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CO2018002557A2 - Ácido nucléico antisentido para tratar la distrofia muscular de duchenne - Google Patents

Ácido nucléico antisentido para tratar la distrofia muscular de duchenne

Info

Publication number
CO2018002557A2
CO2018002557A2 CONC2018/0002557A CO2018002557A CO2018002557A2 CO 2018002557 A2 CO2018002557 A2 CO 2018002557A2 CO 2018002557 A CO2018002557 A CO 2018002557A CO 2018002557 A2 CO2018002557 A2 CO 2018002557A2
Authority
CO
Colombia
Prior art keywords
muscular dystrophy
duchenne muscular
nucleic acid
exon
acid antisense
Prior art date
Application number
CONC2018/0002557A
Other languages
English (en)
Inventor
Yoshitsugu Aoki
Yukiko Enya
Yuichiro TONE
Shin'ichi Takeda
Original Assignee
Nippon Shinyaku Co Ltd
Nat Center Neurology & 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, Nat Center Neurology & Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of CO2018002557A2 publication Critical patent/CO2018002557A2/es

Links

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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)

Abstract

Resumen La presente invención se refiere a un oligómero antisentido que hace posible omitir el exón 45 del gen de distrofina humana y a una composición farmacéutica que comprende dicho oligómero. Este se caracteriza como un oligómenro antisentido obtenido por unión de oligómeros que se dirigen a dos posiciones diferentes del exón 45 del gen de la distrofina humana, lo que hace posible inducir la omisión de dicho exón. Esta invención puede aplicarse en tratamientos de enfermedades musculares progresivas tales como la distrofia muscular de Duchenne
CONC2018/0002557A 2015-09-15 2018-03-09 Ácido nucléico antisentido para tratar la distrofia muscular de duchenne CO2018002557A2 (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
CO2018002557A2 true CO2018002557A2 (es) 2018-05-31

Family

ID=58289325

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2018/0002557A CO2018002557A2 (es) 2015-09-15 2018-03-09 Ácido nucléico antisentido para tratar la distrofia muscular de duchenne

Country Status (29)

Country Link
US (4) US10144931B2 (es)
EP (2) EP3778895A1 (es)
JP (4) JP6384845B2 (es)
KR (3) KR101968880B1 (es)
CN (3) CN108026531B (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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Also Published As

Publication number Publication date
EP3351633B1 (en) 2020-06-24
RS60493B1 (sr) 2020-08-31
HUE050061T2 (hu) 2020-12-28
JPWO2017047707A1 (ja) 2018-07-05
PH12018500568B1 (en) 2022-09-14
US11981894B2 (en) 2024-05-14
SG11201802138TA (en) 2018-04-27
PL3351633T3 (pl) 2020-11-02
RU2018113276A3 (es) 2020-02-06
CA2996280A1 (en) 2017-03-23
NZ740562A (en) 2021-11-26
BR112018004970A2 (pt) 2018-10-09
JP6384845B2 (ja) 2018-09-05
CN113913426B (zh) 2024-08-02
MX2018002955A (es) 2018-05-02
EP3351633A1 (en) 2018-07-25
JP2024038104A (ja) 2024-03-19
CN113913426A (zh) 2022-01-11
PH12018500568A1 (en) 2018-09-24
US10851373B2 (en) 2020-12-01
AU2016324800B2 (en) 2022-02-24
ES2808049T3 (es) 2021-02-25
IL258065B (en) 2022-02-01
WO2017047707A1 (ja) 2017-03-23
KR101968880B1 (ko) 2019-04-12
DK3351633T3 (da) 2020-08-03
TW201718858A (zh) 2017-06-01
US10144931B2 (en) 2018-12-04
US20180265866A1 (en) 2018-09-20
KR20220053048A (ko) 2022-04-28
JP2022033738A (ja) 2022-03-02
CN113930426A (zh) 2022-01-14
SMT202000379T1 (it) 2020-09-10
UA123359C2 (uk) 2021-03-24
KR20190040098A (ko) 2019-04-16
JP6977998B2 (ja) 2021-12-08
HRP20201125T1 (hr) 2020-10-30
IL258065A (en) 2018-05-31
CA2996280C (en) 2024-05-07
MY185390A (en) 2021-05-17
EP3778895A1 (en) 2021-02-17
CY1123119T1 (el) 2021-10-29
JP2018183178A (ja) 2018-11-22
LT3351633T (lt) 2020-08-10
CN108026531B (zh) 2021-09-14
TWI725990B (zh) 2021-05-01
MX391304B (es) 2025-03-21
US20210147839A1 (en) 2021-05-20
RU2018113276A (ru) 2019-10-16
KR20180043244A (ko) 2018-04-27
RU2724554C2 (ru) 2020-06-23
SI3351633T1 (sl) 2020-09-30
AU2016324800A1 (en) 2018-04-05
US20190040387A1 (en) 2019-02-07
US20240368597A1 (en) 2024-11-07
PT3351633T (pt) 2020-07-29
EP3351633A4 (en) 2019-09-04
CN108026531A (zh) 2018-05-11
ZA201801682B (en) 2020-07-29
KR102473431B1 (ko) 2022-12-01

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