BR112012011195B1 - Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica - Google Patents

Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica Download PDF

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Publication number
BR112012011195B1
BR112012011195B1 BR112012011195-7A BR112012011195A BR112012011195B1 BR 112012011195 B1 BR112012011195 B1 BR 112012011195B1 BR 112012011195 A BR112012011195 A BR 112012011195A BR 112012011195 B1 BR112012011195 B1 BR 112012011195B1
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Brazil
Prior art keywords
exon
skipping
antisense
antisense oligonucleotide
seq
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BR112012011195-7A
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English (en)
Portuguese (pt)
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BR112012011195A2 (pt
Inventor
Wilton Stephen
Fletcher Sue
Adams Abbie
Meloni Penny
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The University Of Western Australia
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Priority claimed from AU2009905549A external-priority patent/AU2009905549A0/en
Application filed by The University Of Western Australia filed Critical The University Of Western Australia
Priority to BR122023023194-5A priority Critical patent/BR122023023194A2/pt
Publication of BR112012011195A2 publication Critical patent/BR112012011195A2/pt
Publication of BR112012011195B1 publication Critical patent/BR112012011195B1/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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
    • C12N2310/3233—Morpholino-type ring
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Oncology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
BR112012011195-7A 2009-11-12 2010-11-12 Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica BR112012011195B1 (pt)

Priority Applications (1)

Application Number Priority Date Filing Date Title
BR122023023194-5A BR122023023194A2 (pt) 2009-11-12 2010-11-12 Oligonucleotídeo antissentido, composição e respectivo uso

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2009905549 2009-11-12
AU2009905549A AU2009905549A0 (en) 2009-11-12 Antisense Molecules and Methods for Treating Pathologies
PCT/AU2010/001520 WO2011057350A1 (en) 2009-11-12 2010-11-12 Antisense molecules and methods for treating pathologies

Publications (2)

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BR112012011195A2 BR112012011195A2 (pt) 2022-08-02
BR112012011195B1 true BR112012011195B1 (pt) 2024-02-15

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BR122023023194-5A BR122023023194A2 (pt) 2009-11-12 2010-11-12 Oligonucleotídeo antissentido, composição e respectivo uso
BR112012011195-7A BR112012011195B1 (pt) 2009-11-12 2010-11-12 Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica

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US (7) US8637483B2 (enExample)
EP (2) EP2499249B1 (enExample)
JP (6) JP5963678B2 (enExample)
KR (9) KR102000762B1 (enExample)
CN (2) CN105838714B (enExample)
AU (5) AU2010317599B2 (enExample)
BR (2) BR122023023194A2 (enExample)
CA (1) CA2780563C (enExample)
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DK (1) DK2499249T3 (enExample)
ES (1) ES2693459T3 (enExample)
HR (1) HRP20181824T1 (enExample)
HU (1) HUE040445T2 (enExample)
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NZ (2) NZ626359A (enExample)
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PT (1) PT2499249T (enExample)
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SM (1) SMT201800579T1 (enExample)
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