KR20220053048A - 안티센스 핵산 - Google Patents

안티센스 핵산 Download PDF

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KR20220053048A
KR20220053048A KR1020227012681A KR20227012681A KR20220053048A KR 20220053048 A KR20220053048 A KR 20220053048A KR 1020227012681 A KR1020227012681 A KR 1020227012681A KR 20227012681 A KR20227012681 A KR 20227012681A KR 20220053048 A KR20220053048 A KR 20220053048A
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oligomer
pharmaceutically acceptable
hydrate
nucleic acid
acceptable salt
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Korean (ko)
Inventor
유키코 엔야
유이치로 도네
신이치 다케다
요시쯔구 아오키
Original Assignee
니뽄 신야쿠 가부시키가이샤
고쿠리쯔겡뀨가이하쯔호징 고꾸리쯔 세이신ㆍ신께이 이료겡뀨센따
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Publication of KR20220053048A publication Critical patent/KR20220053048A/ko
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    • 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
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
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    • C12N2310/3233Morpholino-type ring
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  • General Chemical & Material Sciences (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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KR1020227012681A 2015-09-15 2016-09-15 안티센스 핵산 Ceased KR20220053048A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
JP2015182145 2015-09-15
JPJP-P-2015-182145 2015-09-15
KR1020197010069A KR102473431B1 (ko) 2015-09-15 2016-09-15 안티센스 핵산
PCT/JP2016/077305 WO2017047707A1 (ja) 2015-09-15 2016-09-15 アンチセンス核酸

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KR1020197010069A Division KR102473431B1 (ko) 2015-09-15 2016-09-15 안티센스 핵산

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KR20220053048A true KR20220053048A (ko) 2022-04-28

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KR1020187001380A Active KR101968880B1 (ko) 2015-09-15 2016-09-15 안티센스 핵산
KR1020227012681A Ceased KR20220053048A (ko) 2015-09-15 2016-09-15 안티센스 핵산
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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)
CO (1) CO2018002557A2 (enExample)
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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
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
GB201821269D0 (en) * 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
EP4330395A1 (en) 2021-04-30 2024-03-06 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
MX2023014417A (es) * 2021-06-23 2023-12-15 Nippon Shinyaku Co Ltd Combinacion de oligomeros antisentido.
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
AU2022309028A1 (en) 2021-07-09 2024-01-25 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
WO2023127918A1 (ja) 2021-12-27 2023-07-06 日本新薬株式会社 オリゴ核酸化合物の製造方法
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
CA3252177A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. MORPHOLINO PHOSPHORODIAMIDATE OLIGOMER CONJUGATE
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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EP3778895A1 (en) 2021-02-17
EP3351633A1 (en) 2018-07-25
UA123359C2 (uk) 2021-03-24
RU2018113276A3 (enExample) 2020-02-06
MX2018002955A (es) 2018-05-02
US11981894B2 (en) 2024-05-14
KR20190040098A (ko) 2019-04-16
JP2022033738A (ja) 2022-03-02
BR112018004970A2 (pt) 2018-10-09
ZA201801682B (en) 2020-07-29
IL258065A (en) 2018-05-31
HUE050061T2 (hu) 2020-12-28
US10144931B2 (en) 2018-12-04
IL258065B (en) 2022-02-01
ES2808049T3 (es) 2021-02-25
CA2996280C (en) 2024-05-07
JP2018183178A (ja) 2018-11-22
EP3351633A4 (en) 2019-09-04
CA2996280A1 (en) 2017-03-23
TWI725990B (zh) 2021-05-01
RU2018113276A (ru) 2019-10-16
WO2017047707A1 (ja) 2017-03-23
KR101968880B1 (ko) 2019-04-12
US20190040387A1 (en) 2019-02-07
HRP20201125T1 (hr) 2020-10-30
RU2724554C2 (ru) 2020-06-23
CN108026531A (zh) 2018-05-11
TW201718858A (zh) 2017-06-01
MY185390A (en) 2021-05-17
DK3351633T3 (da) 2020-08-03
JP6384845B2 (ja) 2018-09-05
JP2024038104A (ja) 2024-03-19
JPWO2017047707A1 (ja) 2018-07-05
PL3351633T3 (pl) 2020-11-02
CN113913426A (zh) 2022-01-11
AU2016324800A1 (en) 2018-04-05
JP6977998B2 (ja) 2021-12-08
US20180265866A1 (en) 2018-09-20
CN113913426B (zh) 2024-08-02
US20240368597A1 (en) 2024-11-07
SG11201802138TA (en) 2018-04-27
LT3351633T (lt) 2020-08-10
US10851373B2 (en) 2020-12-01
CY1123119T1 (el) 2021-10-29
CN108026531B (zh) 2021-09-14
PT3351633T (pt) 2020-07-29
EP3351633B1 (en) 2020-06-24
PH12018500568B1 (en) 2022-09-14
RS60493B1 (sr) 2020-08-31
AU2016324800B2 (en) 2022-02-24
US20210147839A1 (en) 2021-05-20
SI3351633T1 (sl) 2020-09-30
KR20180043244A (ko) 2018-04-27
NZ740562A (en) 2021-11-26
KR102473431B1 (ko) 2022-12-01
PH12018500568A1 (en) 2018-09-24
SMT202000379T1 (it) 2020-09-10
CN113930426A (zh) 2022-01-14
MX391304B (es) 2025-03-21
CO2018002557A2 (es) 2018-05-31

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