KR101968880B1 - 안티센스 핵산 - Google Patents

안티센스 핵산 Download PDF

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KR101968880B1
KR101968880B1 KR1020187001380A KR20187001380A KR101968880B1 KR 101968880 B1 KR101968880 B1 KR 101968880B1 KR 1020187001380 A KR1020187001380 A KR 1020187001380A KR 20187001380 A KR20187001380 A KR 20187001380A KR 101968880 B1 KR101968880 B1 KR 101968880B1
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nucleic acid
dna
artificial
pharmaceutically acceptable
hydrate
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KR20180043244A (ko
Inventor
유키코 엔야
유이치로 도네
신이치 다케다
요시쯔구 아오키
Original Assignee
니뽄 신야쿠 가부시키가이샤
고쿠리쯔겡뀨가이하쯔호징 고꾸리쯔 세이신ㆍ신께이 이료겡뀨센따
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Priority to KR1020197010069A priority Critical patent/KR102473431B1/ko
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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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/313Phosphorodithioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • CCHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications

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  • Health & Medical Sciences (AREA)
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  • 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)
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KR1020187001380A 2015-09-15 2016-09-15 안티센스 핵산 Active KR101968880B1 (ko)

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KR1020197010069A KR102473431B1 (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

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KR101968880B1 true KR101968880B1 (ko) 2019-04-12

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KR1020227012681A Ceased KR20220053048A (ko) 2015-09-15 2016-09-15 안티센스 핵산
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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)
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HR (1) HRP20201125T1 (enExample)
HU (1) HUE050061T2 (enExample)
IL (1) IL258065B (enExample)
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PH (1) PH12018500568B1 (enExample)
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RU (1) RU2724554C2 (enExample)
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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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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
EP4493693A1 (en) 2022-03-17 2025-01-22 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 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
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
KR102473431B1 (ko) 2022-12-01
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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