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MX2018002955A - Acido nucleico antisentido. - Google Patents

Acido nucleico antisentido.

Info

Publication number
MX2018002955A
MX2018002955A MX2018002955A MX2018002955A MX2018002955A MX 2018002955 A MX2018002955 A MX 2018002955A MX 2018002955 A MX2018002955 A MX 2018002955A MX 2018002955 A MX2018002955 A MX 2018002955A MX 2018002955 A MX2018002955 A MX 2018002955A
Authority
MX
Mexico
Prior art keywords
nucleic acid
antisense nucleic
exon
omission
oligomer
Prior art date
Application number
MX2018002955A
Other languages
English (en)
Other versions
MX391304B (es
Inventor
Enya Yukiko
Tone Yuichiro
Takeda Shin'ichi
AOKI Yoshitsugu
Original Assignee
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 Nippon Shinyaku Co Ltd filed Critical Nippon Shinyaku Co Ltd
Publication of MX2018002955A publication Critical patent/MX2018002955A/es
Publication of MX391304B publication Critical patent/MX391304B/es

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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

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)

Abstract

La presente invención proporciona un oligómero que permite la omisión del exón 45 en el gen de la distrofina humana.
MX2018002955A 2015-09-15 2016-09-15 Acido nucleico antisentido. MX391304B (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 (2)

Publication Number Publication Date
MX2018002955A true MX2018002955A (es) 2018-05-02
MX391304B MX391304B (es) 2025-03-21

Family

ID=58289325

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018002955A MX391304B (es) 2015-09-15 2016-09-15 Acido nucleico antisentido.

Country Status (29)

Country Link
US (4) US10144931B2 (es)
EP (2) EP3351633B1 (es)
JP (4) JP6384845B2 (es)
KR (3) KR102473431B1 (es)
CN (3) CN113930426A (es)
AU (1) AU2016324800B2 (es)
CA (1) CA2996280C (es)
CO (1) CO2018002557A2 (es)
CY (1) CY1123119T1 (es)
DK (1) DK3351633T3 (es)
ES (1) ES2808049T3 (es)
HR (1) HRP20201125T1 (es)
HU (1) HUE050061T2 (es)
IL (1) IL258065B (es)
LT (1) LT3351633T (es)
MX (1) MX391304B (es)
MY (1) MY185390A (es)
PH (1) PH12018500568B1 (es)
PL (1) PL3351633T3 (es)
PT (1) PT3351633T (es)
RS (1) RS60493B1 (es)
RU (1) RU2724554C2 (es)
SG (1) SG11201802138TA (es)
SI (1) SI3351633T1 (es)
SM (1) SMT202000379T1 (es)
TW (1) TWI725990B (es)
UA (1) UA123359C2 (es)
WO (1) WO2017047707A1 (es)
ZA (1) ZA201801682B (es)

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WO2017047707A1 (ja) * 2015-09-15 2017-03-23 日本新薬株式会社 アンチセンス核酸
MA46427A (fr) 2015-10-09 2018-08-15 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2020028864A1 (en) 2018-08-02 2020-02-06 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
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
US12497615B2 (en) 2019-04-25 2025-12-16 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
AU2022267324A1 (en) 2021-04-30 2023-12-14 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
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations 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
CA3244382A1 (en) 2021-12-27 2025-02-26 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
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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Also Published As

Publication number Publication date
CO2018002557A2 (es) 2018-05-31
US20240368597A1 (en) 2024-11-07
SMT202000379T1 (it) 2020-09-10
RU2724554C2 (ru) 2020-06-23
US20180265866A1 (en) 2018-09-20
JP2024038104A (ja) 2024-03-19
IL258065A (en) 2018-05-31
KR20220053048A (ko) 2022-04-28
JP2018183178A (ja) 2018-11-22
DK3351633T3 (da) 2020-08-03
WO2017047707A1 (ja) 2017-03-23
UA123359C2 (uk) 2021-03-24
CN108026531A (zh) 2018-05-11
ZA201801682B (en) 2020-07-29
PT3351633T (pt) 2020-07-29
NZ740562A (en) 2021-11-26
KR102473431B1 (ko) 2022-12-01
US10851373B2 (en) 2020-12-01
CN113913426A (zh) 2022-01-11
EP3351633A1 (en) 2018-07-25
TWI725990B (zh) 2021-05-01
KR20190040098A (ko) 2019-04-16
CN113913426B (zh) 2024-08-02
BR112018004970A2 (pt) 2018-10-09
CN108026531B (zh) 2021-09-14
IL258065B (en) 2022-02-01
RS60493B1 (sr) 2020-08-31
PL3351633T3 (pl) 2020-11-02
LT3351633T (lt) 2020-08-10
JPWO2017047707A1 (ja) 2018-07-05
MX391304B (es) 2025-03-21
MY185390A (en) 2021-05-17
TW201718858A (zh) 2017-06-01
CN113930426A (zh) 2022-01-14
RU2018113276A3 (es) 2020-02-06
EP3778895A1 (en) 2021-02-17
KR20180043244A (ko) 2018-04-27
JP2022033738A (ja) 2022-03-02
JP6384845B2 (ja) 2018-09-05
US20190040387A1 (en) 2019-02-07
US11981894B2 (en) 2024-05-14
SG11201802138TA (en) 2018-04-27
KR101968880B1 (ko) 2019-04-12
SI3351633T1 (sl) 2020-09-30
AU2016324800A1 (en) 2018-04-05
CA2996280A1 (en) 2017-03-23
HRP20201125T1 (hr) 2020-10-30
ES2808049T3 (es) 2021-02-25
EP3351633B1 (en) 2020-06-24
EP3351633A4 (en) 2019-09-04
HUE050061T2 (hu) 2020-12-28
AU2016324800B2 (en) 2022-02-24
JP6977998B2 (ja) 2021-12-08
PH12018500568B1 (en) 2022-09-14
US20210147839A1 (en) 2021-05-20
PH12018500568A1 (en) 2018-09-24
CA2996280C (en) 2024-05-07
CY1123119T1 (el) 2021-10-29
US10144931B2 (en) 2018-12-04
RU2018113276A (ru) 2019-10-16

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