AU2016324800B2 - Antisense nucleic acid - Google Patents

Antisense nucleic acid Download PDF

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Publication number
AU2016324800B2
AU2016324800B2 AU2016324800A AU2016324800A AU2016324800B2 AU 2016324800 B2 AU2016324800 B2 AU 2016324800B2 AU 2016324800 A AU2016324800 A AU 2016324800A AU 2016324800 A AU2016324800 A AU 2016324800A AU 2016324800 B2 AU2016324800 B2 AU 2016324800B2
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Prior art keywords
nucleotide sequence
oligomer
pharmaceutically acceptable
hydrate
acceptable salt
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AU2016324800A
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AU2016324800A1 (en
Inventor
Yoshitsugu Aoki
Yukiko Enya
Shin'ichi Takeda
Yuichiro Tone
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Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
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Nippon Shinyaku Co Ltd
National Center of Neurology and Psychiatry
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    • 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
    • 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
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C12N2310/313Phosphorodithioates
    • 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
    • C12N2310/32Chemical 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
    • C12N2310/323Chemical 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2016324800A 2015-09-15 2016-09-15 Antisense nucleic acid Active AU2016324800B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2015-182145 2015-09-15
JP2015182145 2015-09-15
PCT/JP2016/077305 WO2017047707A1 (ja) 2015-09-15 2016-09-15 アンチセンス核酸

Publications (2)

Publication Number Publication Date
AU2016324800A1 AU2016324800A1 (en) 2018-04-05
AU2016324800B2 true AU2016324800B2 (en) 2022-02-24

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AU2016324800A Active AU2016324800B2 (en) 2015-09-15 2016-09-15 Antisense nucleic acid

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US (4) US10144931B2 (enExample)
EP (2) EP3778895A1 (enExample)
JP (5) JP6384845B2 (enExample)
KR (3) KR101968880B1 (enExample)
CN (3) CN108026531B (enExample)
AU (1) AU2016324800B2 (enExample)
CA (1) CA2996280C (enExample)
CO (1) CO2018002557A2 (enExample)
CY (1) CY1123119T1 (enExample)
DK (1) DK3351633T3 (enExample)
ES (1) ES2808049T3 (enExample)
HR (1) HRP20201125T1 (enExample)
HU (1) HUE050061T2 (enExample)
IL (1) IL258065B (enExample)
LT (1) LT3351633T (enExample)
MX (1) MX391304B (enExample)
MY (1) MY185390A (enExample)
PH (1) PH12018500568B1 (enExample)
PL (1) PL3351633T3 (enExample)
PT (1) PT3351633T (enExample)
RS (1) RS60493B1 (enExample)
RU (1) RU2724554C2 (enExample)
SG (1) SG11201802138TA (enExample)
SI (1) SI3351633T1 (enExample)
SM (1) SMT202000379T1 (enExample)
TW (1) TWI725990B (enExample)
UA (1) UA123359C2 (enExample)
WO (1) WO2017047707A1 (enExample)
ZA (1) ZA201801682B (enExample)

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PH12018500568B1 (en) 2022-09-14
CN108026531A (zh) 2018-05-11
PL3351633T3 (pl) 2020-11-02
KR20190040098A (ko) 2019-04-16
PT3351633T (pt) 2020-07-29
TWI725990B (zh) 2021-05-01
KR102473431B1 (ko) 2022-12-01
JP2026032058A (ja) 2026-02-25
UA123359C2 (uk) 2021-03-24
CA2996280C (en) 2024-05-07
EP3778895A1 (en) 2021-02-17
RS60493B1 (sr) 2020-08-31
KR101968880B1 (ko) 2019-04-12
CN113913426B (zh) 2024-08-02
JP6977998B2 (ja) 2021-12-08
US20180265866A1 (en) 2018-09-20
RU2018113276A3 (enExample) 2020-02-06
KR20220053048A (ko) 2022-04-28
JPWO2017047707A1 (ja) 2018-07-05
US20190040387A1 (en) 2019-02-07
RU2018113276A (ru) 2019-10-16
EP3351633B1 (en) 2020-06-24
JP2022033738A (ja) 2022-03-02
US20240368597A1 (en) 2024-11-07
JP6384845B2 (ja) 2018-09-05
US20210147839A1 (en) 2021-05-20
WO2017047707A1 (ja) 2017-03-23
PH12018500568A1 (en) 2018-09-24
CA2996280A1 (en) 2017-03-23
SMT202000379T1 (it) 2020-09-10
AU2016324800A1 (en) 2018-04-05
MX2018002955A (es) 2018-05-02
SG11201802138TA (en) 2018-04-27
MY185390A (en) 2021-05-17
TW201718858A (zh) 2017-06-01
NZ740562A (en) 2021-11-26
CN113930426A (zh) 2022-01-14
CN113913426A (zh) 2022-01-11
HUE050061T2 (hu) 2020-12-28
EP3351633A1 (en) 2018-07-25
LT3351633T (lt) 2020-08-10
ES2808049T3 (es) 2021-02-25
ZA201801682B (en) 2020-07-29
CN108026531B (zh) 2021-09-14
IL258065B (en) 2022-02-01
US10144931B2 (en) 2018-12-04
IL258065A (en) 2018-05-31
US10851373B2 (en) 2020-12-01
DK3351633T3 (da) 2020-08-03
BR112018004970A2 (pt) 2018-10-09
JP2024038104A (ja) 2024-03-19
JP2018183178A (ja) 2018-11-22
EP3351633A4 (en) 2019-09-04
RU2724554C2 (ru) 2020-06-23
MX391304B (es) 2025-03-21
SI3351633T1 (sl) 2020-09-30
HRP20201125T1 (hr) 2020-10-30
US11981894B2 (en) 2024-05-14
KR20180043244A (ko) 2018-04-27
CO2018002557A2 (es) 2018-05-31
CY1123119T1 (el) 2021-10-29

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