JP2016521119A5 - - Google Patents
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- JP2016521119A5 JP2016521119A5 JP2016503219A JP2016503219A JP2016521119A5 JP 2016521119 A5 JP2016521119 A5 JP 2016521119A5 JP 2016503219 A JP2016503219 A JP 2016503219A JP 2016503219 A JP2016503219 A JP 2016503219A JP 2016521119 A5 JP2016521119 A5 JP 2016521119A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutically acceptable
- stereochemistry
- centered
- phosphorus
- pharmaceutical 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.)
- Withdrawn
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- 0 *CC(CN(**)C1)=NC1N Chemical compound *CC(CN(**)C1)=NC1N 0.000 description 3
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361782706P | 2013-03-14 | 2013-03-14 | |
| US61/782,706 | 2013-03-14 | ||
| PCT/US2014/029766 WO2014153240A2 (en) | 2013-03-14 | 2014-03-14 | Exon skipping compositions for treating muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019212294A Division JP2020039362A (ja) | 2013-03-14 | 2019-11-25 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016521119A JP2016521119A (ja) | 2016-07-21 |
| JP2016521119A5 true JP2016521119A5 (enExample) | 2018-08-09 |
Family
ID=50694008
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016503219A Withdrawn JP2016521119A (ja) | 2013-03-14 | 2014-03-14 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2019212294A Withdrawn JP2020039362A (ja) | 2013-03-14 | 2019-11-25 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2021204872A Withdrawn JP2022031948A (ja) | 2013-03-14 | 2021-12-17 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2024083541A Withdrawn JP2024112929A (ja) | 2013-03-14 | 2024-05-22 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019212294A Withdrawn JP2020039362A (ja) | 2013-03-14 | 2019-11-25 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2021204872A Withdrawn JP2022031948A (ja) | 2013-03-14 | 2021-12-17 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2024083541A Withdrawn JP2024112929A (ja) | 2013-03-14 | 2024-05-22 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
Country Status (26)
| Country | Link |
|---|---|
| US (5) | US20160040162A1 (enExample) |
| EP (3) | EP3760720A1 (enExample) |
| JP (4) | JP2016521119A (enExample) |
| KR (5) | KR20230074606A (enExample) |
| CN (2) | CN110066793A (enExample) |
| AU (3) | AU2014236160A1 (enExample) |
| BR (1) | BR112015023038A2 (enExample) |
| CA (1) | CA2903872A1 (enExample) |
| CY (1) | CY1121876T1 (enExample) |
| DK (1) | DK2970964T3 (enExample) |
| EA (1) | EA201591694A1 (enExample) |
| ES (1) | ES2714290T3 (enExample) |
| HR (1) | HRP20190382T1 (enExample) |
| HU (1) | HUE042218T2 (enExample) |
| IL (2) | IL241047B (enExample) |
| LT (1) | LT2970964T (enExample) |
| MX (2) | MX366274B (enExample) |
| NZ (1) | NZ631132A (enExample) |
| PL (1) | PL2970964T3 (enExample) |
| PT (1) | PT2970964T (enExample) |
| RS (1) | RS58529B1 (enExample) |
| SA (1) | SA515361121B1 (enExample) |
| SI (1) | SI2970964T1 (enExample) |
| SM (1) | SMT201900139T1 (enExample) |
| TR (1) | TR201903009T4 (enExample) |
| WO (1) | WO2014153240A2 (enExample) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI2206781T1 (sl) | 2004-06-28 | 2016-05-31 | The University Of Western Australia | Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe |
| DK2203173T3 (en) * | 2007-10-26 | 2016-02-29 | Academisch Ziekenhuis Leiden | Materials and methods for prevention of muscle diseases |
| EP2762567B1 (en) | 2008-10-24 | 2016-07-13 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for DMD |
| SMT201800579T1 (it) | 2009-11-12 | 2019-01-11 | Univ Western Australia | Molecole antisenso e metodi per trattare patologie |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| AU2014236140B2 (en) | 2013-03-14 | 2019-10-03 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| NZ732507A (en) | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| JOP20200228A1 (ar) | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| MX383656B (es) * | 2016-05-24 | 2025-03-14 | Sarepta Therapeutics Inc | Procesos para preparar oligómeros de morfolino fosforodiamidato. |
| SG11201809497RA (en) * | 2016-05-24 | 2018-12-28 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| SG10202101834QA (en) * | 2016-05-24 | 2021-04-29 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| CN109563114B (zh) * | 2016-05-24 | 2022-08-12 | 萨勒普塔医疗公司 | 用于制备寡聚物的方法 |
| JP7022079B2 (ja) * | 2016-06-30 | 2022-02-17 | サレプタ セラピューティクス， インコーポレイテッド | ホスホロジアミダートモルホリノオリゴマーを調製するためのプロセス |
| US20190262375A1 (en) * | 2016-06-30 | 2019-08-29 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| CN109757108A (zh) * | 2016-07-05 | 2019-05-14 | 比奥马林技术公司 | 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸 |
| EP3485015A4 (en) * | 2016-07-15 | 2020-07-29 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS OF MODULATION OF DYSTROPHINE TRANSCRIPT |
| CN109937042B (zh) | 2016-09-20 | 2023-06-27 | 科罗拉多州立大学董事会法人团体 | 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体 |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR102646318B1 (ko) | 2016-12-19 | 2024-03-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| HRP20240705T1 (hr) | 2016-12-19 | 2024-08-30 | Sarepta Therapeutics, Inc. | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
| AR112538A1 (es) * | 2017-08-31 | 2019-11-06 | Sarepta Therapeutics Inc | Métodos para el tratamiento de distrofia muscular |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| TWI812647B (zh) * | 2017-09-25 | 2023-08-21 | 美商薩羅塔治療公司 | 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程 |
| EP3687519A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| WO2019178479A1 (en) | 2018-03-16 | 2019-09-19 | Sarepta Therapeutics, Inc. | Chimeric peptides for antisense delivery |
| EP3784248A4 (en) * | 2018-04-26 | 2022-08-10 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY |
| US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| WO2019241470A2 (en) * | 2018-06-14 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
| CN112399849A (zh) * | 2018-06-26 | 2021-02-23 | 日本新药株式会社 | 含有反义寡核苷酸的组合物及其在迪谢内肌营养不良症的治疗中的用途 |
| 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 |
| AU2019316103B2 (en) | 2018-08-02 | 2026-02-19 | 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 |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| GB201812980D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| GB201812972D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| EP3890783A1 (en) | 2018-12-07 | 2021-10-13 | Oxford University Innovation Limited | Linkers |
| WO2020209285A1 (ja) * | 2019-04-08 | 2020-10-15 | 国立大学法人東京医科歯科大学 | 筋疾患治療用医薬組成物 |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| WO2022006871A1 (en) * | 2020-07-10 | 2022-01-13 | Changzhou Syntheall Pharmaceuticals Co., Ltd. | Processes for preparing oligonucleotides |
| CA3211038A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| IL307298A (en) | 2021-03-31 | 2023-11-01 | Entrada Therapeutics Inc | Cell-penetrating circular peptides |
| WO2022217366A1 (en) * | 2021-04-14 | 2022-10-20 | The Royal Institution For The Advancement Of Learning/Mcgill University | Modified oligonucleotides for the treatment of duchenne muscular dystrophy |
| AU2022271873A1 (en) | 2021-05-10 | 2024-01-04 | Entrada Therapeutics, Inc. | Compositions and methods for intracellular therapeutics |
| JP2024525608A (ja) | 2021-07-09 | 2024-07-12 | 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製剤 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| AU2022424485A1 (en) | 2021-12-27 | 2024-07-11 | Nippon Shinyaku Co., Ltd. | Method for producing oligonucleic acid compound |
| 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 |
| WO2024073588A1 (en) * | 2022-09-28 | 2024-04-04 | Board Of Regents, The University Of Texas System | Respiratory dry powder delivery |
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2014
- 2014-03-14 KR KR1020237016315A patent/KR20230074606A/ko not_active Ceased
- 2014-03-14 ES ES14723583T patent/ES2714290T3/es active Active
- 2014-03-14 AU AU2014236160A patent/AU2014236160A1/en not_active Abandoned
- 2014-03-14 CN CN201811579820.5A patent/CN110066793A/zh active Pending
- 2014-03-14 DK DK14723583.2T patent/DK2970964T3/en active
- 2014-03-14 KR KR1020227014526A patent/KR20220061280A/ko not_active Ceased
- 2014-03-14 PL PL14723583T patent/PL2970964T3/pl unknown
- 2014-03-14 RS RS20190277A patent/RS58529B1/sr unknown
- 2014-03-14 KR KR1020217000648A patent/KR20210006516A/ko not_active Ceased
- 2014-03-14 MX MX2015012149A patent/MX366274B/es active IP Right Grant
- 2014-03-14 CA CA2903872A patent/CA2903872A1/en active Pending
- 2014-03-14 EP EP20182238.4A patent/EP3760720A1/en not_active Withdrawn
- 2014-03-14 HR HRP20190382TT patent/HRP20190382T1/hr unknown
- 2014-03-14 HU HUE14723583A patent/HUE042218T2/hu unknown
- 2014-03-14 CN CN201480027371.7A patent/CN105392884B/zh active Active
- 2014-03-14 KR KR1020157028188A patent/KR20150133752A/ko not_active Ceased
- 2014-03-14 KR KR1020247018517A patent/KR20240094032A/ko not_active Ceased
- 2014-03-14 EP EP18215621.6A patent/EP3495485A3/en not_active Withdrawn
- 2014-03-14 SM SM20190139T patent/SMT201900139T1/it unknown
- 2014-03-14 EP EP14723583.2A patent/EP2970964B8/en active Active
- 2014-03-14 US US14/776,533 patent/US20160040162A1/en not_active Abandoned
- 2014-03-14 LT LTEP14723583.2T patent/LT2970964T/lt unknown
- 2014-03-14 BR BR112015023038A patent/BR112015023038A2/pt not_active Application Discontinuation
- 2014-03-14 EA EA201591694A patent/EA201591694A1/ru unknown
- 2014-03-14 WO PCT/US2014/029766 patent/WO2014153240A2/en not_active Ceased
- 2014-03-14 US US14/213,641 patent/US20140315977A1/en not_active Abandoned
- 2014-03-14 JP JP2016503219A patent/JP2016521119A/ja not_active Withdrawn
- 2014-03-14 NZ NZ631132A patent/NZ631132A/en unknown
- 2014-03-14 PT PT14723583T patent/PT2970964T/pt unknown
- 2014-03-14 TR TR2019/03009T patent/TR201903009T4/tr unknown
- 2014-03-14 SI SI201431107T patent/SI2970964T1/sl unknown
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2015
- 2015-09-02 IL IL241047A patent/IL241047B/en active IP Right Grant
- 2015-09-10 MX MX2019000224A patent/MX2019000224A/es unknown
- 2015-09-14 SA SA515361121A patent/SA515361121B1/ar unknown
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2017
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2019
- 2019-03-05 CY CY20191100267T patent/CY1121876T1/el unknown
- 2019-03-19 US US16/358,169 patent/US20200040336A1/en not_active Abandoned
- 2019-11-25 JP JP2019212294A patent/JP2020039362A/ja not_active Withdrawn
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2020
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- 2020-05-27 AU AU2020203473A patent/AU2020203473A1/en not_active Abandoned
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2021
- 2021-12-17 JP JP2021204872A patent/JP2022031948A/ja not_active Withdrawn
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2022
- 2022-06-03 US US17/805,402 patent/US20220364093A1/en not_active Abandoned
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2024
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