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BR112014026285A8 - oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares - Google Patents

oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares

Info

Publication number
BR112014026285A8
BR112014026285A8 BR112014026285A BR112014026285A BR112014026285A8 BR 112014026285 A8 BR112014026285 A8 BR 112014026285A8 BR 112014026285 A BR112014026285 A BR 112014026285A BR 112014026285 A BR112014026285 A BR 112014026285A BR 112014026285 A8 BR112014026285 A8 BR 112014026285A8
Authority
BR
Brazil
Prior art keywords
rna
treatment
improved characteristics
neuromuscular disorders
modulating oligonucleotides
Prior art date
Application number
BR112014026285A
Other languages
English (en)
Other versions
BR112014026285B1 (pt
BR112014026285A2 (pt
Inventor
Christian De Visser Peter
Allegonda Maria Mulders Susan
Original Assignee
Biomarin Tech Bv
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 Biomarin Tech Bv filed Critical Biomarin Tech Bv
Publication of BR112014026285A2 publication Critical patent/BR112014026285A2/pt
Publication of BR112014026285A8 publication Critical patent/BR112014026285A8/pt
Publication of BR112014026285B1 publication Critical patent/BR112014026285B1/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
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
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  • Neurosurgery (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
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  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112014026285-3A 2012-04-23 2013-04-23 Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares BR112014026285B1 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261636914P 2012-04-23 2012-04-23
EP12165139 2012-04-23
US61/636,914 2012-04-23
EP12165139.2 2012-04-23
PCT/NL2013/050306 WO2013162363A1 (en) 2012-04-23 2013-04-23 RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders

Publications (3)

Publication Number Publication Date
BR112014026285A2 BR112014026285A2 (pt) 2017-06-27
BR112014026285A8 true BR112014026285A8 (pt) 2018-01-16
BR112014026285B1 BR112014026285B1 (pt) 2021-08-31

Family

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BR112014026285-3A BR112014026285B1 (pt) 2012-04-23 2013-04-23 Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares

Country Status (19)

Country Link
US (4) US9745576B2 (pt)
EP (4) EP4400169A2 (pt)
JP (4) JP6174678B2 (pt)
CN (2) CN110025628B (pt)
AU (1) AU2013253074C1 (pt)
BR (1) BR112014026285B1 (pt)
CA (2) CA3103101C (pt)
DK (1) DK2841578T3 (pt)
ES (2) ES2983421T3 (pt)
HK (1) HK1207118A1 (pt)
HR (1) HRP20171254T1 (pt)
HU (1) HUE033431T2 (pt)
LT (1) LT2841578T (pt)
NZ (1) NZ700561A (pt)
PL (1) PL2841578T3 (pt)
PT (1) PT2841578T (pt)
RS (1) RS56319B1 (pt)
SI (1) SI2841578T1 (pt)
WO (1) WO2013162363A1 (pt)

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