JP6384845B2 - アンチセンス核酸 - Google Patents

アンチセンス核酸 Download PDF

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Publication number
JP6384845B2
JP6384845B2 JP2017539975A JP2017539975A JP6384845B2 JP 6384845 B2 JP6384845 B2 JP 6384845B2 JP 2017539975 A JP2017539975 A JP 2017539975A JP 2017539975 A JP2017539975 A JP 2017539975A JP 6384845 B2 JP6384845 B2 JP 6384845B2
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JP
Japan
Prior art keywords
pharmaceutically acceptable
hydrate
acceptable salt
antisense oligomer
oligomer
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Active
Application number
JP2017539975A
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English (en)
Japanese (ja)
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JPWO2017047707A1 (ja
Inventor
由輝子 塩谷
由輝子 塩谷
悠一郎 戸根
悠一郎 戸根
武田　伸一
伸一 武田
吉嗣 青木
吉嗣 青木
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
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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Application filed by Nippon Shinyaku Co Ltd, National Center of Neurology and Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of JPWO2017047707A1 publication Critical patent/JPWO2017047707A1/ja
Priority to JP2018144099A priority Critical patent/JP6977998B2/ja
Application granted granted Critical
Publication of JP6384845B2 publication Critical patent/JP6384845B2/ja
Priority to JP2021179353A priority patent/JP2022033738A/ja
Priority to JP2023218764A priority patent/JP2024038104A/ja
Priority to JP2025197087A priority patent/JP2026032058A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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

Landscapes

  • 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)
JP2017539975A 2015-09-15 2016-09-15 アンチセンス核酸 Active JP6384845B2 (ja)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2018144099A JP6977998B2 (ja) 2015-09-15 2018-07-31 アンチセンス核酸
JP2021179353A JP2022033738A (ja) 2015-09-15 2021-11-02 アンチセンス核酸
JP2023218764A JP2024038104A (ja) 2015-09-15 2023-12-26 アンチセンス核酸
JP2025197087A JP2026032058A (ja) 2015-09-15 2025-11-18 アンチセンス核酸

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2015182145 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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2018144099A Division JP6977998B2 (ja) 2015-09-15 2018-07-31 アンチセンス核酸

Publications (2)

Publication Number Publication Date
JPWO2017047707A1 JPWO2017047707A1 (ja) 2018-07-05
JP6384845B2 true JP6384845B2 (ja) 2018-09-05

Family

ID=58289325

Family Applications (5)

Application Number Title Priority Date Filing Date
JP2017539975A Active JP6384845B2 (ja) 2015-09-15 2016-09-15 アンチセンス核酸
JP2018144099A Active JP6977998B2 (ja) 2015-09-15 2018-07-31 アンチセンス核酸
JP2021179353A Withdrawn JP2022033738A (ja) 2015-09-15 2021-11-02 アンチセンス核酸
JP2023218764A Pending JP2024038104A (ja) 2015-09-15 2023-12-26 アンチセンス核酸
JP2025197087A Pending JP2026032058A (ja) 2015-09-15 2025-11-18 アンチセンス核酸

Family Applications After (4)

Application Number Title Priority Date Filing Date
JP2018144099A Active JP6977998B2 (ja) 2015-09-15 2018-07-31 アンチセンス核酸
JP2021179353A Withdrawn JP2022033738A (ja) 2015-09-15 2021-11-02 アンチセンス核酸
JP2023218764A Pending JP2024038104A (ja) 2015-09-15 2023-12-26 アンチセンス核酸
JP2025197087A Pending JP2026032058A (ja) 2015-09-15 2025-11-18 アンチセンス核酸

Country Status (29)

Country Link
US (4) US10144931B2 (enExample)
EP (2) EP3778895A1 (enExample)
JP (5) JP6384845B2 (enExample)
KR (3) KR20220053048A (enExample)
CN (3) CN113913426B (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)
HR (1) HRP20201125T1 (enExample)
HU (1) HUE050061T2 (enExample)
IL (1) IL258065B (enExample)
LT (1) LT3351633T (enExample)
MX (1) MX391304B (enExample)
MY (1) MY185390A (enExample)
PH (1) PH12018500568B1 (enExample)
PL (1) PL3351633T3 (enExample)
PT (1) PT3351633T (enExample)
RS (1) RS60493B1 (enExample)
RU (1) RU2724554C2 (enExample)
SG (1) SG11201802138TA (enExample)
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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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
GB201821269D0 (en) * 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
IL293997A (en) * 2019-12-19 2022-08-01 Nippon Shinyaku Co Ltd Antistrand nucleic acids that allow exon skipping
IL295967A (en) * 2020-02-28 2022-10-01 Nippon Shinyaku Co Ltd Antisense nucleic acids for splicing on exon 51
IL307937A (en) 2021-04-30 2023-12-01 Sarepta Therapeutics Inc Treatment methods for muscular dystrophy
WO2022270585A1 (ja) * 2021-06-23 2022-12-29 日本新薬株式会社 アンチセンスオリゴマーの組み合わせ
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
EP4458833A4 (en) 2021-12-27 2025-12-31 Nippon Shinyaku Co Ltd PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
CN119013401A (zh) 2022-03-17 2024-11-22 萨勒普塔医疗公司 二氨基磷酸酯吗啉代寡聚物缀合物
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Also Published As

Publication number Publication date
AU2016324800B2 (en) 2022-02-24
SMT202000379T1 (it) 2020-09-10
PL3351633T3 (pl) 2020-11-02
LT3351633T (lt) 2020-08-10
CN113913426B (zh) 2024-08-02
JP2024038104A (ja) 2024-03-19
JPWO2017047707A1 (ja) 2018-07-05
CA2996280A1 (en) 2017-03-23
EP3351633A1 (en) 2018-07-25
CN113913426A (zh) 2022-01-11
RU2724554C2 (ru) 2020-06-23
CN113930426A (zh) 2022-01-14
KR102473431B1 (ko) 2022-12-01
MX2018002955A (es) 2018-05-02
RS60493B1 (sr) 2020-08-31
US10144931B2 (en) 2018-12-04
MY185390A (en) 2021-05-17
EP3351633A4 (en) 2019-09-04
PH12018500568A1 (en) 2018-09-24
SG11201802138TA (en) 2018-04-27
WO2017047707A1 (ja) 2017-03-23
PT3351633T (pt) 2020-07-29
JP2022033738A (ja) 2022-03-02
PH12018500568B1 (en) 2022-09-14
US10851373B2 (en) 2020-12-01
RU2018113276A3 (enExample) 2020-02-06
DK3351633T3 (da) 2020-08-03
IL258065A (en) 2018-05-31
RU2018113276A (ru) 2019-10-16
CN108026531B (zh) 2021-09-14
IL258065B (en) 2022-02-01
CA2996280C (en) 2024-05-07
US20190040387A1 (en) 2019-02-07
TWI725990B (zh) 2021-05-01
CN108026531A (zh) 2018-05-11
US20240368597A1 (en) 2024-11-07
JP6977998B2 (ja) 2021-12-08
AU2016324800A1 (en) 2018-04-05
MX391304B (es) 2025-03-21
BR112018004970A2 (pt) 2018-10-09
HRP20201125T1 (hr) 2020-10-30
US11981894B2 (en) 2024-05-14
JP2026032058A (ja) 2026-02-25
UA123359C2 (uk) 2021-03-24
ZA201801682B (en) 2020-07-29
EP3351633B1 (en) 2020-06-24
KR101968880B1 (ko) 2019-04-12
US20210147839A1 (en) 2021-05-20
JP2018183178A (ja) 2018-11-22
NZ740562A (en) 2021-11-26
KR20220053048A (ko) 2022-04-28
KR20190040098A (ko) 2019-04-16
SI3351633T1 (sl) 2020-09-30
HUE050061T2 (hu) 2020-12-28
CO2018002557A2 (es) 2018-05-31
CY1123119T1 (el) 2021-10-29
TW201718858A (zh) 2017-06-01
ES2808049T3 (es) 2021-02-25
US20180265866A1 (en) 2018-09-20
EP3778895A1 (en) 2021-02-17
KR20180043244A (ko) 2018-04-27

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