JP2012506703A5 - - Google Patents
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- JP2012506703A5 JP2012506703A5 JP2011533392A JP2011533392A JP2012506703A5 JP 2012506703 A5 JP2012506703 A5 JP 2012506703A5 JP 2011533392 A JP2011533392 A JP 2011533392A JP 2011533392 A JP2011533392 A JP 2011533392A JP 2012506703 A5 JP2012506703 A5 JP 2012506703A5
- Authority
- JP
- Japan
- Prior art keywords
- exon
- antisense compound
- compound
- seq
- nos
- 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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- 150000001875 compounds Chemical class 0.000 claims description 120
- 239000000203 mixture Substances 0.000 claims description 71
- 108020004999 messenger RNA Proteins 0.000 claims description 63
- 108091027305 Heteroduplex Proteins 0.000 claims description 50
- 230000000692 anti-sense effect Effects 0.000 claims description 46
- 230000000295 complement effect Effects 0.000 claims description 25
- 108010069091 Dystrophin Proteins 0.000 claims description 24
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 16
- MHCMWGPPLOSCJY-UHFFFAOYSA-N 4-$l^{1}-azanylmorpholine Chemical compound [N]N1CCOCC1 MHCMWGPPLOSCJY-UHFFFAOYSA-N 0.000 claims description 15
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical compound [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims description 15
- OAICVXFJPJFONN-UHFFFAOYSA-N Phosphorus Chemical compound [P] OAICVXFJPJFONN-UHFFFAOYSA-N 0.000 claims description 15
- 229910052799 carbon Inorganic materials 0.000 claims description 15
- 229910052698 phosphorus Inorganic materials 0.000 claims description 15
- 239000011574 phosphorus Substances 0.000 claims description 15
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims description 13
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 8
- 201000006938 muscular dystrophy Diseases 0.000 claims description 5
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims description 4
- 239000002773 nucleotide Substances 0.000 claims 12
- 125000003729 nucleotide group Chemical group 0.000 claims 12
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 2
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims 2
- 125000002091 cationic group Chemical group 0.000 claims 1
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 229940113082 thymine Drugs 0.000 claims 1
- 229940035893 uracil Drugs 0.000 claims 1
- 238000000034 method Methods 0.000 description 20
- 239000004475 Arginine Substances 0.000 description 14
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 14
- 108090000765 processed proteins & peptides Proteins 0.000 description 14
- 108700024394 Exon Proteins 0.000 description 1
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 108091034117 Oligonucleotide Proteins 0.000 description 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 1
- 238000012230 antisense oligonucleotides Methods 0.000 description 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 description 1
- 235000018417 cysteine Nutrition 0.000 description 1
- 208000016361 genetic disease Diseases 0.000 description 1
- 108090000623 proteins and genes Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US10841608P | 2008-10-24 | 2008-10-24 | |
| US61/108,416 | 2008-10-24 | ||
| PCT/US2009/061960 WO2010048586A1 (en) | 2008-10-24 | 2009-10-23 | Multiple exon skipping compositions for dmd |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014214204A Division JP2015017131A (ja) | 2008-10-24 | 2014-10-21 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012506703A JP2012506703A (ja) | 2012-03-22 |
| JP2012506703A5 true JP2012506703A5 (https=) | 2012-12-13 |
| JP5864257B2 JP5864257B2 (ja) | 2016-02-17 |
Family
ID=41510869
Family Applications (11)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011533392A Active JP5864257B2 (ja) | 2008-10-24 | 2009-10-23 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2014214204A Withdrawn JP2015017131A (ja) | 2008-10-24 | 2014-10-21 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2016167575A Active JP6429285B2 (ja) | 2008-10-24 | 2016-08-30 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2017028390A Withdrawn JP2017086087A (ja) | 2008-10-24 | 2017-02-17 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2018084126A Active JP6406782B2 (ja) | 2008-10-24 | 2018-04-25 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2019121399A Withdrawn JP2019178163A (ja) | 2008-10-24 | 2019-06-28 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021031641A Withdrawn JP2021088595A (ja) | 2008-10-24 | 2021-03-01 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021066507A Pending JP2021100439A (ja) | 2008-10-24 | 2021-04-09 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023019132A Withdrawn JP2023055956A (ja) | 2008-10-24 | 2023-02-10 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023055590A Withdrawn JP2023073441A (ja) | 2008-10-24 | 2023-03-30 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2025284201A Pending JP2026042839A (ja) | 2008-10-24 | 2025-12-26 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
Family Applications After (10)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014214204A Withdrawn JP2015017131A (ja) | 2008-10-24 | 2014-10-21 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2016167575A Active JP6429285B2 (ja) | 2008-10-24 | 2016-08-30 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2017028390A Withdrawn JP2017086087A (ja) | 2008-10-24 | 2017-02-17 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2018084126A Active JP6406782B2 (ja) | 2008-10-24 | 2018-04-25 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2019121399A Withdrawn JP2019178163A (ja) | 2008-10-24 | 2019-06-28 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021031641A Withdrawn JP2021088595A (ja) | 2008-10-24 | 2021-03-01 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2021066507A Pending JP2021100439A (ja) | 2008-10-24 | 2021-04-09 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023019132A Withdrawn JP2023055956A (ja) | 2008-10-24 | 2023-02-10 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2023055590A Withdrawn JP2023073441A (ja) | 2008-10-24 | 2023-03-30 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
| JP2025284201A Pending JP2026042839A (ja) | 2008-10-24 | 2025-12-26 | Ｄｍｄのための複数のエキソンスキッピング組成物 |
Country Status (22)
| Country | Link |
|---|---|
| US (15) | US8871918B2 (https=) |
| EP (7) | EP4174178A1 (https=) |
| JP (11) | JP5864257B2 (https=) |
| KR (6) | KR20250016146A (https=) |
| CN (4) | CN112574988A (https=) |
| AU (1) | AU2009308167B2 (https=) |
| BR (3) | BRPI0920276B1 (https=) |
| CA (2) | CA3066050A1 (https=) |
| CY (1) | CY1122369T1 (https=) |
| DK (1) | DK3133160T3 (https=) |
| ES (2) | ES2955539T3 (https=) |
| HR (1) | HRP20190387T1 (https=) |
| HU (1) | HUE042979T2 (https=) |
| IL (6) | IL313162A (https=) |
| LT (1) | LT3133160T (https=) |
| PL (1) | PL3133160T3 (https=) |
| PT (1) | PT3133160T (https=) |
| SI (1) | SI3133160T1 (https=) |
| SM (1) | SMT201900159T1 (https=) |
| TR (1) | TR201902952T4 (https=) |
| TW (7) | TWI689587B (https=) |
| WO (1) | WO2010048586A1 (https=) |
Families Citing this family (148)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2524255C (en) * | 2003-03-21 | 2014-02-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| CA2523672C (en) | 2003-04-29 | 2012-07-17 | Avi Biopharma, Inc. | Compositions for enhancing transport of molecules into cells |
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| US20050288246A1 (en) | 2004-05-24 | 2005-12-29 | Iversen Patrick L | Peptide conjugated, inosine-substituted antisense oligomer compound and method |
| 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 |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| EP3443976A1 (en) * | 2007-06-29 | 2019-02-20 | Sarepta Therapeutics, Inc. | Tissue specific peptide conjugates and methods |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| EP2235034B1 (en) | 2007-12-28 | 2016-11-23 | Sarepta Therapeutics, Inc. | Immunomodulatory agents and methods of use |
| 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 |
| CN112574988A (zh) | 2008-10-24 | 2021-03-30 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| JP5786109B2 (ja) * | 2008-10-27 | 2015-09-30 | プロセンサ テクノロジーズ ベー．フェー． | Ｄｕｃｈｅｎｎｅ型筋ジストロフィーｍＲＮＡ前駆体のエクソン４５の効率的なスキッピングのための方法及び手段 |
| CA2759899A1 (en) * | 2009-04-24 | 2010-10-28 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| US20110269665A1 (en) | 2009-06-26 | 2011-11-03 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| LT2499249T (lt) * | 2009-11-12 | 2018-12-27 | The University Of Western Australia | Priešprasmės molekulės ir patologijų gydymo būdai |
| KR20200133284A (ko) | 2010-05-28 | 2020-11-26 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
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| US20140080896A1 (en) * | 2011-08-30 | 2014-03-20 | The Regents Of The University Of California | Identification of small molecules that facilitate therapeutic exon skipping |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EA202090338A1 (ru) | 2011-11-18 | 2021-04-30 | Сарепта Терапьютикс, Инк. | Функционально-модифицированные олигонуклеотиды и их субъединицы |
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| CN117721110A (zh) * | 2011-12-28 | 2024-03-19 | 日本新药株式会社 | 反义核酸 |
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| EP3597741A1 (en) | 2012-04-27 | 2020-01-22 | Duke University | Genetic correction of mutated genes |
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| ES2818073T3 (es) | 2013-12-24 | 2021-04-09 | Sentiss Pharma Private Ltd | Solución oftálmica de tartrato de brimonidina tópica |
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| BR112016027236A2 (pt) | 2014-05-23 | 2017-10-17 | Genzyme Corp | porções de múltiplos oligonucleotídeos em veículo de peptídeo |
| RU2695430C2 (ru) * | 2014-06-17 | 2019-07-23 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
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