HRP20201125T1 - Antisens nukleinska kiselina - Google Patents

Antisens nukleinska kiselina Download PDF

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Publication number
HRP20201125T1
HRP20201125T1 HRP20201125TT HRP20201125T HRP20201125T1 HR P20201125 T1 HRP20201125 T1 HR P20201125T1 HR P20201125T T HRP20201125T T HR P20201125TT HR P20201125 T HRP20201125 T HR P20201125T HR P20201125 T1 HRP20201125 T1 HR P20201125T1
Authority
HR
Croatia
Prior art keywords
nucleotide sequence
pharmaceutically acceptable
acceptable salt
hydrate
antisense oligomer
Prior art date
Application number
HRP20201125TT
Other languages
English (en)
Croatian (hr)
Inventor
Yukiko Enya
Yuichiro Tone
Shin'ichi Takeda
Yoshitsugu Aoki
Original Assignee
Nippon Shinyaku Co., Ltd.
National Center Of Neurology And Psychiatry
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Nippon Shinyaku Co., Ltd., National Center Of Neurology And Psychiatry filed Critical Nippon Shinyaku Co., Ltd.
Publication of HRP20201125T1 publication Critical patent/HRP20201125T1/hr

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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)
HRP20201125TT 2015-09-15 2016-09-15 Antisens nukleinska kiselina HRP20201125T1 (hr)

Applications Claiming Priority (3)

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
EP16846578.9A EP3351633B1 (en) 2015-09-15 2016-09-15 Antisense nucleic acid

Publications (1)

Publication Number Publication Date
HRP20201125T1 true HRP20201125T1 (hr) 2020-10-30

Family

ID=58289325

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20201125TT HRP20201125T1 (hr) 2015-09-15 2016-09-15 Antisens nukleinska kiselina

Country Status (29)

Country Link
US (4) US10144931B2 (OSRAM)
EP (2) EP3778895A1 (OSRAM)
JP (4) JP6384845B2 (OSRAM)
KR (3) KR101968880B1 (OSRAM)
CN (3) CN113913426B (OSRAM)
AU (1) AU2016324800B2 (OSRAM)
CA (1) CA2996280C (OSRAM)
CO (1) CO2018002557A2 (OSRAM)
CY (1) CY1123119T1 (OSRAM)
DK (1) DK3351633T3 (OSRAM)
ES (1) ES2808049T3 (OSRAM)
HR (1) HRP20201125T1 (OSRAM)
HU (1) HUE050061T2 (OSRAM)
IL (1) IL258065B (OSRAM)
LT (1) LT3351633T (OSRAM)
MX (1) MX391304B (OSRAM)
MY (1) MY185390A (OSRAM)
PH (1) PH12018500568B1 (OSRAM)
PL (1) PL3351633T3 (OSRAM)
PT (1) PT3351633T (OSRAM)
RS (1) RS60493B1 (OSRAM)
RU (1) RU2724554C2 (OSRAM)
SG (1) SG11201802138TA (OSRAM)
SI (1) SI3351633T1 (OSRAM)
SM (1) SMT202000379T1 (OSRAM)
TW (1) TWI725990B (OSRAM)
UA (1) UA123359C2 (OSRAM)
WO (1) WO2017047707A1 (OSRAM)
ZA (1) ZA201801682B (OSRAM)

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RU2724554C2 (ru) * 2015-09-15 2020-06-23 Ниппон Синяку Ко., Лтд. Антисмысловая нуклеиновая кислота
JP7511326B2 (ja) 2015-10-09 2024-07-05 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物およびその方法
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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AU2020263487A1 (en) 2019-04-25 2021-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
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
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
MX2024000489A (es) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Complejos dirigidos al musculo y formulaciones para el tratamiento de las distrofinopatías.
US20250066396A1 (en) 2021-12-27 2025-02-27 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
US20250195667A1 (en) 2022-03-17 2025-06-19 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
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UA121117C2 (uk) 2014-06-17 2020-04-10 Ніппон Шин'Яку Ко., Лтд. Антисенсова нуклеїнова кислота
RU2724554C2 (ru) * 2015-09-15 2020-06-23 Ниппон Синяку Ко., Лтд. Антисмысловая нуклеиновая кислота

Also Published As

Publication number Publication date
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
CN108026531B (zh) 2021-09-14
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
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 (OSRAM) 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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