaku Co., Ltd. | Oligo double-stranded rna and medicinal composition |
| WO2006129594A1 (ja) | 2005-05-30 | 2006-12-07 | Nippon Shinyaku Co., Ltd. | 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 |
| EP2119738B1 (en) | 2007-02-05 | 2014-04-16 | Nippon Shinyaku Co., Ltd. | Polyethylene glycol derivative |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| CA2704049A1 (en) * | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| CA2704261C (en) | 2007-11-15 | 2015-05-26 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| 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 |
| WO2010048586A1 (en) | 2008-10-24 | 2010-04-29 | Avi Biopharma, Inc. | Multiple exon skipping compositions for dmd |
| SI2607484T1 (sl) | 2008-10-27 | 2016-11-30 | Biomarin Technologies B.V. | Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije |
| 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 |
| KR102113306B1 (ko) | 2009-11-12 | 2020-05-21 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US9078911B2 (en) | 2011-02-08 | 2015-07-14 | The Charlotte-Mecklenburg Hospital Authority | Antisense oligonucleotides |
| CN104024414A (zh) * | 2011-12-28 | 2014-09-03 | 日本新药株式会社 | 反义核酸 |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| JP6460983B2 (ja) * | 2012-07-03 | 2019-01-30 | バイオマリン テクノロジーズ ベー．フェー． | 筋ジストロフィー患者の治療のためのオリゴヌクレオチド |
| CN113633787A (zh) | 2013-03-15 | 2021-11-12 | 萨勒普塔医疗公司 | 改进的用于治疗肌营养不良的组合物 |
| HUE042283T2 (hu) | 2014-03-12 | 2019-06-28 | Nippon Shinyaku Co Ltd | Antiszensz nukleinsav |
| 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 |
| SI3159409T1 (sl) | 2014-06-17 | 2020-02-28 | Nippon Shinyaku Co., Ltd. | Protismiselna nukleinska kislina za uporabo pri zdravljenju Duchennove mišične distrofije |
| EP3778895A1 (en) * | 2015-09-15 | 2021-02-17 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
-
2016
- 2016-09-15 EP EP20181465.4A patent/EP3778895A1/en active Pending
- 2016-09-15 SG SG11201802138TA patent/SG11201802138TA/en unknown
- 2016-09-15 SM SM20200379T patent/SMT202000379T1/it unknown
- 2016-09-15 KR KR1020197010069A patent/KR102473431B1/ko active Active
- 2016-09-15 CA CA2996280A patent/CA2996280C/en active Active
- 2016-09-15 US US15/759,267 patent/US10144931B2/en active Active
- 2016-09-15 UA UAA201802093A patent/UA123359C2/uk unknown
- 2016-09-15 CN CN201680053726.9A patent/CN108026531B/zh active Active
- 2016-09-15 RU RU2018113276A patent/RU2724554C2/ru active
- 2016-09-15 WO PCT/JP2016/077305 patent/WO2017047707A1/ja not_active Ceased
- 2016-09-15 JP JP2017539975A patent/JP6384845B2/ja active Active
- 2016-09-15 PT PT168465789T patent/PT3351633T/pt unknown
- 2016-09-15 EP EP16846578.9A patent/EP3351633B1/en active Active
- 2016-09-15 AU AU2016324800A patent/AU2016324800B2/en active Active
- 2016-09-15 KR KR1020227012681A patent/KR20220053048A/ko not_active Ceased
- 2016-09-15 CN CN202111073029.9A patent/CN113913426B/zh active Active
- 2016-09-15 KR KR1020187001380A patent/KR101968880B1/ko active Active
- 2016-09-15 PH PH1/2018/500568A patent/PH12018500568B1/en unknown
- 2016-09-15 LT LTEP16846578.9T patent/LT3351633T/lt unknown
- 2016-09-15 ES ES16846578T patent/ES2808049T3/es active Active
- 2016-09-15 MX MX2018002955A patent/MX391304B/es unknown
- 2016-09-15 MY MYPI2018000333A patent/MY185390A/en unknown
- 2016-09-15 HR HRP20201125TT patent/HRP20201125T1/hr unknown
- 2016-09-15 PL PL16846578T patent/PL3351633T3/pl unknown
- 2016-09-15 HU HUE16846578A patent/HUE050061T2/hu unknown
- 2016-09-15 SI SI201630848T patent/SI3351633T1/sl unknown
- 2016-09-15 CN CN202111073060.2A patent/CN113930426A/zh active Pending
- 2016-09-15 DK DK16846578.9T patent/DK3351633T3/da active
- 2016-09-15 RS RS20200811A patent/RS60493B1/sr 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
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