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.)
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- 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 |
| 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 |
| JP2021008202A Active JP6867636B1 (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 |
| 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 |
| JP2021008202A Active JP6867636B1 (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) | EP3543341A1 (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) | HUE046364T2 (enExample) |
| LT (1) | LT3018211T (enExample) |
| PL (2) | PL2612917T3 (enExample) |
| PT (1) | PT3018211T (enExample) |
| RS (2) | RS54649B1 (enExample) |
| SI (2) | SI3018211T1 (enExample) |
| SM (2) | SMT201900559T1 (enExample) |
| TW (1) | TWI541024B (enExample) |
| WO (1) | WO2012029986A1 (enExample) |
Families Citing this family (75)
| Publication number | Priority date | Publication date | Assignee | Title |
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| SI2206781T1 (sl) | 2004-06-28 | 2016-05-31 | The University Of Western Australia | Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| ES2852549T3 (es) | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| DK2203173T3 (en) | 2007-10-26 | 2016-02-29 | Academisch Ziekenhuis Leiden | Materials and methods for prevention of muscle diseases |
| 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 |
| 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 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| ES2907250T3 (es) | 2012-01-27 | 2022-04-22 | Biomarin Tech Bv | Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker |
| KR20150099804A (ko) * | 2012-12-20 | 2015-09-01 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물 |
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| KR20230074606A (ko) * | 2013-03-14 | 2023-05-30 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| NZ732507A (en) | 2013-03-15 | 2018-08-31 | Sarepta Therapeutics Inc | Improved compositions for treating muscular dystrophy |
| RU2702424C2 (ru) * | 2014-03-12 | 2019-10-08 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
| 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 |
| WO2015194520A1 (ja) * | 2014-06-17 | 2015-12-23 | 日本新薬株式会社 | アンチセンス核酸 |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| CN113913426B (zh) * | 2015-09-15 | 2024-08-02 | 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 |
| WO2017062835A2 (en) | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| 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 |
| 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 |
| 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 |
| SG10202101834QA (en) | 2016-05-24 | 2021-04-29 | 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 |
| 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 |
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| 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 |
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