HK1160169A1 - Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna (dmd) mrna 45 - Google Patents
Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna (dmd) mrna 45Info
- Publication number
- HK1160169A1 HK1160169A1 HK12100495.6A HK12100495A HK1160169A1 HK 1160169 A1 HK1160169 A1 HK 1160169A1 HK 12100495 A HK12100495 A HK 12100495A HK 1160169 A1 HK1160169 A1 HK 1160169A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- mrna
- dmd
- exon
- methods
- muscular dystrophy
- 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 (2)
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 |
PCT/NL2009/050006 WO2010050801A1 (en) | 2008-10-27 | 2009-01-13 | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna |
Publications (1)
Publication Number | Publication Date |
---|---|
HK1160169A1 true HK1160169A1 (en) | 2012-08-10 |
Family
ID=40873329
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HK12100495.6A HK1160169A1 (en) | 2008-10-27 | 2012-01-16 | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna (dmd) mrna 45 |
Country Status (15)
Country | Link |
---|---|
EP (4) | EP2344637B2 (xx) |
JP (2) | JP5786109B2 (xx) |
AU (1) | AU2009310557B2 (xx) |
CA (3) | CA2741629C (xx) |
CY (1) | CY1116305T1 (xx) |
DK (2) | DK2344637T4 (xx) |
ES (2) | ES2562658T3 (xx) |
HK (1) | HK1160169A1 (xx) |
HR (1) | HRP20160078T1 (xx) |
IL (2) | IL212509A (xx) |
NZ (1) | NZ592498A (xx) |
PL (1) | PL2607484T3 (xx) |
PT (2) | PT2344637E (xx) |
SI (1) | SI2607484T1 (xx) |
WO (2) | WO2010050801A1 (xx) |
Families Citing this family (49)
Publication number | Priority date | Publication date | Assignee | Title |
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US7807816B2 (en) | 2004-06-28 | 2010-10-05 | 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 |
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 |
KR20230025924A (ko) * | 2008-10-24 | 2023-02-23 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Dmd를 위한 다중 엑손 스키핑 조성물 |
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 |
EP2499249B1 (en) | 2009-11-12 | 2018-08-08 | The University Of Western Australia | Antisense molecules and methods for treating pathologies |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
CN103501793A (zh) * | 2011-02-08 | 2014-01-08 | 夏洛特-梅克伦堡医院(商业用名:卡罗来纳保健系统) | 反义寡核苷酸 |
CN110055243B (zh) | 2011-12-28 | 2024-03-26 | 日本新药株式会社 | 反义核酸 |
CN104203289B (zh) * | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
CN104603271B (zh) * | 2012-07-03 | 2019-07-19 | 马林生物科技有限公司 | 用于治疗肌肉萎缩症患者的寡核苷酸 |
JP6449231B2 (ja) | 2013-03-14 | 2019-01-09 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
EA201591792A1 (ru) | 2013-03-15 | 2016-02-29 | Сарепта Терапьютикс, Инк. | Улучшенные композиции для лечения мышечной дистрофии |
EP3041935A1 (en) * | 2013-09-05 | 2016-07-13 | Sage Therapeutics, Inc. | 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 |
US9988629B2 (en) * | 2014-03-12 | 2018-06-05 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids |
AU2015272128B2 (en) | 2014-06-10 | 2021-10-28 | Erasmus University Medical Center Rotterdam | Antisense oligonucleotides useful in treatment of Pompe disease |
MY185390A (en) * | 2015-09-15 | 2021-05-17 | Nippon Shinyaku Co Ltd | Antisense nucleic acids |
TW201722439A (zh) | 2015-10-09 | 2017-07-01 | 波濤生命科學有限公司 | 寡核苷酸組合物及其方法 |
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 |
CA3014776A1 (en) * | 2016-03-02 | 2017-09-08 | 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 |
KR102522059B1 (ko) | 2016-04-18 | 2023-04-14 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 안티센스 올리고머, 및 산성 알파-글루코시다제 유전자와 연관된 질환을 치료하기 위한 이의 사용 방법 |
MA45290A (fr) * | 2016-05-04 | 2019-03-13 | Wave Life Sciences Ltd | Procédés et compositions d'agents biologiquement actifs |
AU2017290231A1 (en) | 2016-06-30 | 2019-02-07 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
EP4252845A2 (en) * | 2016-07-05 | 2023-10-04 | BioMarin Technologies B.V. | Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
CN110636866A (zh) | 2016-12-19 | 2019-12-31 | 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 |
HUE060065T2 (hu) | 2016-12-19 | 2023-01-28 | Sarepta Therapeutics Inc | Exonátugró oligomerkonjugátumok izomdisztrófiára |
BR112019012647A2 (pt) | 2016-12-19 | 2019-11-19 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
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 |
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 |
US20210145852A1 (en) | 2017-09-28 | 2021-05-20 | Sarepta Therapeutics, Inc. | Combination Therapies for Treating Muscular Dystrophy |
US11110180B2 (en) | 2017-10-04 | 2021-09-07 | Avidity Biosciences Inc. | Nucleic acid-polypeptide compositions and uses thereof |
SG11202005022YA (en) | 2017-12-06 | 2020-06-29 | Avidity Biosciences Inc | Compositions and methods of treating muscle atrophy and myotonic dystrophy |
US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
WO2019241385A2 (en) * | 2018-06-13 | 2019-12-19 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystropy |
CA3108289A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
JP2021532831A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用 |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
WO2020118246A1 (en) * | 2018-12-06 | 2020-06-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
EP3980436A4 (en) | 2019-06-06 | 2023-12-20 | Avidity Biosciences, Inc. | NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF |
KR20220118459A (ko) * | 2019-12-19 | 2022-08-25 | 니뽄 신야쿠 가부시키가이샤 | 엑손 스키핑을 가능하게 하는 안티센스 핵산 |
BR112022012622A2 (pt) | 2019-12-26 | 2022-09-06 | Nippon Shinyaku Co Ltd | Ácido nucleico antissenso que induz o salto do éxon 50 |
WO2021172498A1 (ja) | 2020-02-28 | 2021-09-02 | 日本新薬株式会社 | エクソン51のスキッピングを誘導するアンチセンス核酸 |
CN115666589A (zh) | 2020-03-19 | 2023-01-31 | 艾维迪提生物科学公司 | 治疗面肩肱型肌营养不良的组合物和方法 |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
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Publication number | Priority date | Publication date | Assignee | Title |
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JP2003511362A (ja) | 1999-10-04 | 2003-03-25 | ユニバーシティー オブ メディシン アンド デンティストリー オブ ニュー ジャージー | 新規カルバメートおよび尿素類 |
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 |
WO2004083432A1 (en) * | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
US7807816B2 (en) * | 2004-06-28 | 2010-10-05 | University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
CA2605512A1 (en) * | 2005-04-22 | 2006-10-26 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the binding of 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 |
KR20230025924A (ko) * | 2008-10-24 | 2023-02-23 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | Dmd를 위한 다중 엑손 스키핑 조성물 |
US9607308B2 (en) | 2011-06-29 | 2017-03-28 | American Express Travel Related Services Company, Inc. | Spend based digital ad targeting and measurement |
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2009
- 2009-01-13 DK DK09788160.1T patent/DK2344637T4/en active
- 2009-01-13 EP EP09788160.1A patent/EP2344637B2/en active Active
- 2009-01-13 SI SI200931381A patent/SI2607484T1/sl unknown
- 2009-01-13 WO PCT/NL2009/050006 patent/WO2010050801A1/en active Application Filing
- 2009-01-13 ES ES13160338.3T patent/ES2562658T3/es active Active
- 2009-01-13 DK DK13160338.3T patent/DK2607484T3/en active
- 2009-01-13 CA CA2741629A patent/CA2741629C/en active Active
- 2009-01-13 PT PT09788160T patent/PT2344637E/pt unknown
- 2009-01-13 ES ES09788160.1T patent/ES2532634T5/es active Active
- 2009-01-13 PL PL13160338T patent/PL2607484T3/pl unknown
- 2009-01-13 AU AU2009310557A patent/AU2009310557B2/en active Active
- 2009-01-13 EP EP13160338.3A patent/EP2607484B1/en not_active Revoked
- 2009-01-13 PT PT131603383T patent/PT2607484E/pt unknown
- 2009-01-13 JP JP2011533126A patent/JP5786109B2/ja active Active
- 2009-03-11 EP EP09788170.0A patent/EP2349287B1/en active Active
- 2009-03-11 NZ NZ592498A patent/NZ592498A/en unknown
- 2009-03-11 JP JP2011533127A patent/JP5905260B2/ja active Active
- 2009-03-11 CA CA2741793A patent/CA2741793C/en active Active
- 2009-03-11 EP EP18180952.6A patent/EP3400948B1/en active Active
- 2009-03-11 WO PCT/NL2009/050113 patent/WO2010050802A2/en active Application Filing
- 2009-03-11 CA CA3017539A patent/CA3017539A1/en active Pending
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2011
- 2011-04-27 IL IL212509A patent/IL212509A/en active IP Right Grant
- 2011-04-27 IL IL212508A patent/IL212508A/en active IP Right Grant
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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
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2016
- 2016-01-25 HR HRP20160078TT patent/HRP20160078T1/hr unknown
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