KR102473431B1 - 안티센스 핵산 - Google Patents

안티센스 핵산 Download PDF

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KR102473431B1
KR102473431B1 KR1020197010069A KR20197010069A KR102473431B1 KR 102473431 B1 KR102473431 B1 KR 102473431B1 KR 1020197010069 A KR1020197010069 A KR 1020197010069A KR 20197010069 A KR20197010069 A KR 20197010069A KR 102473431 B1 KR102473431 B1 KR 102473431B1
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oligomer
nucleotide sequence
nucleic acid
dna
artificial
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KR20190040098A (ko
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유키코 엔야
유이치로 도네
신이치 다케다
요시쯔구 아오키
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니뽄 신야쿠 가부시키가이샤
고쿠리쯔겡뀨가이하쯔호징 고꾸리쯔 세이신ㆍ신께이 이료겡뀨센따
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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
    • 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
    • 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
    • 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
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    • 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
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    • 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
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    • 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)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
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  • Biomedical Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Veterinary Medicine (AREA)
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  • 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)
KR1020197010069A 2015-09-15 2016-09-15 안티센스 핵산 Active KR102473431B1 (ko)

Priority Applications (1)

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KR1020227012681A KR20220053048A (ko) 2015-09-15 2016-09-15 안티센스 핵산

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JPJP-P-2015-182145 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
KR1020187001380A KR101968880B1 (ko) 2015-09-15 2016-09-15 안티센스 핵산

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KR20190040098A KR20190040098A (ko) 2019-04-16
KR102473431B1 true KR102473431B1 (ko) 2022-12-01

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KR1020227012681A Ceased KR20220053048A (ko) 2015-09-15 2016-09-15 안티센스 핵산
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US (4) US10144931B2 (enExample)
EP (2) EP3351633B1 (enExample)
JP (4) JP6384845B2 (enExample)
KR (3) KR102473431B1 (enExample)
CN (3) CN113930426A (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)
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RU (1) RU2724554C2 (enExample)
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SI (1) SI3351633T1 (enExample)
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UA (1) UA123359C2 (enExample)
WO (1) WO2017047707A1 (enExample)
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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
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
GB201821269D0 (en) * 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
AU2022267324A1 (en) 2021-04-30 2023-12-14 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
AU2022298028A1 (en) * 2021-06-23 2023-12-21 National Center Of Neurology And Psychiatry Combination of antisense oligomers
EP4366784A1 (en) 2021-07-09 2024-05-15 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JPWO2023127918A1 (enExample) 2021-12-27 2023-07-06
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
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US20240368597A1 (en) 2024-11-07
WO2017047707A1 (ja) 2017-03-23
CY1123119T1 (el) 2021-10-29
MY185390A (en) 2021-05-17
NZ740562A (en) 2021-11-26
MX2018002955A (es) 2018-05-02
JP2022033738A (ja) 2022-03-02
AU2016324800B2 (en) 2022-02-24
UA123359C2 (uk) 2021-03-24
JP6977998B2 (ja) 2021-12-08
PL3351633T3 (pl) 2020-11-02
BR112018004970A2 (pt) 2018-10-09
KR101968880B1 (ko) 2019-04-12
RU2018113276A (ru) 2019-10-16
AU2016324800A1 (en) 2018-04-05
PH12018500568B1 (en) 2022-09-14
CO2018002557A2 (es) 2018-05-31
SMT202000379T1 (it) 2020-09-10
IL258065A (en) 2018-05-31
TW201718858A (zh) 2017-06-01
JP2024038104A (ja) 2024-03-19
IL258065B (en) 2022-02-01
SG11201802138TA (en) 2018-04-27
US20210147839A1 (en) 2021-05-20
CN113913426B (zh) 2024-08-02
LT3351633T (lt) 2020-08-10
ZA201801682B (en) 2020-07-29
EP3351633B1 (en) 2020-06-24
CN108026531A (zh) 2018-05-11
US10851373B2 (en) 2020-12-01
KR20190040098A (ko) 2019-04-16
JP6384845B2 (ja) 2018-09-05
RS60493B1 (sr) 2020-08-31
US11981894B2 (en) 2024-05-14
PH12018500568A1 (en) 2018-09-24
EP3351633A1 (en) 2018-07-25
MX391304B (es) 2025-03-21
JPWO2017047707A1 (ja) 2018-07-05
TWI725990B (zh) 2021-05-01
SI3351633T1 (sl) 2020-09-30
KR20220053048A (ko) 2022-04-28
RU2018113276A3 (enExample) 2020-02-06
RU2724554C2 (ru) 2020-06-23
HRP20201125T1 (hr) 2020-10-30
EP3351633A4 (en) 2019-09-04
DK3351633T3 (da) 2020-08-03
CN113913426A (zh) 2022-01-11
JP2018183178A (ja) 2018-11-22
EP3778895A1 (en) 2021-02-17
US20190040387A1 (en) 2019-02-07
CA2996280A1 (en) 2017-03-23
KR20180043244A (ko) 2018-04-27
CN108026531B (zh) 2021-09-14
US10144931B2 (en) 2018-12-04
CA2996280C (en) 2024-05-07
CN113930426A (zh) 2022-01-14
US20180265866A1 (en) 2018-09-20
PT3351633T (pt) 2020-07-29
ES2808049T3 (es) 2021-02-25
HUE050061T2 (hu) 2020-12-28

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