HK1209456A1 - Oligonucleotide for the treatment of muscular dystrophy patients - Google Patents
Oligonucleotide for the treatment of muscular dystrophy patientsInfo
- Publication number
- HK1209456A1 HK1209456A1 HK15110181.1A HK15110181A HK1209456A1 HK 1209456 A1 HK1209456 A1 HK 1209456A1 HK 15110181 A HK15110181 A HK 15110181A HK 1209456 A1 HK1209456 A1 HK 1209456A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- oligonucleotide
- treatment
- muscular dystrophy
- dystrophy patients
- patients
- Prior art date
Links
Classifications
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2310/33—Chemical structure of the base
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201261667517P | 2012-07-03 | 2012-07-03 | |
EP12174781 | 2012-07-03 | ||
PCT/NL2013/050487 WO2014007620A2 (en) | 2012-07-03 | 2013-07-03 | Oligonucleotide for the treatment of muscular dystrophy patients |
Publications (1)
Publication Number | Publication Date |
---|---|
HK1209456A1 true HK1209456A1 (en) | 2016-04-01 |
Family
ID=49882554
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HK15110181.1A HK1209456A1 (en) | 2012-07-03 | 2015-10-16 | Oligonucleotide for the treatment of muscular dystrophy patients |
Country Status (11)
Country | Link |
---|---|
US (1) | US20150191725A1 (ru) |
EP (2) | EP2870246B1 (ru) |
JP (1) | JP6460983B2 (ru) |
CN (2) | CN110257379B (ru) |
AU (1) | AU2013285698A1 (ru) |
BR (1) | BR112014033004B1 (ru) |
CA (1) | CA2877644A1 (ru) |
HK (1) | HK1209456A1 (ru) |
IL (1) | IL236504B (ru) |
RU (1) | RU2674600C2 (ru) |
WO (1) | WO2014007620A2 (ru) |
Families Citing this family (41)
Publication number | Priority date | Publication date | Assignee | Title |
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EP4047096A1 (en) | 2004-06-28 | 2022-08-24 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
CA2660523C (en) | 2006-08-11 | 2019-03-19 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
EP2614827B1 (en) | 2007-10-26 | 2017-06-28 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
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 |
EP3428278A1 (en) | 2008-10-24 | 2019-01-16 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
CN103003430A (zh) | 2009-11-12 | 2013-03-27 | 西澳大利亚大学 | 反义分子和治疗疾病的方法 |
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 |
EP4043039A1 (en) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
EP2870246B1 (en) * | 2012-07-03 | 2019-09-11 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
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 |
LT3159409T (lt) * | 2014-06-17 | 2020-01-27 | Nippon Shinyaku Co., Ltd. | Antiprasminė nukleorūgštis, skirta panaudoti diušeno raumenų distrofijos gydymui |
MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
KR20240035901A (ko) * | 2015-05-19 | 2024-03-18 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 펩티드 올리고뉴클레오티드 콘주게이트 |
CN104894651B (zh) * | 2015-06-29 | 2017-04-12 | 天津诺禾医学检验所有限公司 | 微量起始dna的高通量测序文库构建方法及其所构建的高通量测序文库 |
CN108026531B (zh) * | 2015-09-15 | 2021-09-14 | 日本新药株式会社 | 反义核酸 |
EP3359523A4 (en) * | 2015-10-09 | 2019-07-24 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
EP3359668A4 (en) * | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
JP7039800B2 (ja) * | 2016-03-09 | 2022-03-23 | 学校法人東京理科大学 | Rnaハイブリッド形成用核酸オリゴマー |
WO2018005805A1 (en) | 2016-06-30 | 2018-01-04 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
CA3029772A1 (en) * | 2016-07-05 | 2018-01-11 | Biomarin Technologies B.V. | Pre-mrna splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders |
CN110267970A (zh) * | 2016-11-16 | 2019-09-20 | 比奥马林技术公司 | 用于靶向各种选定器官或组织的物质 |
WO2018118599A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
CA3047010A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
LT3554554T (lt) | 2016-12-19 | 2022-11-25 | Sarepta Therapeutics, Inc. | Egzoną praleidžiantys oligomero konjugatai nuo raumenų distrofijos |
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 |
CN112004928A (zh) * | 2018-04-12 | 2020-11-27 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
MX2021001284A (es) | 2018-08-02 | 2021-07-15 | Dyne Therapeutics Inc | Complejos dirigidos al musculo y sus usos para el tratamiento de distrofia muscular facioescapulohumeral. |
CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
AU2020263487A1 (en) * | 2019-04-25 | 2021-12-16 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
US20220288218A1 (en) * | 2019-07-09 | 2022-09-15 | The Governors Of The University Of Alberta | Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the dmd gene |
CN111647663B (zh) * | 2020-06-18 | 2022-07-01 | 安徽农业大学 | 一种鸡步行数性状的分子遗传标记及应用 |
BR112023027298A2 (pt) | 2021-06-23 | 2024-03-12 | Nat Center Neurology & Psychiatry | Combinação de oligômeros antissenso |
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 |
WO2024064237A2 (en) * | 2022-09-21 | 2024-03-28 | Sarepta Therapeutics, Inc. | Dmd antisense oligonucleotide-mediated exon skipping efficiency |
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EP1191097A1 (en) * | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
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 |
EP4047096A1 (en) * | 2004-06-28 | 2022-08-24 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
CN101184841A (zh) * | 2005-04-22 | 2008-05-21 | 莱顿教学医院 | 通过干扰SR蛋白的结合以及干扰RNA二级结构调节前mRNA中的外显子识别 |
EP1857548A1 (en) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
EP2614827B1 (en) * | 2007-10-26 | 2017-06-28 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
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 |
EP3428278A1 (en) * | 2008-10-24 | 2019-01-16 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
PT2607484E (pt) * | 2008-10-27 | 2016-03-09 | Academisch Ziekenhuis Leiden | Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne |
EP2421971B1 (en) * | 2009-04-24 | 2016-07-06 | BioMarin Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
US20120172415A1 (en) * | 2009-08-31 | 2012-07-05 | Thomas Voit | Exon Skipping Therapy for Functional Amelioration of Semifunctional Dystrophin in Becker and Duchenne Muscular Dystrophy |
US20120270930A1 (en) * | 2009-10-29 | 2012-10-25 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Methods and compositions for dysferlin exon-skipping |
CN103003430A (zh) * | 2009-11-12 | 2013-03-27 | 西澳大利亚大学 | 反义分子和治疗疾病的方法 |
WO2011078797A2 (en) * | 2009-12-22 | 2011-06-30 | Singapore Health Services Pte. Ltd | Antisense oligonucleotides and uses threreof |
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KR20140052963A (ko) * | 2011-02-08 | 2014-05-07 | 더 샬롯테-맥클렌버그 하스피털 오쏘러티 디/비/에이 카롤리나스 헬스케어 시스템 | 안티센스 올리고뉴클레오티드 |
US20130013378A1 (en) | 2011-07-08 | 2013-01-10 | Jeremy Michael Norris | Method of evaluating lost revenue based on web page response time |
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US20140350080A1 (en) | 2011-10-28 | 2014-11-27 | University Of The Witwatersrand, Johannesburg | Inhibition of viral gene expression |
DK2790736T3 (en) * | 2011-12-12 | 2018-05-07 | Oncoimmunin Inc | In vivo delivery of oligonucleotides |
EP4043039A1 (en) * | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
EP2870246B1 (en) * | 2012-07-03 | 2019-09-11 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
-
2013
- 2013-07-03 EP EP13739847.5A patent/EP2870246B1/en active Active
- 2013-07-03 CA CA2877644A patent/CA2877644A1/en active Pending
- 2013-07-03 BR BR112014033004-2A patent/BR112014033004B1/pt active IP Right Grant
- 2013-07-03 EP EP19189136.5A patent/EP3608407A1/en active Pending
- 2013-07-03 WO PCT/NL2013/050487 patent/WO2014007620A2/en active Application Filing
- 2013-07-03 RU RU2015103227A patent/RU2674600C2/ru active
- 2013-07-03 CN CN201910551250.7A patent/CN110257379B/zh active Active
- 2013-07-03 JP JP2015520090A patent/JP6460983B2/ja active Active
- 2013-07-03 CN CN201380045771.6A patent/CN104603271B/zh active Active
- 2013-07-03 AU AU2013285698A patent/AU2013285698A1/en not_active Abandoned
-
2014
- 2014-12-23 US US14/581,633 patent/US20150191725A1/en not_active Abandoned
- 2014-12-28 IL IL236504A patent/IL236504B/en active IP Right Grant
-
2015
- 2015-10-16 HK HK15110181.1A patent/HK1209456A1/xx unknown
Also Published As
Publication number | Publication date |
---|---|
IL236504A0 (en) | 2015-02-26 |
RU2018141234A (ru) | 2018-12-27 |
JP6460983B2 (ja) | 2019-01-30 |
CA2877644A1 (en) | 2014-01-09 |
US20150191725A1 (en) | 2015-07-09 |
WO2014007620A3 (en) | 2014-02-27 |
JP2015522275A (ja) | 2015-08-06 |
EP2870246A2 (en) | 2015-05-13 |
CN110257379B (zh) | 2023-08-11 |
RU2015103227A (ru) | 2016-08-20 |
CN104603271B (zh) | 2019-07-19 |
RU2674600C2 (ru) | 2018-12-11 |
IL236504B (en) | 2019-02-28 |
BR112014033004B1 (pt) | 2021-10-19 |
WO2014007620A2 (en) | 2014-01-09 |
CN104603271A (zh) | 2015-05-06 |
EP2870246B1 (en) | 2019-09-11 |
BR112014033004A2 (pt) | 2017-08-01 |
CN110257379A (zh) | 2019-09-20 |
AU2013285698A1 (en) | 2015-02-19 |
EP3608407A1 (en) | 2020-02-12 |
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