PH12018500568B1 - Antisense nucleic acid - Google Patents

Antisense nucleic acid

Info

Publication number
PH12018500568B1
PH12018500568B1 PH1/2018/500568A PH12018500568A PH12018500568B1 PH 12018500568 B1 PH12018500568 B1 PH 12018500568B1 PH 12018500568 A PH12018500568 A PH 12018500568A PH 12018500568 B1 PH12018500568 B1 PH 12018500568B1
Authority
PH
Philippines
Prior art keywords
nucleic acid
antisense nucleic
exon
oligomer
provides
Prior art date
Application number
PH1/2018/500568A
Other languages
English (en)
Other versions
PH12018500568A1 (en
Inventor
Yoshitsugu Aoki
Yukiko Enya
Shin'ichi Takeda
Yuichiro TONE
Original Assignee
Nat Center Neurology & Psychiatry
Nippon Shinyaku Co Ltd
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 Nat Center Neurology & Psychiatry, Nippon Shinyaku Co Ltd filed Critical Nat Center Neurology & Psychiatry
Publication of PH12018500568A1 publication Critical patent/PH12018500568A1/en
Publication of PH12018500568B1 publication Critical patent/PH12018500568B1/en

Links

Classifications

    • 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
    • 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
    • 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

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)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (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)
PH1/2018/500568A 2015-09-15 2016-09-15 Antisense nucleic acid PH12018500568B1 (en)

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 (2)

Publication Number Publication Date
PH12018500568A1 PH12018500568A1 (en) 2018-09-24
PH12018500568B1 true PH12018500568B1 (en) 2022-09-14

Family

ID=58289325

Family Applications (1)

Application Number Title Priority Date Filing Date
PH1/2018/500568A PH12018500568B1 (en) 2015-09-15 2016-09-15 Antisense nucleic acid

Country Status (29)

Country Link
US (4) US10144931B2 (enExample)
EP (2) EP3778895A1 (enExample)
JP (4) JP6384845B2 (enExample)
KR (3) KR102473431B1 (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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US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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US20250195667A1 (en) 2022-03-17 2025-06-19 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
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Also Published As

Publication number Publication date
KR20190040098A (ko) 2019-04-16
AU2016324800B2 (en) 2022-02-24
RU2018113276A (ru) 2019-10-16
CO2018002557A2 (es) 2018-05-31
DK3351633T3 (da) 2020-08-03
KR20220053048A (ko) 2022-04-28
CY1123119T1 (el) 2021-10-29
UA123359C2 (uk) 2021-03-24
KR102473431B1 (ko) 2022-12-01
PH12018500568A1 (en) 2018-09-24
AU2016324800A1 (en) 2018-04-05
CA2996280A1 (en) 2017-03-23
EP3351633B1 (en) 2020-06-24
JP2024038104A (ja) 2024-03-19
KR101968880B1 (ko) 2019-04-12
CN113930426A (zh) 2022-01-14
CA2996280C (en) 2024-05-07
ZA201801682B (en) 2020-07-29
KR20180043244A (ko) 2018-04-27
JP2018183178A (ja) 2018-11-22
MY185390A (en) 2021-05-17
US10144931B2 (en) 2018-12-04
RU2724554C2 (ru) 2020-06-23
CN113913426A (zh) 2022-01-11
HUE050061T2 (hu) 2020-12-28
CN108026531B (zh) 2021-09-14
IL258065B (en) 2022-02-01
TW201718858A (zh) 2017-06-01
US20180265866A1 (en) 2018-09-20
JP6977998B2 (ja) 2021-12-08
EP3778895A1 (en) 2021-02-17
ES2808049T3 (es) 2021-02-25
SI3351633T1 (sl) 2020-09-30
CN113913426B (zh) 2024-08-02
MX391304B (es) 2025-03-21
US10851373B2 (en) 2020-12-01
LT3351633T (lt) 2020-08-10
EP3351633A1 (en) 2018-07-25
RU2018113276A3 (enExample) 2020-02-06
US20240368597A1 (en) 2024-11-07
SG11201802138TA (en) 2018-04-27
IL258065A (en) 2018-05-31
JP2022033738A (ja) 2022-03-02
TWI725990B (zh) 2021-05-01
WO2017047707A1 (ja) 2017-03-23
NZ740562A (en) 2021-11-26
BR112018004970A2 (pt) 2018-10-09
HRP20201125T1 (hr) 2020-10-30
US11981894B2 (en) 2024-05-14
PL3351633T3 (pl) 2020-11-02
MX2018002955A (es) 2018-05-02
SMT202000379T1 (it) 2020-09-10
PT3351633T (pt) 2020-07-29
JP6384845B2 (ja) 2018-09-05
US20190040387A1 (en) 2019-02-07
CN108026531A (zh) 2018-05-11
US20210147839A1 (en) 2021-05-20
EP3351633A4 (en) 2019-09-04
JPWO2017047707A1 (ja) 2018-07-05
RS60493B1 (sr) 2020-08-31

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