TWI725990B - 反義核酸 - Google Patents

反義核酸 Download PDF

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Publication number
TWI725990B
TWI725990B TW105130156A TW105130156A TWI725990B TW I725990 B TWI725990 B TW I725990B TW 105130156 A TW105130156 A TW 105130156A TW 105130156 A TW105130156 A TW 105130156A TW I725990 B TWI725990 B TW I725990B
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Taiwan
Prior art keywords
nucleic acid
dna
labor
oligomer
antisense oligomer
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TW105130156A
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English (en)
Chinese (zh)
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TW201718858A (zh
Inventor
塩谷由輝子
戸根悠一郎
武田伸一
青木吉嗣
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日商日本新藥股份有限公司
國立研究開發法人國立精神 神經醫療研究中心
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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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • C07H21/04—Compounds 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
    • 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
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/32—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
    • C12N2310/323—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

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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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (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)
TW105130156A 2015-09-15 2016-09-19 反義核酸 TWI725990B (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2015182145 2015-09-15
JP2015-182145 2015-09-15

Publications (2)

Publication Number Publication Date
TW201718858A TW201718858A (zh) 2017-06-01
TWI725990B true TWI725990B (zh) 2021-05-01

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Family Applications (1)

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TW105130156A TWI725990B (zh) 2015-09-15 2016-09-19 反義核酸

Country Status (29)

Country Link
US (4) US10144931B2 (OSRAM)
EP (2) EP3778895A1 (OSRAM)
JP (4) JP6384845B2 (OSRAM)
KR (3) KR101968880B1 (OSRAM)
CN (3) CN113913426B (OSRAM)
AU (1) AU2016324800B2 (OSRAM)
CA (1) CA2996280C (OSRAM)
CO (1) CO2018002557A2 (OSRAM)
CY (1) CY1123119T1 (OSRAM)
DK (1) DK3351633T3 (OSRAM)
ES (1) ES2808049T3 (OSRAM)
HR (1) HRP20201125T1 (OSRAM)
HU (1) HUE050061T2 (OSRAM)
IL (1) IL258065B (OSRAM)
LT (1) LT3351633T (OSRAM)
MX (1) MX391304B (OSRAM)
MY (1) MY185390A (OSRAM)
PH (1) PH12018500568B1 (OSRAM)
PL (1) PL3351633T3 (OSRAM)
PT (1) PT3351633T (OSRAM)
RS (1) RS60493B1 (OSRAM)
RU (1) RU2724554C2 (OSRAM)
SG (1) SG11201802138TA (OSRAM)
SI (1) SI3351633T1 (OSRAM)
SM (1) SMT202000379T1 (OSRAM)
TW (1) TWI725990B (OSRAM)
UA (1) UA123359C2 (OSRAM)
WO (1) WO2017047707A1 (OSRAM)
ZA (1) ZA201801682B (OSRAM)

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US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
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Publication number Publication date
KR20220053048A (ko) 2022-04-28
SI3351633T1 (sl) 2020-09-30
MY185390A (en) 2021-05-17
AU2016324800B2 (en) 2022-02-24
KR102473431B1 (ko) 2022-12-01
CA2996280A1 (en) 2017-03-23
KR20190040098A (ko) 2019-04-16
EP3351633B1 (en) 2020-06-24
US11981894B2 (en) 2024-05-14
MX2018002955A (es) 2018-05-02
ZA201801682B (en) 2020-07-29
EP3778895A1 (en) 2021-02-17
ES2808049T3 (es) 2021-02-25
US20240368597A1 (en) 2024-11-07
PT3351633T (pt) 2020-07-29
JP6977998B2 (ja) 2021-12-08
US10851373B2 (en) 2020-12-01
DK3351633T3 (da) 2020-08-03
JP2024038104A (ja) 2024-03-19
HUE050061T2 (hu) 2020-12-28
US20180265866A1 (en) 2018-09-20
SMT202000379T1 (it) 2020-09-10
US20210147839A1 (en) 2021-05-20
CN113913426B (zh) 2024-08-02
CN108026531B (zh) 2021-09-14
JP2018183178A (ja) 2018-11-22
RU2724554C2 (ru) 2020-06-23
NZ740562A (en) 2021-11-26
KR101968880B1 (ko) 2019-04-12
TW201718858A (zh) 2017-06-01
JPWO2017047707A1 (ja) 2018-07-05
US20190040387A1 (en) 2019-02-07
CA2996280C (en) 2024-05-07
PL3351633T3 (pl) 2020-11-02
CN108026531A (zh) 2018-05-11
IL258065A (en) 2018-05-31
HRP20201125T1 (hr) 2020-10-30
US10144931B2 (en) 2018-12-04
PH12018500568B1 (en) 2022-09-14
EP3351633A4 (en) 2019-09-04
CN113913426A (zh) 2022-01-11
RS60493B1 (sr) 2020-08-31
AU2016324800A1 (en) 2018-04-05
LT3351633T (lt) 2020-08-10
SG11201802138TA (en) 2018-04-27
CO2018002557A2 (es) 2018-05-31
EP3351633A1 (en) 2018-07-25
RU2018113276A3 (OSRAM) 2020-02-06
PH12018500568A1 (en) 2018-09-24
CN113930426A (zh) 2022-01-14
RU2018113276A (ru) 2019-10-16
BR112018004970A2 (pt) 2018-10-09
KR20180043244A (ko) 2018-04-27
JP6384845B2 (ja) 2018-09-05
IL258065B (en) 2022-02-01
JP2022033738A (ja) 2022-03-02
CY1123119T1 (el) 2021-10-29
WO2017047707A1 (ja) 2017-03-23
UA123359C2 (uk) 2021-03-24
MX391304B (es) 2025-03-21

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