IN2014DN06220A - - Google Patents

Download PDF

Info

Publication number
IN2014DN06220A
IN2014DN06220A IN6220DEN2014A IN2014DN06220A IN 2014DN06220 A IN2014DN06220 A IN 2014DN06220A IN 6220DEN2014 A IN6220DEN2014 A IN 6220DEN2014A IN 2014DN06220 A IN2014DN06220 A IN 2014DN06220A
Authority
IN
India
Prior art keywords
exons
dystrophin gene
human dystrophin
oligomer
drug
Prior art date
Application number
Other languages
English (en)
Inventor
Naoki Watanabe
Haruna Seo
Shinichi Takeda
Tetsuya Nagata
Original Assignee
Nippon Shinyaku Co Ltd
Nat Ct Neurology & Psychiatry
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Nippon Shinyaku Co Ltd, Nat Ct Neurology & Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of IN2014DN06220A publication Critical patent/IN2014DN06220A/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (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)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
IN6220DEN2014 2011-12-28 2012-12-27 IN2014DN06220A (OSRAM)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2011288040 2011-12-28
JP2012043092 2012-02-29
PCT/JP2012/084295 WO2013100190A1 (ja) 2011-12-28 2012-12-27 アンチセンス核酸

Publications (1)

Publication Number Publication Date
IN2014DN06220A true IN2014DN06220A (OSRAM) 2015-10-23

Family

ID=48697653

Family Applications (1)

Application Number Title Priority Date Filing Date
IN6220DEN2014 IN2014DN06220A (OSRAM) 2011-12-28 2012-12-27

Country Status (12)

Country Link
US (5) US9512424B2 (OSRAM)
EP (2) EP3594347A1 (OSRAM)
JP (6) JP6141770B2 (OSRAM)
KR (2) KR102240139B1 (OSRAM)
CN (10) CN110055243B (OSRAM)
AU (2) AU2012360702C1 (OSRAM)
CA (2) CA3132111A1 (OSRAM)
ES (1) ES2748868T3 (OSRAM)
IN (1) IN2014DN06220A (OSRAM)
RU (3) RU2619184C2 (OSRAM)
TW (1) TWI606059B (OSRAM)
WO (1) WO2013100190A1 (OSRAM)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
USRE47769E1 (en) 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling 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
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
IN2014DN06220A (OSRAM) * 2011-12-28 2015-10-23 Nippon Shinyaku Co Ltd
CN111893117B (zh) 2012-01-27 2024-06-04 比奥马林技术公司 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
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
MX373959B (es) 2013-03-15 2020-07-13 Sarepta Therapeutics Inc Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd).
EP3514234A1 (en) * 2014-03-12 2019-07-24 Nippon Shinyaku Co., Ltd. Antisense nucleic acid
ES2930274T3 (es) * 2014-05-19 2022-12-09 Knc Laboratories Co Ltd Fármaco de ácido nucleico para inducir la omisión de variantes exónicas del gen de CD44 y aumentar la expresión de ARNm de CD44 de tipo normal
UA121117C2 (uk) 2014-06-17 2020-04-10 Ніппон Шин'Яку Ко., Лтд. Антисенсова нуклеїнова кислота
CN106795186B (zh) 2014-10-14 2021-03-02 味之素株式会社 吗啉代寡核苷酸的制备方法
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
RU2724554C2 (ru) * 2015-09-15 2020-06-23 Ниппон Синяку Ко., Лтд. Антисмысловая нуклеиновая кислота
JP7511326B2 (ja) 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
EP3464305B1 (en) * 2016-05-24 2024-08-21 Sarepta Therapeutics, Inc. Processes for preparing oligomers
CN109311920B (zh) * 2016-05-24 2021-11-09 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
JP2019525742A (ja) 2016-06-30 2019-09-12 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
CA3046220A1 (en) * 2016-12-08 2018-06-14 The Board Of Regents Of The University Of Texas System Dmd reporter models containing humanized duschene muscular dystrophy mutations
KR20240006023A (ko) * 2016-12-19 2024-01-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
EP3554553B1 (en) 2016-12-19 2022-07-20 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
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
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
EA202191601A1 (ru) * 2018-12-13 2022-01-19 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
AU2018454784A1 (en) * 2018-12-25 2021-08-05 National Center Of Neurology And Psychiatry Method for inducing muscular cells using cells in spot urine
AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
MX2022007491A (es) * 2019-12-19 2022-08-15 Nippon Shinyaku Co Ltd Acido nucleico antisentido que permite la omision de exones.
CN118109469A (zh) 2019-12-26 2024-05-31 日本新药株式会社 诱导外显子50的跳读的反义核酸
IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
WO2022239863A1 (ja) 2021-05-13 2022-11-17 国立大学法人千葉大学 アンチセンスオリゴマー
TW202307208A (zh) 2021-06-23 2023-02-16 日商日本新藥股份有限公司 反義寡聚物之組合
CN118201606A (zh) 2021-07-08 2024-06-14 日本新药株式会社 肾毒性减轻剂
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
TW202317147A (zh) 2021-07-08 2023-05-01 日商日本新藥股份有限公司 析出抑制劑
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20240318176A1 (en) * 2021-07-09 2024-09-26 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
MX2024000489A (es) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías.
WO2023026994A1 (ja) 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド-薬物コンジュゲート
US20250066396A1 (en) 2021-12-27 2025-02-27 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
JP2025079347A (ja) * 2022-03-11 2025-05-22 日本新薬株式会社 キャリアペプチドが連結された核酸
US20250195667A1 (en) 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
US12071621B2 (en) * 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991009033A1 (en) 1989-12-20 1991-06-27 Anti-Gene Development Group Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体,デバイス及び医薬組成物
FI951138L (fi) * 1993-07-13 1995-04-13 Rhone Poulenc Rorer Sa Puuttellisia adenovirusvektoreita ja niiden käyttö geeniterapiassa
US6727355B2 (en) * 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
CA2942791C (en) 2002-11-25 2019-08-20 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
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
USRE47769E1 (en) * 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP1811024A1 (en) 2004-10-05 2007-07-25 Nippon Shinyaku Co., Ltd. Oligo double-stranded rna and medicinal composition
KR20080031164A (ko) * 2005-04-22 2008-04-08 아카데미슈 지켄후이스 라이덴 SR 단백질의 결합의 방해에 의해 그리고 이차 RNA구조의 방해에 의한 pre-mRNA에서 엑손 인식의조절
WO2006129594A1 (ja) 2005-05-30 2006-12-07 Nippon Shinyaku Co., Ltd. 核酸含有複合体製剤の製造方法
US8067571B2 (en) * 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
EP1857548A1 (en) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US8466255B2 (en) 2007-02-05 2013-06-18 Nippon Shinyaku Co., Ltd. Polyethylene glycol derivative
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
HRP20140646T1 (hr) 2007-11-15 2014-09-26 Sarepta Therapeutics, Inc. Postupak sinteze morfolino oligomera
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
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
TR201902952T4 (tr) * 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
PT2344637E (pt) 2008-10-27 2015-03-23 Academisch Ziekenhuis Leiden Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne
JP2012524540A (ja) * 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー.ブイ. Dmdを処置するためのイノシンを含むオリゴヌクレオチド
US20120270930A1 (en) * 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies
CA2799501C (en) * 2010-05-28 2022-02-15 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP5593949B2 (ja) 2010-08-17 2014-09-24 横河電機株式会社 プログラマブルロジックコントローラ
IN2014DN06220A (OSRAM) * 2011-12-28 2015-10-23 Nippon Shinyaku Co Ltd

Also Published As

Publication number Publication date
KR20140108676A (ko) 2014-09-12
US20180142245A1 (en) 2018-05-24
KR102240139B1 (ko) 2021-04-13
EP2799548B1 (en) 2019-08-21
CN110079525A (zh) 2019-08-02
CN107881175A (zh) 2018-04-06
US20170067052A1 (en) 2017-03-09
RU2014130600A (ru) 2016-02-20
AU2018229530A1 (en) 2018-10-04
WO2013100190A1 (ja) 2013-07-04
KR20200008663A (ko) 2020-01-28
US10781448B2 (en) 2020-09-22
US20210222169A1 (en) 2021-07-22
RU2681470C1 (ru) 2019-03-06
CN104024414A (zh) 2014-09-03
EP3594347A1 (en) 2020-01-15
CN117721110A (zh) 2024-03-19
JPWO2013100190A1 (ja) 2015-05-11
CN107881175B (zh) 2022-05-13
RU2619184C2 (ru) 2017-05-12
AU2012360702C1 (en) 2018-09-20
CA2861247A1 (en) 2013-07-04
JP2022058379A (ja) 2022-04-12
AU2012360702B2 (en) 2018-06-28
US9890381B2 (en) 2018-02-13
CN108486116A (zh) 2018-09-04
CN110055244A (zh) 2019-07-26
JP2020114215A (ja) 2020-07-30
AU2018229530B2 (en) 2021-05-13
CN108588073A (zh) 2018-09-28
KR102071729B1 (ko) 2020-01-31
AU2012360702A1 (en) 2014-07-24
RU2651468C1 (ru) 2018-04-19
TW201336859A (zh) 2013-09-16
JP2017163994A (ja) 2017-09-21
CA3132111A1 (en) 2013-07-04
JP2025176110A (ja) 2025-12-03
JP7038365B2 (ja) 2022-03-18
CN110055243B (zh) 2024-03-26
CN118207212A (zh) 2024-06-18
ES2748868T3 (es) 2020-03-18
EP2799548A4 (en) 2015-11-18
CA2861247C (en) 2021-11-16
JP6734222B2 (ja) 2020-08-05
CN110055243A (zh) 2019-07-26
EP2799548A1 (en) 2014-11-05
TWI606059B (zh) 2017-11-21
US9512424B2 (en) 2016-12-06
US20250188459A1 (en) 2025-06-12
JP6141770B2 (ja) 2017-06-07
CN108611349A (zh) 2018-10-02
JP2024038103A (ja) 2024-03-19
US20140343266A1 (en) 2014-11-20

Similar Documents

Publication Publication Date Title
IN2014DN06220A (OSRAM)
PH12019502292A1 (en) Methods and compositions for treatment of a genetic condition
EP2828275A4 (en) SYNTHESIS OF 3-O-FUCOSYLLACTOSE TYPE TRISACCHARIDE AND ITS INTERMEDIATES
PH12016501761A1 (en) Antisense nucleic acids
SI3378862T1 (sl) Dihidropirimidinoizokuinolini in farmacevtski sestavki le-teh za zdravljenje multiple skleroze
IL225769A0 (en) Piperidine-4-yl-azetidine diamides as monoacylglycerol lipase inhibitors
PH12015500525A1 (en) Formulations of enzalutamide
IT1401855B1 (it) Apparecchiatura per il trattamento di un prodotto.
IL235349A0 (en) Therapeutic use of Chardonnay grape products
ZA201307956B (en) Pharmaceutical compositions comprising sulbactam and beta-lactamase inhibitor
HUE036248T2 (hu) Aminoglükozidok és alkalmazásaik genetikai rendellenességek kezelésére
EP2971156A4 (en) Genes and gene signatures for diagnosis and treatment of melanoma
IN2015DN01524A (OSRAM)
IL225406B (en) Therapeutic applications of smad7
EP3052654A4 (en) Identification of structurally similar small molecules that enhance therapeutic exon skipping
EP2798450A4 (en) TRIGGERING THE DISPLAY OF ELEMENTS
ZA201408062B (en) Compositions and methods for the treatment of multiple sclerosis
MY165088A (en) Pharmaceutical compositions comprising alisporivir
EP2661434A4 (en) NEW URGES FOR THE TREATMENT AND PREVENTION OF CANCER
ZA201306420B (en) Regulated gene expression systems and constructs thereof
ZA201408058B (en) Compositions and methods for the treatment of epilepsy
IL237781A0 (en) A novel phenylacetamide compound, and a drug containing it
IN2015DN02647A (OSRAM)
IT1403250B1 (it) Macroapparato per la produzione e il trattamento di gas
GB201008916D0 (en) Means for inhibiting the expression of gene associated with retinoid IFN-beta induced mortality-19 (grim-19)