PT2607484E - Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne - Google Patents
Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne Download PDFInfo
- Publication number
- PT2607484E PT2607484E PT131603383T PT13160338T PT2607484E PT 2607484 E PT2607484 E PT 2607484E PT 131603383 T PT131603383 T PT 131603383T PT 13160338 T PT13160338 T PT 13160338T PT 2607484 E PT2607484 E PT 2607484E
- Authority
- PT
- Portugal
- Prior art keywords
- exon
- mrna
- methods
- muscular dystrophy
- duchenne muscular
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/NL2008/050673 WO2009054725A2 (en) | 2007-10-26 | 2008-10-27 | Means and methods for counteracting muscle disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT2607484E true PT2607484E (pt) | 2016-03-09 |
Family
ID=40873329
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT131603383T PT2607484E (pt) | 2008-10-27 | 2009-01-13 | Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne |
| PT09788160T PT2344637E (pt) | 2008-10-27 | 2009-01-13 | Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT09788160T PT2344637E (pt) | 2008-10-27 | 2009-01-13 | Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne |
Country Status (15)
| Country | Link |
|---|---|
| EP (4) | EP2607484B1 (pt) |
| JP (2) | JP5786109B2 (pt) |
| AU (1) | AU2009310557B2 (pt) |
| CA (3) | CA2741629C (pt) |
| CY (1) | CY1116305T1 (pt) |
| DK (2) | DK2607484T3 (pt) |
| ES (2) | ES2562658T3 (pt) |
| HK (1) | HK1160169A1 (pt) |
| HR (1) | HRP20160078T1 (pt) |
| IL (2) | IL212508A (pt) |
| NZ (1) | NZ592498A (pt) |
| PL (1) | PL2607484T3 (pt) |
| PT (2) | PT2607484E (pt) |
| SI (1) | SI2607484T1 (pt) |
| WO (2) | WO2010050801A1 (pt) |
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI1766010T1 (sl) | 2004-06-28 | 2011-06-30 | Univ Western Australia | Protismiselni oligonukleotidi za induciranje preskakovanja eksonov in postopki za njihovo uporabo |
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| 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 |
| EP3133160B1 (en) | 2008-10-24 | 2018-12-12 | Sarepta Therapeutics, Inc. | Exon skipping compositions for dmd |
| EP2480659A2 (en) * | 2009-09-24 | 2012-08-01 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
| 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 |
| JP2014507143A (ja) * | 2011-02-08 | 2014-03-27 | ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング／ビジネス／アズ キャロライナズ ヘルスケア システム | アンチセンスオリゴヌクレオチド |
| CN108588073A (zh) | 2011-12-28 | 2018-09-28 | 日本新药株式会社 | 反义核酸 |
| AU2013212758A1 (en) * | 2012-01-27 | 2014-08-14 | Biomarin Technologies B.V. | RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy |
| WO2014007620A2 (en) * | 2012-07-03 | 2014-01-09 | Prosensa Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| NZ775701A (en) | 2013-03-14 | 2022-08-26 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| EP2968991A2 (en) | 2013-03-15 | 2016-01-20 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| AU2014317961B2 (en) * | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| JP6519842B2 (ja) * | 2013-10-04 | 2019-05-29 | 国立大学法人神戸大学 | 福山型筋ジストロフィー治療用アンチセンス核酸 |
| TR201901939T4 (tr) | 2014-03-12 | 2019-03-21 | Nat Center Neurology & Psychiatry | Antisens nükleik asit. |
| CN106661580B (zh) | 2014-06-10 | 2022-02-15 | 鹿特丹伊拉斯谟大学医疗中心 | 用于治疗庞帕病的反义寡核苷酸 |
| LT3351633T (lt) | 2015-09-15 | 2020-08-10 | Nippon Shinyaku Co., Ltd. | Priešprasmė nukleorūgštis |
| IL258230B (en) | 2015-10-09 | 2022-09-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods thereof |
| US11339392B2 (en) | 2016-03-02 | 2022-05-24 | The Board Of Trustees Of The Leland Stanford Junior University | Pan-genotypic agents against influenza virus and methods of using the same |
| US10597658B2 (en) * | 2016-03-02 | 2020-03-24 | The Board Of Trustees Of The Leland Stanford Junior University | Pan-genotypic agents against influenza virus and methods of using the same |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| CN109414511B (zh) | 2016-04-18 | 2023-05-23 | 萨勒普塔医疗公司 | 用于治疗与酸性α-葡糖苷酶基因相关的疾病的反义寡聚物及其使用方法 |
| MA45290A (fr) * | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
| KR20190024977A (ko) | 2016-06-30 | 2019-03-08 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 |
| JP2019522972A (ja) * | 2016-07-05 | 2019-08-22 | バイオマリン テクノロジーズ ベー．フェー． | 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド |
| IL297528A (en) | 2016-12-19 | 2022-12-01 | Sarepta Therapeutics Inc | Exon-skipping oligomer conjugates for muscular dystrophy |
| BR112019012651A2 (pt) | 2016-12-19 | 2020-01-28 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
| WO2018118599A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP3565577A4 (en) | 2017-01-06 | 2020-10-07 | Avidity Biosciences, Inc. | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCTION OF EXON SKIP |
| 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 |
| EP3687577A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| MA50753A (fr) | 2017-10-04 | 2020-08-12 | Avidity Biosciences Inc | Compositions d'acide nucléique-polypeptide et utilisations de celles-ci |
| KR102443358B1 (ko) | 2017-12-06 | 2022-09-14 | 어비디티 바이오사이언시스 인크. | 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법 |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| JP2021526796A (ja) * | 2018-06-13 | 2021-10-11 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマー |
| CN112955153A (zh) | 2018-08-02 | 2021-06-11 | 达因疗法公司 | 肌肉靶向复合物及其用于治疗肌养蛋白病的用途 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| EP3830259A4 (en) | 2018-08-02 | 2022-05-04 | Dyne Therapeutics, Inc. | MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY |
| CN113383078A (zh) * | 2018-12-06 | 2021-09-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| CN114375296A (zh) | 2019-06-06 | 2022-04-19 | 艾维迪提生物科学公司 | 核酸多肽组合物及其用途 |
| TW202136511A (zh) * | 2019-12-19 | 2021-10-01 | 日商日本新藥股份有限公司 | 能進行外顯子跳讀之反義核酸 |
| WO2021132591A1 (ja) | 2019-12-26 | 2021-07-01 | 日本新薬株式会社 | エクソン５０のスキッピングを誘導するアンチセンス核酸 |
| CA3173049A1 (en) | 2020-02-28 | 2021-09-02 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid inducing skipping of exon 51 |
| KR20220156867A (ko) | 2020-03-19 | 2022-11-28 | 어비디티 바이오사이언시스 인크. | 안면견갑상완 근이영양증의 치료용 조성물 및 방법 |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2386239A1 (en) | 1999-10-04 | 2001-04-12 | University Of Medicine And Dentistry Of New Jersey | Methods for identifying rna binding compounds |
| JP4836366B2 (ja) * | 2000-08-25 | 2011-12-14 | 雅文 松尾 | デュシェンヌ型筋ジストロフィー治療剤 |
| US6727355B2 (en) * | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| AU2003225410A1 (en) * | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| SI1766010T1 (sl) * | 2004-06-28 | 2011-06-30 | Univ Western Australia | Protismiselni oligonukleotidi za induciranje preskakovanja eksonov in postopki za njihovo uporabo |
| NZ563206A (en) * | 2005-04-22 | 2009-09-25 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mRNA by interfering with the binding or SR proteins and by interfering with secondary RNA structure |
| EP1857548A1 (en) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| EP3133160B1 (en) * | 2008-10-24 | 2018-12-12 | Sarepta Therapeutics, Inc. | Exon skipping compositions for dmd |
| US9607308B2 (en) | 2011-06-29 | 2017-03-28 | American Express Travel Related Services Company, Inc. | Spend based digital ad targeting and measurement |
-
2009
- 2009-01-13 WO PCT/NL2009/050006 patent/WO2010050801A1/en active Application Filing
- 2009-01-13 PL PL13160338T patent/PL2607484T3/pl unknown
- 2009-01-13 PT PT131603383T patent/PT2607484E/pt unknown
- 2009-01-13 ES ES13160338.3T patent/ES2562658T3/es active Active
- 2009-01-13 EP EP13160338.3A patent/EP2607484B1/en not_active Revoked
- 2009-01-13 JP JP2011533126A patent/JP5786109B2/ja active Active
- 2009-01-13 ES ES09788160.1T patent/ES2532634T5/es active Active
- 2009-01-13 DK DK13160338.3T patent/DK2607484T3/en active
- 2009-01-13 PT PT09788160T patent/PT2344637E/pt unknown
- 2009-01-13 SI SI200931381A patent/SI2607484T1/sl unknown
- 2009-01-13 CA CA2741629A patent/CA2741629C/en active Active
- 2009-01-13 AU AU2009310557A patent/AU2009310557B2/en active Active
- 2009-01-13 EP EP09788160.1A patent/EP2344637B2/en active Active
- 2009-01-13 DK DK09788160.1T patent/DK2344637T4/en active
- 2009-03-11 WO PCT/NL2009/050113 patent/WO2010050802A2/en active Application Filing
- 2009-03-11 EP EP18180952.6A patent/EP3400948B1/en active Active
- 2009-03-11 EP EP09788170.0A patent/EP2349287B1/en active Active
- 2009-03-11 JP JP2011533127A patent/JP5905260B2/ja active Active
- 2009-03-11 CA CA3017539A patent/CA3017539A1/en active Pending
- 2009-03-11 CA CA2741793A patent/CA2741793C/en active Active
- 2009-03-11 NZ NZ592498A patent/NZ592498A/en unknown
-
2011
- 2011-04-27 IL IL212508A patent/IL212508A/en active IP Right Grant
- 2011-04-27 IL IL212509A patent/IL212509A/en active IP Right Grant
-
2012
- 2012-01-16 HK HK12100495.6A patent/HK1160169A1/xx active IP Right Maintenance
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2015
- 2015-03-04 CY CY20151100228T patent/CY1116305T1/el unknown
-
2016
- 2016-01-25 HR HRP20160078TT patent/HRP20160078T1/hr unknown
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