JP2020072724A5 - - Google Patents
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- JP2020072724A5 JP2020072724A5 JP2020003338A JP2020003338A JP2020072724A5 JP 2020072724 A5 JP2020072724 A5 JP 2020072724A5 JP 2020003338 A JP2020003338 A JP 2020003338A JP 2020003338 A JP2020003338 A JP 2020003338A JP 2020072724 A5 JP2020072724 A5 JP 2020072724A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- morpholino oligomer
- morpholino
- nucleotides
- 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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- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 4
- 125000002091 cationic group Chemical group 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 1
- 229920006317 cationic polymer Polymers 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 description 2
- UCLQCQZSVIFYKB-UHFFFAOYSA-N CN(C)P(N(CC1)CCN1C(OCCOCCOCCO)=O)(OC)=O Chemical compound CN(C)P(N(CC1)CCN1C(OCCOCCOCCO)=O)(OC)=O UCLQCQZSVIFYKB-UHFFFAOYSA-N 0.000 description 1
- 238000007857 nested PCR Methods 0.000 description 1
- 238000003757 reverse transcription PCR Methods 0.000 description 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2010196032 | 2010-09-01 | ||
| JP2010196032 | 2010-09-01 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019001091A Division JP6647430B2 (ja) | 2010-09-01 | 2019-01-08 | アンチセンス核酸 |
Related Child Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021008202A Division JP6867636B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008200A Division JP6867620B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008201A Division JP6867621B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020072724A JP2020072724A (ja) | 2020-05-14 |
| JP2020072724A5 true JP2020072724A5 (OSRAM) | 2021-03-04 |
| JP6867619B2 JP6867619B2 (ja) | 2021-04-28 |
Family
ID=45773054
Family Applications (12)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012531987A Active JP5363655B2 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
| JP2013184193A Active JP6141728B2 (ja) | 2010-09-01 | 2013-09-05 | アンチセンス核酸 |
| JP2015256962A Active JP6193343B2 (ja) | 2010-09-01 | 2015-12-28 | アンチセンス核酸 |
| JP2017154320A Active JP6465932B2 (ja) | 2010-09-01 | 2017-08-09 | アンチセンス核酸 |
| JP2019001091A Active JP6647430B2 (ja) | 2010-09-01 | 2019-01-08 | アンチセンス核酸 |
| JP2020003338A Active JP6867619B2 (ja) | 2010-09-01 | 2020-01-14 | アンチセンス核酸 |
| JP2021008202A Active JP6867636B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008200A Active JP6867620B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008201A Active JP6867621B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021058526A Pending JP2021104037A (ja) | 2010-09-01 | 2021-03-30 | アンチセンス核酸 |
| JP2022209270A Pending JP2023036865A (ja) | 2010-09-01 | 2022-12-27 | アンチセンス核酸 |
| JP2024145055A Pending JP2024170458A (ja) | 2010-09-01 | 2024-08-27 | アンチセンス核酸 |
Family Applications Before (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012531987A Active JP5363655B2 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
| JP2013184193A Active JP6141728B2 (ja) | 2010-09-01 | 2013-09-05 | アンチセンス核酸 |
| JP2015256962A Active JP6193343B2 (ja) | 2010-09-01 | 2015-12-28 | アンチセンス核酸 |
| JP2017154320A Active JP6465932B2 (ja) | 2010-09-01 | 2017-08-09 | アンチセンス核酸 |
| JP2019001091A Active JP6647430B2 (ja) | 2010-09-01 | 2019-01-08 | アンチセンス核酸 |
Family Applications After (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021008202A Active JP6867636B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008200A Active JP6867620B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021008201A Active JP6867621B1 (ja) | 2010-09-01 | 2021-01-21 | アンチセンス核酸 |
| JP2021058526A Pending JP2021104037A (ja) | 2010-09-01 | 2021-03-30 | アンチセンス核酸 |
| JP2022209270A Pending JP2023036865A (ja) | 2010-09-01 | 2022-12-27 | アンチセンス核酸 |
| JP2024145055A Pending JP2024170458A (ja) | 2010-09-01 | 2024-08-27 | アンチセンス核酸 |
Country Status (20)
| Country | Link |
|---|---|
| US (16) | US9079934B2 (OSRAM) |
| EP (6) | EP4403632A3 (OSRAM) |
| JP (12) | JP5363655B2 (OSRAM) |
| KR (1) | KR101310569B1 (OSRAM) |
| CN (1) | CN103154245B (OSRAM) |
| AU (1) | AU2011296882B2 (OSRAM) |
| CA (1) | CA2809637C (OSRAM) |
| CY (2) | CY1117367T1 (OSRAM) |
| DK (2) | DK2612917T3 (OSRAM) |
| ES (2) | ES2567411T3 (OSRAM) |
| HR (2) | HRP20160336T1 (OSRAM) |
| HU (2) | HUE027321T2 (OSRAM) |
| LT (1) | LT3018211T (OSRAM) |
| PL (2) | PL2612917T3 (OSRAM) |
| PT (1) | PT3018211T (OSRAM) |
| RS (2) | RS59361B1 (OSRAM) |
| SI (2) | SI3018211T1 (OSRAM) |
| SM (2) | SMT201900559T1 (OSRAM) |
| TW (1) | TWI541024B (OSRAM) |
| WO (1) | WO2012029986A1 (OSRAM) |
Families Citing this family (73)
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| PL1766010T3 (pl) | 2004-06-28 | 2011-07-29 | Univ Western Australia | Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| 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 |
| SMT201900159T1 (it) | 2008-10-24 | 2019-05-10 | Sarepta Therapeutics Inc | Composizioni di salto di esone per dmd |
| KR20230137491A (ko) | 2009-11-12 | 2023-10-04 | 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 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| BR112014018427B1 (pt) * | 2012-01-27 | 2021-11-03 | Biomarin Technologies B.V. | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker |
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| NZ631245A (en) * | 2013-03-14 | 2017-09-29 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| HK1220154A1 (zh) | 2013-03-15 | 2017-04-28 | Sarepta Therapeutics, Inc. | 改进的用於治疗肌营养不良的组合物 |
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| US10174319B2 (en) | 2014-05-19 | 2019-01-08 | Knc Laboratories Co., Ltd. | Nucleic acid drug for inducing skipping of variant exon of CD44 gene and increasing expression of normal type CD44 mRNA |
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| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| CN108026531B (zh) * | 2015-09-15 | 2021-09-14 | 日本新药株式会社 | 反义核酸 |
| JP6875684B2 (ja) * | 2015-09-16 | 2021-05-26 | 日本新薬株式会社 | 筋萎縮症治療用アンチセンス核酸 |
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| EP3359668A4 (en) | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
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| DK3464306T3 (da) | 2016-05-24 | 2024-05-21 | Sarepta Therapeutics Inc | Fremgangsmåder til fremstilling af phosphorodiamidat-morpholino-oligomerer |
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| KR102824513B1 (ko) * | 2016-07-05 | 2025-06-25 | 바이오마린 테크놀로지스 비.브이. | 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드 |
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