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- Withdrawn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 (https=) | 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| JP2016503219A Withdrawn JP2016521119A (ja) | 2013-03-14 | 2014-03-14 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2019212294A Withdrawn JP2020039362A (ja) | 2013-03-14 | 2019-11-25 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2021204872A Withdrawn JP2022031948A (ja) | 2013-03-14 | 2021-12-17 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2024083541A Withdrawn JP2024112929A (ja) | 2013-03-14 | 2024-05-22 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |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| JP2019212294A Withdrawn JP2020039362A (ja) | 2013-03-14 | 2019-11-25 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2021204872A Withdrawn JP2022031948A (ja) | 2013-03-14 | 2021-12-17 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
| JP2024083541A Withdrawn JP2024112929A (ja) | 2013-03-14 | 2024-05-22 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |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) | US20140315977A1 (https=) |
| EP (3) | EP3495485A3 (https=) |
| JP (4) | JP2016521119A (https=) |
| KR (5) | KR20240094032A (https=) |
| CN (2) | CN110066793A (https=) |
| AU (3) | AU2014236160A1 (https=) |
| BR (1) | BR112015023038A2 (https=) |
| CA (1) | CA2903872A1 (https=) |
| CY (1) | CY1121876T1 (https=) |
| DK (1) | DK2970964T3 (https=) |
| EA (1) | EA201591694A1 (https=) |
| ES (1) | ES2714290T3 (https=) |
| HR (1) | HRP20190382T1 (https=) |
| HU (1) | HUE042218T2 (https=) |
| IL (2) | IL241047B (https=) |
| LT (1) | LT2970964T (https=) |
| MX (2) | MX366274B (https=) |
| NZ (1) | NZ631132A (https=) |
| PL (1) | PL2970964T3 (https=) |
| PT (1) | PT2970964T (https=) |
| RS (1) | RS58529B1 (https=) |
| SA (1) | SA515361121B1 (https=) |
| SI (1) | SI2970964T1 (https=) |
| SM (1) | SMT201900139T1 (https=) |
| TR (1) | TR201903009T4 (https=) |
| WO (1) | WO2014153240A2 (https=) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| ES2639852T3 (es) * | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| CN110218727A (zh) | 2013-03-14 | 2019-09-10 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的外显子跳跃组合物 |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | 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 |
| PH12018501207B1 (en) | 2015-12-21 | 2024-02-23 | Novartis Ag | Compositions and methods for decreasing tau expression |
| BR112018074346B1 (pt) * | 2016-05-24 | 2023-01-03 | Sarepta Therapeutics, Inc. | Composto oligomérico e processo para preparar um composto oligomérico |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MD3464306T2 (ro) * | 2016-05-24 | 2024-08-31 | Sarepta Therapeutics Inc | Procedee de preparare a oligomerilor morfolino fosforodiamidați |
| US10947533B2 (en) * | 2016-05-24 | 2021-03-16 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| CA3024456A1 (en) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| KR102523527B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| KR102523522B1 (ko) * | 2016-06-30 | 2023-04-20 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 포스포로디아미데이트 모르폴리노 올리고머의 제조 방법 |
| JP2019525742A (ja) | 2016-06-30 | 2019-09-12 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマー |
| MX2018016253A (es) * | 2016-07-05 | 2019-09-09 | Biomarin Tech Bv | Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos. |
| US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
| AU2017330276B2 (en) * | 2016-09-20 | 2022-01-20 | The Regents Of The University Of Colorado, A Body Corporate | Synthesis of backbone modified morpholino oligonucleotides and chimeras using phosphoramidite chemistry |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR20240006023A (ko) | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| PT3554552T (pt) * | 2016-12-19 | 2022-10-03 | Sarepta Therapeutics Inc | Conjugados de oligómero de skipping de exão para distrofia muscular |
| US20200131231A1 (en) | 2017-02-17 | 2020-04-30 | Oxford University Innovation Limited | Cell penetrating peptides |
| EP3675836A4 (en) * | 2017-08-31 | 2021-05-26 | Sarepta Therapeutics, Inc. | METHODS FOR TREATING MUSCLE DYSTROPHY |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| WO2019060862A1 (en) | 2017-09-25 | 2019-03-28 | Sarepta Therapeutics, Inc. | METHODS FOR PREPARING MORPHOLINO PHOSPHORODIAMIDATE OLIGOMERS VIA FAST FLOW SYNTHESIS |
| WO2019067979A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| EP3765030A4 (en) * | 2018-03-16 | 2022-01-26 | Sarepta Therapeutics, Inc. | CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION |
| JP2021521794A (ja) * | 2018-04-26 | 2021-08-30 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| JP7579147B2 (ja) * | 2018-06-14 | 2024-11-07 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| 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 |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| 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 |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | 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 |
| 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 |
| US12465646B2 (en) | 2018-12-07 | 2025-11-11 | Oxford University Innovation Limited | Linkers |
| CN113677374A (zh) * | 2019-04-08 | 2021-11-19 | 国立大学法人东京医科齿科大学 | 肌疾病治疗用药物组合物 |
| 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 |
| CN116096724B (zh) * | 2020-07-10 | 2025-10-31 | 常州合全药业有限公司 | 用于制备寡核苷酸的方法 |
| WO2022171972A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| EP4314016A1 (en) | 2021-03-31 | 2024-02-07 | Entrada Therapeutics, Inc. | Cyclic cell penetrating 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 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| 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 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP4458833A4 (en) | 2021-12-27 | 2025-12-31 | Nippon Shinyaku Co Ltd | PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS |
| CN119013401A (zh) * | 2022-03-17 | 2024-11-22 | 萨勒普塔医疗公司 | 二氨基磷酸酯吗啉代寡聚物缀合物 |
| 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 US US14/213,641 patent/US20140315977A1/en not_active Abandoned
- 2014-03-14 EA EA201591694A patent/EA201591694A1/ru unknown
- 2014-03-14 HU HUE14723583A patent/HUE042218T2/hu unknown
- 2014-03-14 PT PT14723583T patent/PT2970964T/pt unknown
- 2014-03-14 AU AU2014236160A patent/AU2014236160A1/en not_active Abandoned
- 2014-03-14 KR KR1020247018517A patent/KR20240094032A/ko not_active Ceased
- 2014-03-14 HR HRP20190382TT patent/HRP20190382T1/hr unknown
- 2014-03-14 LT LTEP14723583.2T patent/LT2970964T/lt unknown
- 2014-03-14 MX MX2015012149A patent/MX366274B/es active IP Right Grant
- 2014-03-14 KR KR1020237016315A patent/KR20230074606A/ko not_active Ceased
- 2014-03-14 EP EP18215621.6A patent/EP3495485A3/en not_active Withdrawn
- 2014-03-14 CA CA2903872A patent/CA2903872A1/en active Pending
- 2014-03-14 JP JP2016503219A patent/JP2016521119A/ja not_active Withdrawn
- 2014-03-14 DK DK14723583.2T patent/DK2970964T3/en active
- 2014-03-14 CN CN201811579820.5A patent/CN110066793A/zh active Pending
- 2014-03-14 EP EP20182238.4A patent/EP3760720A1/en not_active Withdrawn
- 2014-03-14 PL PL14723583T patent/PL2970964T3/pl unknown
- 2014-03-14 SI SI201431107T patent/SI2970964T1/sl unknown
- 2014-03-14 BR BR112015023038A patent/BR112015023038A2/pt not_active Application Discontinuation
- 2014-03-14 EP EP14723583.2A patent/EP2970964B8/en active Active
- 2014-03-14 SM SM20190139T patent/SMT201900139T1/it unknown
- 2014-03-14 KR KR1020227014526A patent/KR20220061280A/ko not_active Ceased
- 2014-03-14 WO PCT/US2014/029766 patent/WO2014153240A2/en not_active Ceased
- 2014-03-14 RS RS20190277A patent/RS58529B1/sr unknown
- 2014-03-14 ES ES14723583T patent/ES2714290T3/es active Active
- 2014-03-14 CN CN201480027371.7A patent/CN105392884B/zh active Active
- 2014-03-14 TR TR2019/03009T patent/TR201903009T4/tr unknown
- 2014-03-14 KR KR1020157028188A patent/KR20150133752A/ko not_active Ceased
- 2014-03-14 US US14/776,533 patent/US20160040162A1/en not_active Abandoned
- 2014-03-14 KR KR1020217000648A patent/KR20210006516A/ko not_active Ceased
- 2014-03-14 NZ NZ631132A patent/NZ631132A/en 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
- 2017-01-27 US US15/417,401 patent/US20180002689A1/en not_active Abandoned
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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
- 2020-02-02 IL IL272415A patent/IL272415A/en unknown
- 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
- 2022-10-13 AU AU2022252769A patent/AU2022252769A1/en not_active Abandoned
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2024
- 2024-05-22 JP JP2024083541A patent/JP2024112929A/ja not_active Withdrawn
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