LT3118311T - Priešprasminė nukleorūgštis - Google Patents

Priešprasminė nukleorūgštis

Info

Publication number
LT3118311T
LT3118311T LTEP15761392.8T LT15761392T LT3118311T LT 3118311 T LT3118311 T LT 3118311T LT 15761392 T LT15761392 T LT 15761392T LT 3118311 T LT3118311 T LT 3118311T
Authority
LT
Lithuania
Prior art keywords
nucleic acid
antisense nucleic
antisense
acid
nucleic
Prior art date
Application number
LTEP15761392.8T
Other languages
English (en)
Inventor
Tatsushi Wakayama
Haruna Seo
Youhei Satou
Shin`ichi TAKEDA
Tetsuya Nagata
Original Assignee
Nippon Shinyaku Co., Ltd.
National Center Of Neurology And 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., National Center Of Neurology And Psychiatry filed Critical Nippon Shinyaku Co., Ltd.
Publication of LT3118311T publication Critical patent/LT3118311T/lt

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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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3535—Nitrogen
    • 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)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (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)
LTEP15761392.8T 2014-03-12 2015-03-11 Priešprasminė nukleorūgštis LT3118311T (lt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2014048897 2014-03-12
PCT/JP2015/057180 WO2015137409A1 (ja) 2014-03-12 2015-03-11 アンチセンス核酸

Publications (1)

Publication Number Publication Date
LT3118311T true LT3118311T (lt) 2019-04-10

Family

ID=54071850

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP15761392.8T LT3118311T (lt) 2014-03-12 2015-03-11 Priešprasminė nukleorūgštis

Country Status (30)

Country Link
US (4) US9988629B2 (lt)
EP (2) EP3118311B1 (lt)
JP (1) JP6606488B2 (lt)
KR (1) KR102335801B1 (lt)
CN (2) CN106459955B (lt)
AU (2) AU2015227733B2 (lt)
BR (1) BR112016020618B1 (lt)
CA (1) CA2939948A1 (lt)
CY (1) CY1121322T1 (lt)
DK (1) DK3118311T3 (lt)
ES (1) ES2710802T3 (lt)
HR (1) HRP20190235T1 (lt)
HU (1) HUE042283T2 (lt)
IL (1) IL247663B (lt)
LT (1) LT3118311T (lt)
MX (1) MX2016011538A (lt)
MY (1) MY193708A (lt)
NZ (1) NZ724836A (lt)
PH (1) PH12016501761B1 (lt)
PL (1) PL3118311T3 (lt)
PT (1) PT3118311T (lt)
RS (1) RS58573B1 (lt)
RU (2) RU2730681C2 (lt)
SG (1) SG11201607095RA (lt)
SI (1) SI3118311T1 (lt)
TR (1) TR201901939T4 (lt)
TW (2) TW201945383A (lt)
UA (1) UA120849C2 (lt)
WO (1) WO2015137409A1 (lt)
ZA (1) ZA201605864B (lt)

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SG11202105626TA (en) * 2018-12-06 2021-06-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
WO2020158792A1 (ja) * 2019-01-30 2020-08-06 国立研究開発法人国立精神・神経医療研究センター 核酸送達複合体
BR112022017066A2 (pt) 2020-02-28 2022-11-16 Nippon Shinyaku Co Ltd Ácido nucleico antissentido indutor de salto do éxon 51
BR112023021849A2 (pt) 2021-04-30 2023-12-19 Sarepta Therapeutics Inc Métodos de tratamento para distrofia muscular
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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Also Published As

Publication number Publication date
EP3118311A4 (en) 2017-10-11
TW201945383A (zh) 2019-12-01
MY193708A (en) 2022-10-26
RU2019130513A (ru) 2019-10-10
RU2016139108A3 (lt) 2018-08-06
SG11201607095RA (en) 2016-10-28
RU2016139108A (ru) 2018-04-13
CY1121322T1 (el) 2020-05-29
EP3118311B1 (en) 2018-12-26
CN110951732A (zh) 2020-04-03
BR112016020618A2 (pt) 2017-08-15
JPWO2015137409A1 (ja) 2017-04-06
PH12016501761A1 (en) 2016-11-07
RU2702424C2 (ru) 2019-10-08
IL247663B (en) 2020-06-30
NZ724836A (en) 2022-07-01
EP3118311A1 (en) 2017-01-18
DK3118311T3 (en) 2019-03-11
PL3118311T3 (pl) 2019-09-30
US20220162604A1 (en) 2022-05-26
SI3118311T1 (sl) 2019-05-31
PH12016501761B1 (en) 2016-11-07
JP6606488B2 (ja) 2019-11-13
ZA201605864B (en) 2018-12-19
HUE042283T2 (hu) 2019-06-28
AU2020200679A1 (en) 2020-02-20
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RS58573B1 (sr) 2019-05-31
BR112016020618B1 (pt) 2023-04-04
BR112016020618A8 (pt) 2021-07-13
UA120849C2 (uk) 2020-02-25
TWI672314B (zh) 2019-09-21
AU2015227733B2 (en) 2019-10-31
HRP20190235T1 (hr) 2019-05-03
TR201901939T4 (tr) 2019-03-21
CN106459955B (zh) 2019-12-20
AU2020200679B2 (en) 2022-03-10
KR102335801B1 (ko) 2021-12-03
ES2710802T3 (es) 2019-04-26
WO2015137409A1 (ja) 2015-09-17
US20180179538A1 (en) 2018-06-28
US20170067048A1 (en) 2017-03-09
US20200149040A1 (en) 2020-05-14
MX2016011538A (es) 2017-04-13
EP3514234A1 (en) 2019-07-24
PT3118311T (pt) 2019-02-15
US11053497B2 (en) 2021-07-06
IL247663A0 (en) 2016-11-30
CA2939948A1 (en) 2015-09-17
RU2730681C2 (ru) 2020-08-24
KR20160132056A (ko) 2016-11-16
TW201540724A (zh) 2015-11-01
US9988629B2 (en) 2018-06-05
AU2015227733A1 (en) 2016-10-20

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