JP6679476B2 - Ｃ９ｏｒｆ７２発現を調節するための組成物 - Google Patents

Ｃ９ｏｒｆ７２発現を調節するための組成物 Download PDF

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JP6679476B2
JP6679476B2 JP2016521622A JP2016521622A JP6679476B2 JP 6679476 B2 JP6679476 B2 JP 6679476B2 JP 2016521622 A JP2016521622 A JP 2016521622A JP 2016521622 A JP2016521622 A JP 2016521622A JP 6679476 B2 JP6679476 B2 JP 6679476B2
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certain embodiments
c9orf72
modified oligonucleotide
modified
antisense
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JP2016533717A5 (https=
JP2016533717A (ja
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フレアー，スーザン・エム
リゴ，フランク
シング，プリヤム
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • 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
    • A61P25/16—Anti-Parkinson drugs
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--

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JP2016521622A 2013-10-11 2014-10-11 Ｃ９ｏｒｆ７２発現を調節するための組成物 Expired - Fee Related JP6679476B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201361890108P 2013-10-11 2013-10-11
US61/890,108 2013-10-11
US201361891313P 2013-10-15 2013-10-15
US61/891,313 2013-10-15
US201361919540P 2013-12-20 2013-12-20
US61/919,540 2013-12-20
US201461927903P 2014-01-15 2014-01-15
US61/927,903 2014-01-15
US201461980502P 2014-04-16 2014-04-16
US61/980,502 2014-04-16
PCT/US2014/060194 WO2015054676A2 (en) 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression

Related Child Applications (1)

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JP2020047126A Division JP2020115865A (ja) 2013-10-11 2020-03-18 Ｃ９ｏｒｆ７２発現を調節するための組成物

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JP2016533717A JP2016533717A (ja) 2016-11-04
JP2016533717A5 JP2016533717A5 (https=) 2017-11-24
JP6679476B2 true JP6679476B2 (ja) 2020-04-15

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JP2020047126A Ceased JP2020115865A (ja) 2013-10-11 2020-03-18 Ｃ９ｏｒｆ７２発現を調節するための組成物

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US (3) US10221414B2 (https=)
EP (2) EP4166667A3 (https=)
JP (2) JP6679476B2 (https=)
KR (1) KR20160062069A (https=)
CN (2) CN112080502A (https=)
AU (2) AU2014331652B2 (https=)
BR (1) BR112016007751A2 (https=)
CA (1) CA2926408A1 (https=)
CL (3) CL2016000839A1 (https=)
IL (1) IL244759A0 (https=)
MX (2) MX2016004651A (https=)
MY (1) MY192689A (https=)
PH (1) PH12016500603A1 (https=)
RU (1) RU2748426C2 (https=)
SG (2) SG10201808903UA (https=)
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