BR112014009066B8 - molécula de ácido nucleico, método para a sintetização de uma molécula de dna de triciclo-fosforotioato e composição farmacêutica - Google Patents

molécula de ácido nucleico, método para a sintetização de uma molécula de dna de triciclo-fosforotioato e composição farmacêutica

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Publication number
BR112014009066B8
BR112014009066B8 BR112014009066A BR112014009066A BR112014009066B8 BR 112014009066 B8 BR112014009066 B8 BR 112014009066B8 BR 112014009066 A BR112014009066 A BR 112014009066A BR 112014009066 A BR112014009066 A BR 112014009066A BR 112014009066 B8 BR112014009066 B8 BR 112014009066B8
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Brazil
Prior art keywords
nucleic acid
tricyclic
synthesizing
pharmaceutical composition
acid molecule
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BR112014009066A
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English (en)
Other versions
BR112014009066A8 (pt
BR112014009066B1 (pt
BR112014009066A2 (pt
Inventor
Leumann Christian
Garcia Luis
Voit Thomas
Original Assignee
Association Inst De Myologie
Centre Nat Rech Scient
Inst Nat Sante Rech Med
Univ Pierre Et Marie Curie Paris 6
Univ Bern
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Application filed by Association Inst De Myologie, Centre Nat Rech Scient, Inst Nat Sante Rech Med, Univ Pierre Et Marie Curie Paris 6, Univ Bern filed Critical Association Inst De Myologie
Publication of BR112014009066A2 publication Critical patent/BR112014009066A2/pt
Publication of BR112014009066A8 publication Critical patent/BR112014009066A8/pt
Publication of BR112014009066B1 publication Critical patent/BR112014009066B1/pt
Publication of BR112014009066B8 publication Critical patent/BR112014009066B8/pt

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

dna triciclo-fosforotioato. a presente invenção refere-se a uma molécula de ácido nucleico que contém uma sequência de nucleosídeos tricíclicos unidos por meio de ligação de fosforotioato de internucleosídeo. a invenção também se refere a oligonucleotídeos antisense sintéticos e a métodos que empregam os mesmo.
BR112014009066A 2011-10-13 2012-10-12 molécula de ácido nucleico, método para a sintetização de uma molécula de dna de triciclo-fosforotioato e composição farmacêutica BR112014009066B8 (pt)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201161546942P 2011-10-13 2011-10-13
US61/546,942 2011-10-13
EP11185129.1A EP2581448B1 (en) 2011-10-13 2011-10-13 Tricyclo-phosphorothioate DNA
EP11185129.1 2011-10-13
CA2,776,651 2012-04-27
CA2776651A CA2776651C (en) 2011-10-13 2012-04-27 Tricyclo-phosphorothioate dna
PCT/EP2012/070349 WO2013053928A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna

Publications (4)

Publication Number Publication Date
BR112014009066A2 BR112014009066A2 (pt) 2017-04-18
BR112014009066A8 BR112014009066A8 (pt) 2018-01-09
BR112014009066B1 BR112014009066B1 (pt) 2020-08-25
BR112014009066B8 true BR112014009066B8 (pt) 2021-02-23

Family

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BR112014009066A BR112014009066B8 (pt) 2011-10-13 2012-10-12 molécula de ácido nucleico, método para a sintetização de uma molécula de dna de triciclo-fosforotioato e composição farmacêutica

Country Status (15)

Country Link
US (1) US9738891B2 (pt)
EP (2) EP2581448B1 (pt)
JP (1) JP6181653B2 (pt)
CN (1) CN104245935B (pt)
AU (1) AU2012322903B2 (pt)
BR (1) BR112014009066B8 (pt)
CA (2) CA2776651C (pt)
DK (1) DK2581448T3 (pt)
ES (2) ES2535654T3 (pt)
HK (1) HK1186205A1 (pt)
IL (1) IL231983B (pt)
IN (1) IN2014DN03463A (pt)
PL (1) PL2581448T3 (pt)
PT (1) PT2581448E (pt)
WO (1) WO2013053928A1 (pt)

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Publication number Publication date
CN104245935A (zh) 2014-12-24
IL231983A0 (en) 2014-05-28
AU2012322903A1 (en) 2014-05-29
JP2015501144A (ja) 2015-01-15
HK1186205A1 (en) 2014-03-07
BR112014009066A8 (pt) 2018-01-09
US9738891B2 (en) 2017-08-22
WO2013053928A1 (en) 2013-04-18
CN104245935B (zh) 2018-05-08
CA2776651A1 (en) 2013-04-13
BR112014009066B1 (pt) 2020-08-25
IN2014DN03463A (pt) 2015-06-05
EP2766479A1 (en) 2014-08-20
PL2581448T3 (pl) 2015-08-31
ES2651216T3 (es) 2018-01-25
PT2581448E (pt) 2015-05-21
JP6181653B2 (ja) 2017-08-16
CA2776651C (en) 2021-06-08
BR112014009066A2 (pt) 2017-04-18
DK2581448T3 (en) 2015-04-27
US20140296323A1 (en) 2014-10-02
ES2535654T3 (es) 2015-05-13
EP2581448B1 (en) 2015-01-28
IL231983B (en) 2018-04-30
AU2012322903B2 (en) 2017-09-14
CA2851970A1 (en) 2013-04-18
EP2766479B1 (en) 2017-09-13
EP2581448A1 (en) 2013-04-17

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