IN2014DN06220A - - Google Patents

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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

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/314—Phosphoramidates
    • 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
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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)
  • 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)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
IN6220DEN2014 2011-12-28 2012-12-27 IN2014DN06220A (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2011288040 2011-12-28
JP2012043092 2012-02-29
PCT/JP2012/084295 WO2013100190A1 (fr) 2011-12-28 2012-12-27 Acide nucléique antisens

Publications (1)

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

Family

ID=48697653

Family Applications (1)

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

Country Status (12)

Country Link
US (5) US9512424B2 (fr)
EP (2) EP2799548B1 (fr)
JP (6) JP6141770B2 (fr)
KR (2) KR102240139B1 (fr)
CN (10) CN118207212A (fr)
AU (2) AU2012360702C1 (fr)
CA (2) CA3132111A1 (fr)
ES (1) ES2748868T3 (fr)
IN (1) IN2014DN06220A (fr)
RU (3) RU2651468C1 (fr)
TW (1) TWI606059B (fr)
WO (1) WO2013100190A1 (fr)

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EP3554552B1 (fr) 2016-12-19 2022-08-17 Sarepta Therapeutics, Inc. Conjugués oligomères de sauts d'exons pour la dystrophie musculaire
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Publication number Publication date
US20170067052A1 (en) 2017-03-09
JP2017163994A (ja) 2017-09-21
US10781448B2 (en) 2020-09-22
KR20140108676A (ko) 2014-09-12
JP2024038103A (ja) 2024-03-19
US20140343266A1 (en) 2014-11-20
US9512424B2 (en) 2016-12-06
CA2861247C (fr) 2021-11-16
CN110055244A (zh) 2019-07-26
TWI606059B (zh) 2017-11-21
US20180142245A1 (en) 2018-05-24
EP2799548A4 (fr) 2015-11-18
EP3594347A1 (fr) 2020-01-15
US20250188459A1 (en) 2025-06-12
AU2012360702A1 (en) 2014-07-24
AU2018229530A1 (en) 2018-10-04
TW201336859A (zh) 2013-09-16
CN108611349A (zh) 2018-10-02
US9890381B2 (en) 2018-02-13
CN118207212A (zh) 2024-06-18
JPWO2013100190A1 (ja) 2015-05-11
CA3132111A1 (fr) 2013-07-04
RU2681470C1 (ru) 2019-03-06
CN110079525A (zh) 2019-08-02
AU2012360702C1 (en) 2018-09-20
JP7038365B2 (ja) 2022-03-18
KR102071729B1 (ko) 2020-01-31
RU2014130600A (ru) 2016-02-20
US20210222169A1 (en) 2021-07-22
JP6141770B2 (ja) 2017-06-07
CN107881175A (zh) 2018-04-06
AU2018229530B2 (en) 2021-05-13
JP2022058379A (ja) 2022-04-12
CN117721110A (zh) 2024-03-19
JP2025176110A (ja) 2025-12-03
CN108486116A (zh) 2018-09-04
JP6734222B2 (ja) 2020-08-05
CN110055243B (zh) 2024-03-26
KR102240139B1 (ko) 2021-04-13
ES2748868T3 (es) 2020-03-18
WO2013100190A1 (fr) 2013-07-04
CA2861247A1 (fr) 2013-07-04
JP2020114215A (ja) 2020-07-30
CN107881175B (zh) 2022-05-13
AU2012360702B2 (en) 2018-06-28
RU2651468C1 (ru) 2018-04-19
CN104024414A (zh) 2014-09-03
CN110055243A (zh) 2019-07-26
EP2799548B1 (fr) 2019-08-21
RU2619184C2 (ru) 2017-05-12
CN108588073A (zh) 2018-09-28
EP2799548A1 (fr) 2014-11-05
KR20200008663A (ko) 2020-01-28

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