JP2016502858A5 - - Google Patents
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- Publication number
- JP2016502858A5 JP2016502858A5 JP2015549818A JP2015549818A JP2016502858A5 JP 2016502858 A5 JP2016502858 A5 JP 2016502858A5 JP 2015549818 A JP2015549818 A JP 2015549818A JP 2015549818 A JP2015549818 A JP 2015549818A JP 2016502858 A5 JP2016502858 A5 JP 2016502858A5
- Authority
- JP
- Japan
- Prior art keywords
- antisense oligonucleotide
- base
- oligonucleotide
- antisense
- target region
- 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- Withdrawn
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261739968P | 2012-12-20 | 2012-12-20 | |
| US61/739,968 | 2012-12-20 | ||
| PCT/US2013/077216 WO2014100714A1 (en) | 2012-12-20 | 2013-12-20 | Improved exon skipping compositions for treating muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018081272A Division JP2018110601A (ja) | 2012-12-20 | 2018-04-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016502858A JP2016502858A (ja) | 2016-02-01 |
| JP2016502858A5 true JP2016502858A5 (enExample) | 2017-02-02 |
Family
ID=49950080
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015549818A Withdrawn JP2016502858A (ja) | 2012-12-20 | 2013-12-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
| JP2018081272A Pending JP2018110601A (ja) | 2012-12-20 | 2018-04-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
| JP2019003308A Pending JP2019050834A (ja) | 2012-12-20 | 2019-01-11 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018081272A Pending JP2018110601A (ja) | 2012-12-20 | 2018-04-20 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
| JP2019003308A Pending JP2019050834A (ja) | 2012-12-20 | 2019-01-11 | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
Country Status (13)
| Country | Link |
|---|---|
| US (5) | US20150361428A1 (enExample) |
| EP (2) | EP2935584A1 (enExample) |
| JP (3) | JP2016502858A (enExample) |
| KR (2) | KR20200143739A (enExample) |
| CN (2) | CN111440796A (enExample) |
| AU (3) | AU2013364158A1 (enExample) |
| BR (1) | BR112015014987A2 (enExample) |
| CA (1) | CA2894899A1 (enExample) |
| EA (1) | EA201591178A1 (enExample) |
| HK (1) | HK1216902A1 (enExample) |
| IL (1) | IL239491A0 (enExample) |
| MX (2) | MX2015008035A (enExample) |
| WO (1) | WO2014100714A1 (enExample) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL1766010T3 (pl) | 2004-06-28 | 2011-07-29 | Univ Western Australia | Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania |
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| CN105779453A (zh) | 2008-10-24 | 2016-07-20 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| BR122023023194A2 (pt) | 2009-11-12 | 2023-12-26 | The University Of Western Australia | Oligonucleotídeo antissentido, composição e respectivo uso |
| 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 |
| HRP20190382T1 (hr) * | 2013-03-14 | 2019-04-19 | Sarepta Therapeutics, Inc. | Pripravci koji preskaču ekson za liječenje mišićne distrofije |
| EA202090946A3 (ru) | 2013-03-14 | 2021-07-30 | Сарепта Терапьютикс, Инк. | Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии |
| NZ631289A (en) * | 2013-03-15 | 2017-08-25 | Sarepta Therapeutics Inc | Improved compositions for treating 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 |
| PT3283500T (pt) | 2015-04-08 | 2021-01-28 | Univ Chicago | Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão |
| WO2017062835A2 (en) | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
| EP4678748A2 (en) | 2015-12-21 | 2026-01-14 | Novartis AG | Compositions and methods for decreasing tau expression |
| WO2017136435A1 (en) | 2016-02-01 | 2017-08-10 | The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health | Compounds for modulating fc-epsilon-ri-beta expression and uses thereof |
| KR102775461B1 (ko) | 2016-04-01 | 2025-02-28 | 어비디티 바이오사이언시스 인크. | 핵산-폴리펩타이드 조성물 및 이의 용도 |
| AU2017290231A1 (en) | 2016-06-30 | 2019-02-07 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| US11655470B2 (en) | 2016-07-05 | 2023-05-23 | The Usa, As Represented By The Secretary, Dept. Of Health And Human Services | Diagnosing COL6-related disorders and methods for treating same |
| WO2018052549A1 (en) | 2016-09-19 | 2018-03-22 | The Usa, As Represented By The Secretary, Dept. Of Health And Human Services | Zika virus vaccines |
| 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 |
| EP3554552B1 (en) | 2016-12-19 | 2022-08-17 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| LT3554553T (lt) | 2016-12-19 | 2022-08-25 | Sarepta Therapeutics, Inc. | Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos |
| CN110381980A (zh) | 2017-01-06 | 2019-10-25 | 艾维迪提生物科学有限责任公司 | 核酸-多肽组合物以及诱导外显子跳读的方法 |
| WO2018152526A1 (en) | 2017-02-20 | 2018-08-23 | The Usa, As Represented By The Secretary, Dept. Of Health And Human Services | Zika virus vaccines |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| 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 |
| IL321667A (en) * | 2018-12-13 | 2025-08-01 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| CN118516348B (zh) * | 2024-07-23 | 2025-01-17 | 深川生物科技(山东)股份公司 | 单链核酸或双链核酸及在制备治疗杜氏肌营养不良的药物中的应用 |
Family Cites Families (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CH445129A (fr) | 1964-04-29 | 1967-10-15 | Nestle Sa | Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé |
| US3459731A (en) | 1966-12-16 | 1969-08-05 | Corn Products Co | Cyclodextrin polyethers and their production |
| US3426011A (en) | 1967-02-13 | 1969-02-04 | Corn Products Co | Cyclodextrins with anionic properties |
| US3453257A (en) | 1967-02-13 | 1969-07-01 | Corn Products Co | Cyclodextrin with cationic properties |
| US3453259A (en) | 1967-03-22 | 1969-07-01 | Corn Products Co | Cyclodextrin polyol ethers and their oxidation products |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US4458066A (en) | 1980-02-29 | 1984-07-03 | University Patents, Inc. | Process for preparing polynucleotides |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| WO1986005519A1 (en) | 1985-03-15 | 1986-09-25 | James Summerton | Polynucleotide assay reagent and method |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
| KR0166088B1 (ko) | 1990-01-23 | 1999-01-15 | . | 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도 |
| US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
| EP1251170A3 (en) | 1992-07-17 | 2002-10-30 | Ribozyme Pharmaceuticals, Inc. | Method and reagent for treatment of NF-kappaB dependent animal diseases |
| EP1897942A1 (en) | 1993-05-11 | 2008-03-12 | The University Of North Carolina At Chapel Hill | Antisense oligonucleotides which combat aberrant splicing and methods of using the same |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| IL115849A0 (en) | 1994-11-03 | 1996-01-31 | Merz & Co Gmbh & Co | Tangential filtration preparation of liposomal drugs and liposome product thereof |
| WO1999042091A2 (en) | 1998-02-19 | 1999-08-26 | Massachusetts Institute Of Technology | Use of polycations as endosomolytic agents |
| US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
| US6210892B1 (en) | 1998-10-07 | 2001-04-03 | Isis Pharmaceuticals, Inc. | Alteration of cellular behavior by antisense modulation of mRNA processing |
| AU2281201A (en) | 1999-12-29 | 2001-07-09 | A. James Mixson | Histidine copolymer and methods for using same |
| US7070807B2 (en) | 1999-12-29 | 2006-07-04 | Mixson A James | Branched histidine copolymers and methods for using same |
| CA2448120A1 (en) | 2001-05-24 | 2002-11-28 | Genzyme Corporation | Muscle-specific expression vectors |
| PL1766010T3 (pl) | 2004-06-28 | 2011-07-29 | Univ Western Australia | Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania |
| US20070105807A1 (en) | 2005-11-10 | 2007-05-10 | Sazani Peter L | Splice switch oligomers for TNF superfamily receptors and their use in treatment of disease |
| ES2645410T3 (es) | 2006-05-10 | 2017-12-05 | Sarepta Therapeutics, Inc. | Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| WO2009127230A1 (en) | 2008-04-16 | 2009-10-22 | Curevac Gmbh | MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION |
| US8084601B2 (en) * | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| CN105779453A (zh) * | 2008-10-24 | 2016-07-20 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| BR122023023194A2 (pt) | 2009-11-12 | 2023-12-26 | The University Of Western Australia | Oligonucleotídeo antissentido, composição e respectivo uso |
| KR102182663B1 (ko) | 2010-05-28 | 2020-11-25 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| IL273838B (en) | 2011-05-05 | 2022-09-01 | Sarepta Therapeutics Inc | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
-
2013
- 2013-12-20 JP JP2015549818A patent/JP2016502858A/ja not_active Withdrawn
- 2013-12-20 KR KR1020207033842A patent/KR20200143739A/ko not_active Ceased
- 2013-12-20 EA EA201591178A patent/EA201591178A1/ru unknown
- 2013-12-20 BR BR112015014987A patent/BR112015014987A2/pt not_active Application Discontinuation
- 2013-12-20 CN CN202010276936.2A patent/CN111440796A/zh active Pending
- 2013-12-20 EP EP13819166.3A patent/EP2935584A1/en not_active Withdrawn
- 2013-12-20 MX MX2015008035A patent/MX2015008035A/es unknown
- 2013-12-20 CA CA2894899A patent/CA2894899A1/en not_active Abandoned
- 2013-12-20 CN CN201380073536.XA patent/CN106414739A/zh active Pending
- 2013-12-20 WO PCT/US2013/077216 patent/WO2014100714A1/en not_active Ceased
- 2013-12-20 HK HK16104638.2A patent/HK1216902A1/zh unknown
- 2013-12-20 EP EP21152285.9A patent/EP3885439A1/en not_active Withdrawn
- 2013-12-20 KR KR1020157019523A patent/KR20150099804A/ko not_active Ceased
- 2013-12-20 AU AU2013364158A patent/AU2013364158A1/en not_active Abandoned
-
2015
- 2015-06-18 US US14/743,856 patent/US20150361428A1/en not_active Abandoned
- 2015-06-18 IL IL239491A patent/IL239491A0/en unknown
- 2015-06-19 MX MX2019008164A patent/MX2019008164A/es unknown
-
2017
- 2017-01-31 US US15/420,823 patent/US20170369875A1/en not_active Abandoned
-
2018
- 2018-04-20 JP JP2018081272A patent/JP2018110601A/ja active Pending
-
2019
- 2019-01-11 JP JP2019003308A patent/JP2019050834A/ja active Pending
- 2019-10-21 AU AU2019253778A patent/AU2019253778A1/en not_active Abandoned
- 2019-12-04 US US16/703,110 patent/US20200339985A1/en not_active Abandoned
-
2021
- 2021-10-11 US US17/498,115 patent/US20220259592A1/en not_active Abandoned
-
2022
- 2022-03-09 AU AU2022201628A patent/AU2022201628A1/en not_active Abandoned
- 2022-05-11 US US17/741,758 patent/US20230104708A1/en not_active Abandoned
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