ES2808750T3 - Compuestos oligonucleotídicos dirigidos a ARNm de huntingtina - Google Patents

Compuestos oligonucleotídicos dirigidos a ARNm de huntingtina Download PDF

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Publication number
ES2808750T3
ES2808750T3 ES16716412T ES16716412T ES2808750T3 ES 2808750 T3 ES2808750 T3 ES 2808750T3 ES 16716412 T ES16716412 T ES 16716412T ES 16716412 T ES16716412 T ES 16716412T ES 2808750 T3 ES2808750 T3 ES 2808750T3
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sirna
rna
gene
target
mrna
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Spanish (es)
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Anastasia Khvorova
Neil Aronin
Julia Alterman
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University of Massachusetts Amherst
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University of Massachusetts Amherst
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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3517—Marker; Tag
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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
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/52—Physical structure branched
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Chemical Kinetics & Catalysis (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
ES16716412T 2015-04-03 2016-04-01 Compuestos oligonucleotídicos dirigidos a ARNm de huntingtina Active ES2808750T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562142731P 2015-04-03 2015-04-03
US201662289274P 2016-01-31 2016-01-31
PCT/US2016/025722 WO2016161374A1 (en) 2015-04-03 2016-04-01 Oligonucleotide compounds for targeting huntingtin mrna

Publications (1)

Publication Number Publication Date
ES2808750T3 true ES2808750T3 (es) 2021-03-01

Family

ID=55752785

Family Applications (1)

Application Number Title Priority Date Filing Date
ES16716412T Active ES2808750T3 (es) 2015-04-03 2016-04-01 Compuestos oligonucleotídicos dirigidos a ARNm de huntingtina

Country Status (18)

Country Link
US (5) US9809817B2 (OSRAM)
EP (2) EP3766973A1 (OSRAM)
JP (5) JP7009356B2 (OSRAM)
CN (2) CN116004624A (OSRAM)
AU (4) AU2016244027B2 (OSRAM)
CA (1) CA2980337A1 (OSRAM)
CY (1) CY1123289T1 (OSRAM)
DK (1) DK3277814T3 (OSRAM)
ES (1) ES2808750T3 (OSRAM)
HR (1) HRP20201200T1 (OSRAM)
HU (1) HUE050441T2 (OSRAM)
LT (1) LT3277814T (OSRAM)
PL (1) PL3277814T3 (OSRAM)
PT (1) PT3277814T (OSRAM)
RS (1) RS60762B1 (OSRAM)
SI (1) SI3277814T1 (OSRAM)
SM (1) SMT202000454T1 (OSRAM)
WO (1) WO2016161374A1 (OSRAM)

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