ES2808049T3 - Acido nucleico antisentido - Google Patents

Acido nucleico antisentido Download PDF

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Publication number
ES2808049T3
ES2808049T3 ES16846578T ES16846578T ES2808049T3 ES 2808049 T3 ES2808049 T3 ES 2808049T3 ES 16846578 T ES16846578 T ES 16846578T ES 16846578 T ES16846578 T ES 16846578T ES 2808049 T3 ES2808049 T3 ES 2808049T3
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ES
Spain
Prior art keywords
oligomer
nucleotide sequence
exon
pharmaceutically acceptable
hydrates
Prior art date
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Active
Application number
ES16846578T
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English (en)
Spanish (es)
Inventor
Yukiko Enya
Yuichiro TONE
Shin'ichi Takeda
Yoshitsugu Aoki
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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    • 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/313Phosphorodithioates
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • 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
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications

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  • 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)
ES16846578T 2015-09-15 2016-09-15 Acido nucleico antisentido Active ES2808049T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2015182145 2015-09-15
PCT/JP2016/077305 WO2017047707A1 (ja) 2015-09-15 2016-09-15 アンチセンス核酸

Publications (1)

Publication Number Publication Date
ES2808049T3 true ES2808049T3 (es) 2021-02-25

Family

ID=58289325

Family Applications (1)

Application Number Title Priority Date Filing Date
ES16846578T Active ES2808049T3 (es) 2015-09-15 2016-09-15 Acido nucleico antisentido

Country Status (29)

Country Link
US (4) US10144931B2 (enExample)
EP (2) EP3778895A1 (enExample)
JP (4) JP6384845B2 (enExample)
KR (3) KR20220053048A (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)
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)

Families Citing this family (17)

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PL3351633T3 (pl) * 2015-09-15 2020-11-02 Nippon Shinyaku Co., Ltd. Antysensowny kwas nukleinowy
CN108137492B (zh) 2015-10-09 2021-12-21 波涛生命科学有限公司 寡核苷酸组合物及其方法
CN112955153A (zh) 2018-08-02 2021-06-11 达因疗法公司 肌肉靶向复合物及其用于治疗肌养蛋白病的用途
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
CN112912499A (zh) 2018-08-02 2021-06-04 达因疗法公司 肌肉靶向复合物及其用于治疗面肩肱型肌营养不良的用途
GB201821269D0 (en) * 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
KR20240024176A (ko) * 2021-06-23 2024-02-23 니뽄 신야쿠 가부시키가이샤 안티센스 올리고머의 조합
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
IL309936A (en) 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of dystrophinopathy
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
EP4458833A1 (en) 2021-12-27 2024-11-06 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
EP4486386A1 (en) 2022-03-03 2025-01-08 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
IL315280A (en) 2022-03-17 2024-10-01 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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JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体,デバイス及び医薬組成物
JP2000325085A (ja) * 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
JP4836366B2 (ja) * 2000-08-25 2011-12-14 雅文 松尾 デュシェンヌ型筋ジストロフィー治療剤
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WO2004083432A1 (en) * 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
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Also Published As

Publication number Publication date
JP6384845B2 (ja) 2018-09-05
DK3351633T3 (da) 2020-08-03
BR112018004970A2 (pt) 2018-10-09
KR102473431B1 (ko) 2022-12-01
US11981894B2 (en) 2024-05-14
US10851373B2 (en) 2020-12-01
HRP20201125T1 (hr) 2020-10-30
KR101968880B1 (ko) 2019-04-12
JP2022033738A (ja) 2022-03-02
JP2018183178A (ja) 2018-11-22
JP2024038104A (ja) 2024-03-19
TW201718858A (zh) 2017-06-01
EP3778895A1 (en) 2021-02-17
EP3351633A1 (en) 2018-07-25
WO2017047707A1 (ja) 2017-03-23
PL3351633T3 (pl) 2020-11-02
SMT202000379T1 (it) 2020-09-10
KR20180043244A (ko) 2018-04-27
KR20220053048A (ko) 2022-04-28
CN108026531B (zh) 2021-09-14
IL258065B (en) 2022-02-01
UA123359C2 (uk) 2021-03-24
US20180265866A1 (en) 2018-09-20
JPWO2017047707A1 (ja) 2018-07-05
PH12018500568A1 (en) 2018-09-24
MX391304B (es) 2025-03-21
RU2018113276A3 (enExample) 2020-02-06
HUE050061T2 (hu) 2020-12-28
CN113913426B (zh) 2024-08-02
AU2016324800A1 (en) 2018-04-05
CN108026531A (zh) 2018-05-11
CY1123119T1 (el) 2021-10-29
CA2996280C (en) 2024-05-07
MX2018002955A (es) 2018-05-02
SI3351633T1 (sl) 2020-09-30
PT3351633T (pt) 2020-07-29
LT3351633T (lt) 2020-08-10
SG11201802138TA (en) 2018-04-27
US10144931B2 (en) 2018-12-04
MY185390A (en) 2021-05-17
JP6977998B2 (ja) 2021-12-08
RU2724554C2 (ru) 2020-06-23
EP3351633B1 (en) 2020-06-24
CA2996280A1 (en) 2017-03-23
CN113930426A (zh) 2022-01-14
US20240368597A1 (en) 2024-11-07
AU2016324800B2 (en) 2022-02-24
RU2018113276A (ru) 2019-10-16
RS60493B1 (sr) 2020-08-31
US20210147839A1 (en) 2021-05-20
TWI725990B (zh) 2021-05-01
EP3351633A4 (en) 2019-09-04
NZ740562A (en) 2021-11-26
KR20190040098A (ko) 2019-04-16
CO2018002557A2 (es) 2018-05-31
PH12018500568B1 (en) 2022-09-14
CN113913426A (zh) 2022-01-11
US20190040387A1 (en) 2019-02-07
ZA201801682B (en) 2020-07-29
IL258065A (en) 2018-05-31

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