JP2014054250A5 - - Google Patents
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- JP2014054250A5 JP2014054250A5 JP2013184193A JP2013184193A JP2014054250A5 JP 2014054250 A5 JP2014054250 A5 JP 2014054250A5 JP 2013184193 A JP2013184193 A JP 2013184193A JP 2013184193 A JP2013184193 A JP 2013184193A JP 2014054250 A5 JP2014054250 A5 JP 2014054250A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligomer
- group
- oligomer according
- bond
- antisense
- 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 11
- 108091034117 Oligonucleotide Proteins 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 2
- 108010058514 Phosphate-Binding Proteins Proteins 0.000 claims 2
- 102000006335 Phosphate-Binding Proteins Human genes 0.000 claims 2
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims 1
- 239000004480 active ingredient Substances 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000002947 alkylene group Chemical group 0.000 claims 1
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- UXTZGKSGGBHJOE-UHFFFAOYSA-N CN(C)P(N(C)CC(N)=O)(OC)=O Chemical compound CN(C)P(N(C)CC(N)=O)(OC)=O UXTZGKSGGBHJOE-UHFFFAOYSA-N 0.000 description 1
- UCLQCQZSVIFYKB-UHFFFAOYSA-N CN(C)P(N(CC1)CCN1C(OCCOCCOCCO)=O)(OC)=O Chemical compound CN(C)P(N(CC1)CCN1C(OCCOCCOCCO)=O)(OC)=O UCLQCQZSVIFYKB-UHFFFAOYSA-N 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2013184193A JP6141728B2 (ja) | 2010-09-01 | 2013-09-05 | アンチセンス核酸 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2010196032 | 2010-09-01 | ||
| JP2010196032 | 2010-09-01 | ||
| JP2013184193A JP6141728B2 (ja) | 2010-09-01 | 2013-09-05 | アンチセンス核酸 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012531987A Division JP5363655B2 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015256962A Division JP6193343B2 (ja) | 2010-09-01 | 2015-12-28 | アンチセンス核酸 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014054250A JP2014054250A (ja) | 2014-03-27 |
| JP2014054250A5 true JP2014054250A5 (https=) | 2014-06-05 |
| JP6141728B2 JP6141728B2 (ja) | 2017-06-07 |
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 |
| JP2021008202A Active JP6867636B1 (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 |
| 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 (1)
| 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 |
Family Applications After (10)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
| JP2021008202A Active JP6867636B1 (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 |
| 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 (https=) |
| EP (6) | EP4400168A3 (https=) |
| JP (12) | JP5363655B2 (https=) |
| KR (1) | KR101310569B1 (https=) |
| CN (1) | CN103154245B (https=) |
| AU (1) | AU2011296882B2 (https=) |
| CA (1) | CA2809637C (https=) |
| CY (2) | CY1117367T1 (https=) |
| DK (2) | DK3018211T3 (https=) |
| ES (2) | ES2567411T3 (https=) |
| HR (2) | HRP20160336T1 (https=) |
| HU (2) | HUE027321T2 (https=) |
| LT (1) | LT3018211T (https=) |
| PL (2) | PL2612917T3 (https=) |
| PT (1) | PT3018211T (https=) |
| RS (2) | RS59361B1 (https=) |
| SI (2) | SI3018211T1 (https=) |
| SM (2) | SMT201900559T1 (https=) |
| TW (1) | TWI541024B (https=) |
| WO (1) | WO2012029986A1 (https=) |
Families Citing this family (76)
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| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| ES2639852T3 (es) * | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| 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 |
| 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. |
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| 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 |
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| MD3464306T2 (ro) | 2016-05-24 | 2024-08-31 | Sarepta Therapeutics Inc | Procedee de preparare a oligomerilor morfolino fosforodiamidați |
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| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
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| EP3675836A4 (en) * | 2017-08-31 | 2021-05-26 | Sarepta Therapeutics, Inc. | METHODS FOR TREATING MUSCLE DYSTROPHY |
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