JP2019062913A5 - - Google Patents
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- JP2019062913A5 JP2019062913A5 JP2019001091A JP2019001091A JP2019062913A5 JP 2019062913 A5 JP2019062913 A5 JP 2019062913A5 JP 2019001091 A JP2019001091 A JP 2019001091A JP 2019001091 A JP2019001091 A JP 2019001091A JP 2019062913 A5 JP2019062913 A5 JP 2019062913A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligomer
- pmo
- oligomer
- base
- mrna
- 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 21
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 8
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 8
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 8
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims 8
- 108020004999 messenger RNA Proteins 0.000 claims 8
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 8
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims 4
- 229930024421 Adenine Natural products 0.000 claims 4
- 229960000643 adenine Drugs 0.000 claims 4
- 230000000295 complement effect Effects 0.000 claims 4
- 229940104302 cytosine Drugs 0.000 claims 4
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 4
- 239000000178 monomer Substances 0.000 claims 4
- 229940113082 thymine Drugs 0.000 claims 4
- 229940035893 uracil Drugs 0.000 claims 4
- 230000004962 physiological condition Effects 0.000 claims 2
- 239000002243 precursor Substances 0.000 claims 2
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 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 |
|---|---|---|---|
| JP2017154320A Division JP6465932B2 (ja) | 2010-09-01 | 2017-08-09 | アンチセンス核酸 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020003338A Division JP6867619B2 (ja) | 2010-09-01 | 2020-01-14 | アンチセンス核酸 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019062913A JP2019062913A (ja) | 2019-04-25 |
| JP2019062913A5 true JP2019062913A5 (enExample) | 2020-01-23 |
| JP6647430B2 JP6647430B2 (ja) | 2020-02-14 |
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 |
| JP2021008201A Active JP6867621B1 (ja) | 2010-09-01 | 2021-01-21 | 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 |
| 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 (4)
| 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 |
Family Applications After (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020003338A Active JP6867619B2 (ja) | 2010-09-01 | 2020-01-14 | 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 |
| 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 |
| 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 (enExample) |
| EP (6) | EP4403632A3 (enExample) |
| JP (12) | JP5363655B2 (enExample) |
| KR (1) | KR101310569B1 (enExample) |
| CN (1) | CN103154245B (enExample) |
| AU (1) | AU2011296882B2 (enExample) |
| CA (1) | CA2809637C (enExample) |
| CY (2) | CY1117367T1 (enExample) |
| DK (2) | DK2612917T3 (enExample) |
| ES (2) | ES2750748T3 (enExample) |
| HR (2) | HRP20160336T1 (enExample) |
| HU (2) | HUE027321T2 (enExample) |
| LT (1) | LT3018211T (enExample) |
| PL (2) | PL3018211T3 (enExample) |
| PT (1) | PT3018211T (enExample) |
| RS (2) | RS59361B1 (enExample) |
| SI (2) | SI3018211T1 (enExample) |
| SM (2) | SMT201900559T1 (enExample) |
| TW (1) | TWI541024B (enExample) |
| WO (1) | WO2012029986A1 (enExample) |
Families Citing this family (73)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE498685T1 (de) * | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| 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 |
| TR201902952T4 (tr) | 2008-10-24 | 2019-03-21 | Sarepta Therapeutics Inc | Dmd için ekson atlama bileşimleri. |
| CA2780563C (en) | 2009-11-12 | 2025-05-06 | Univ Western Australia | Antisense Molecules and Methods for Treating Pathologies |
| 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 |
| 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 |
| WO2014100714A1 (en) * | 2012-12-20 | 2014-06-26 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| NZ775701A (en) * | 2013-03-14 | 2022-08-26 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| HUE042218T2 (hu) * | 2013-03-14 | 2019-06-28 | Sarepta Therapeutics Inc | Exonátugrást elõsegítõ készítmények izomdisztrófia kezelésére |
| NZ732507A (en) | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
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| JP6536911B2 (ja) * | 2014-05-19 | 2019-07-03 | 神戸天然物化学株式会社 | ＣＤ４４遺伝子のバリアントエクソンのスキッピングを誘導し、正常型ＣＤ４４ｍＲＮＡの発現を増加させる核酸医薬 |
| EP3159409B1 (en) | 2014-06-17 | 2019-12-04 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid for use in the treatment of duchenne's muscular dystrophy |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| SMT202000379T1 (it) * | 2015-09-15 | 2020-09-10 | Nippon Shinyaku Co Ltd | Acido nucleico antisenso |
| EP3351634A4 (en) * | 2015-09-16 | 2019-05-08 | Nippon Shinyaku Co., Ltd. | ANTISENSE NUCLEIC ACID FOR THE TREATMENT OF AMYOTROPHY |
| MA45819A (fr) | 2015-10-09 | 2018-08-15 | Sarepta Therapeutics Inc | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods 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 |
| KR101686379B1 (ko) | 2016-02-05 | 2016-12-13 | 박정열 | 휴대용 구이기 |
| EP3464305B1 (en) * | 2016-05-24 | 2024-08-21 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| IL263044B2 (en) | 2016-05-24 | 2024-06-01 | 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 |
| HRP20240583T1 (hr) | 2016-05-24 | 2024-07-19 | Sarepta Therapeutics, Inc. | Postupci za pripremu fosforodiamidat morfolino oligomera |
| AU2017270598B2 (en) * | 2016-05-24 | 2022-12-01 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| KR20190024977A (ko) | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| KR20230175312A (ko) * | 2016-07-05 | 2023-12-29 | 바이오마린 테크놀로지스 비.브이. | 유전적 장애 치료를 위한, 특징이 개선된, 비사이클릭 스캐폴드 모이어티를 포함하는 프리-mrna 스플라이스 스위칭 또는 조정 올리고뉴클레오티드 |
| KR101715572B1 (ko) | 2016-10-11 | 2017-03-22 | 박종찬 | 구이대 승강구조를 갖는 양방향 구이기 |
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| US20200254002A1 (en) | 2017-09-28 | 2020-08-13 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| WO2019209764A2 (en) * | 2018-04-26 | 2019-10-31 | Sarepta Therapeutics, Inc. | Exon skipping oligomers and oligomer conjugates for muscular dystrophy |
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| US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| MX2020013880A (es) * | 2018-06-26 | 2021-03-09 | Nippon Shinyaku Co Ltd | Composición que comprende el oligonucleótido antisentido y su uso para el tratamiento de la distrofia muscular de duchenne. |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | 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 |
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| JP2025079347A (ja) * | 2022-03-11 | 2025-05-22 | 日本新薬株式会社 | キャリアペプチドが連結された核酸 |
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