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- Withdrawn
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- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 150000003839 salts Chemical class 0.000 claims 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 0 *CC(CN(**)C1)=NC1N Chemical compound *CC(CN(**)C1)=NC1N 0.000 description 3
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 description 2
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 description 2
- 229940113082 thymine Drugs 0.000 description 1
- 229940035893 uracil Drugs 0.000 description 1
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| JP2016503219A Withdrawn JP2016521119A (ja) | 2013-03-14 | 2014-03-14 | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |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) | US20140315977A1 (enExample) |
| EP (3) | EP3495485A3 (enExample) |
| JP (4) | JP2016521119A (enExample) |
| KR (5) | KR20240094032A (enExample) |
| CN (2) | CN105392884B (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 |
|---|---|---|---|---|
| DK2206781T3 (en) | 2004-06-28 | 2016-03-07 | Univ Western Australia | The antisense oligonucleotides in inducing exon-skipping and methods of use thereof |
| ES2914775T3 (es) * | 2007-10-26 | 2022-06-16 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarresta trastornos del músculo |
| IL304042A (en) | 2008-10-24 | 2023-08-01 | Sarepta Therapeutics Inc | Antisense compounds for inducing axon termination and preparations containing them for the treatment of muscular dystrophy |
| IL297299A (en) | 2009-11-12 | 2022-12-01 | Univ Western Australia | Antisense oligonucleotides for inducing exon skipping in the dystrophin gene |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| KR20250047401A (ko) | 2013-03-14 | 2025-04-03 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | 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 |
| CA3024182A1 (en) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| US10875880B2 (en) * | 2016-05-24 | 2020-12-29 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| MA45362A (fr) * | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | 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 |
| CN109152775B (zh) * | 2016-06-30 | 2022-04-26 | 萨勒普塔医疗公司 | 制备磷酸二酰胺吗啉代寡聚物的方法 |
| CA3024153A1 (en) * | 2016-06-30 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| WO2018005805A1 (en) * | 2016-06-30 | 2018-01-04 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| EP4252845A3 (en) * | 2016-07-05 | 2024-05-22 | BioMarin Technologies B.V. | Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
| EP3485015A4 (en) * | 2016-07-15 | 2020-07-29 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS OF MODULATION OF DYSTROPHINE TRANSCRIPT |
| EP3515445A4 (en) | 2016-09-20 | 2020-05-27 | The Regents of The University of Colorado, A Body Corporate | SYNTHESIS OF BACKBONE-MODIFIED MORPHOLINO-OLIGONUCLEOTIDES AND CHIMERES WITH THE AID OF PHOSPHORAMIDITE CHEMISTRY |
| ES3051834T3 (en) * | 2016-12-19 | 2025-12-30 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| PT3554553T (pt) | 2016-12-19 | 2022-08-04 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
| CN117298290A (zh) | 2016-12-19 | 2023-12-29 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| MX2020002038A (es) * | 2017-08-31 | 2020-09-18 | 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 |
| JP7218361B2 (ja) * | 2017-09-25 | 2023-02-06 | サレプタ セラピューティクス， インコーポレイテッド | 高速流動合成を介してホスホロジアミデートモルホリノオリゴマーを調製するための方法 |
| US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| EP3765030A4 (en) * | 2018-03-16 | 2022-01-26 | Sarepta Therapeutics, Inc. | CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION |
| EP3784248A4 (en) * | 2018-04-26 | 2022-08-10 | Sarepta Therapeutics, Inc. | EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY |
| 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 |
| NZ770540A (en) * | 2018-06-26 | 2025-12-19 | Nippon Shinyaku Co Ltd | Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| JP2021533200A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用 |
| 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 |
| GB201812972D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| GB201812980D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| BR112021010982A2 (pt) | 2018-12-07 | 2021-08-31 | Oxford University Innovation Limited | Ligantes |
| EP3954395A4 (en) * | 2019-04-08 | 2023-06-28 | National University Corporation Tokyo Medical and Dental University | Pharmaceutical composition for muscle disease treatment |
| WO2021025899A1 (en) | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| CA3153902A1 (en) | 2019-10-16 | 2021-04-22 | Pardis SABETI | Engineered muscle targeting compositions |
| WO2022006871A1 (en) * | 2020-07-10 | 2022-01-13 | Changzhou Syntheall Pharmaceuticals Co., Ltd. | Processes for preparing oligonucleotides |
| WO2022171972A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| WO2022213118A1 (en) | 2021-03-31 | 2022-10-06 | 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 |
| EP4337264A1 (en) | 2021-05-10 | 2024-03-20 | Entrada Therapeutics, Inc. | Compositions and methods for modulating tissue distribution of intracellular therapeutics |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| MX2024000489A (es) | 2021-07-09 | 2024-04-09 | Dyne Therapeutics Inc | Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías. |
| 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 |
| US20250066396A1 (en) | 2021-12-27 | 2025-02-27 | Nippon Shinyaku Co., Ltd. | Method for producing oligonucleic acid compound |
| EP4493693A1 (en) * | 2022-03-17 | 2025-01-22 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
| WO2024073588A1 (en) * | 2022-09-28 | 2024-04-04 | Board Of Regents, The University Of Texas System | Respiratory dry powder delivery |
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| CN107353317A (zh) | 2010-05-28 | 2017-11-17 | 萨勒普塔医疗公司 | 具有修饰的亚基间键和/或端基的寡核苷酸类似物 |
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-
2014
- 2014-03-14 KR KR1020247018517A patent/KR20240094032A/ko not_active Ceased
- 2014-03-14 BR BR112015023038A patent/BR112015023038A2/pt not_active Application Discontinuation
- 2014-03-14 CN CN201480027371.7A patent/CN105392884B/zh active Active
- 2014-03-14 EA EA201591694A patent/EA201591694A1/ru unknown
- 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 WO PCT/US2014/029766 patent/WO2014153240A2/en not_active Ceased
- 2014-03-14 SI SI201431107T patent/SI2970964T1/sl unknown
- 2014-03-14 KR KR1020217000648A patent/KR20210006516A/ko not_active Ceased
- 2014-03-14 ES ES14723583T patent/ES2714290T3/es active Active
- 2014-03-14 PT PT14723583T patent/PT2970964T/pt unknown
- 2014-03-14 KR KR1020227014526A patent/KR20220061280A/ko not_active Ceased
- 2014-03-14 KR KR1020157028188A patent/KR20150133752A/ko not_active Ceased
- 2014-03-14 KR KR1020237016315A patent/KR20230074606A/ko not_active Ceased
- 2014-03-14 CN CN201811579820.5A patent/CN110066793A/zh active Pending
- 2014-03-14 EP EP14723583.2A patent/EP2970964B8/en active Active
- 2014-03-14 TR TR2019/03009T patent/TR201903009T4/tr unknown
- 2014-03-14 PL PL14723583T patent/PL2970964T3/pl unknown
- 2014-03-14 HR HRP20190382TT patent/HRP20190382T1/hr unknown
- 2014-03-14 EP EP20182238.4A patent/EP3760720A1/en not_active Withdrawn
- 2014-03-14 US US14/213,641 patent/US20140315977A1/en not_active Abandoned
- 2014-03-14 HU HUE14723583A patent/HUE042218T2/hu unknown
- 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 DK DK14723583.2T patent/DK2970964T3/en active
- 2014-03-14 JP JP2016503219A patent/JP2016521119A/ja not_active Withdrawn
- 2014-03-14 MX MX2015012149A patent/MX366274B/es active IP Right Grant
- 2014-03-14 AU AU2014236160A patent/AU2014236160A1/en not_active Abandoned
- 2014-03-14 NZ NZ631132A patent/NZ631132A/en unknown
- 2014-03-14 SM SM20190139T patent/SMT201900139T1/it unknown
- 2014-03-14 RS RS20190277A patent/RS58529B1/sr unknown
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2015
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- 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
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- 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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2021
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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
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