CN108026531B - 反义核酸 - Google Patents

反义核酸 Download PDF

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Publication number
CN108026531B
CN108026531B CN201680053726.9A CN201680053726A CN108026531B CN 108026531 B CN108026531 B CN 108026531B CN 201680053726 A CN201680053726 A CN 201680053726A CN 108026531 B CN108026531 B CN 108026531B
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dna
nucleic acids
pharmaceutically acceptable
artificial
hydrate
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CN108026531A (zh
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盐谷由辉子
户根悠一郎
武田伸一
青木吉嗣
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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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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Chemical Kinetics & Catalysis (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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CN201680053726.9A 2015-09-15 2016-09-15 反义核酸 Active CN108026531B (zh)

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CN202111073060.2A CN113930426A (zh) 2015-09-15 2016-09-15 反义核酸
CN202111073029.9A CN113913426B (zh) 2015-09-15 2016-09-15 反义核酸

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JP2015182145 2015-09-15
JP2015-182145 2015-09-15
PCT/JP2016/077305 WO2017047707A1 (ja) 2015-09-15 2016-09-15 アンチセンス核酸

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CN202111073029.9A Division CN113913426B (zh) 2015-09-15 2016-09-15 反义核酸

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CN108026531B true CN108026531B (zh) 2021-09-14

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US (4) US10144931B2 (enExample)
EP (2) EP3778895A1 (enExample)
JP (4) JP6384845B2 (enExample)
KR (3) KR101968880B1 (enExample)
CN (3) CN113913426B (enExample)
AU (1) AU2016324800B2 (enExample)
CA (1) CA2996280C (enExample)
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SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
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
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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IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
TW202307208A (zh) * 2021-06-23 2023-02-16 日商日本新藥股份有限公司 反義寡聚物之組合
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
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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
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
TWI725990B (zh) 2021-05-01
CO2018002557A2 (es) 2018-05-31
EP3351633A1 (en) 2018-07-25
RU2018113276A3 (enExample) 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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