JP2018531037A - 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物 - Google Patents

長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物 Download PDF

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JP2018531037A
JP2018531037A JP2018521276A JP2018521276A JP2018531037A JP 2018531037 A JP2018531037 A JP 2018531037A JP 2018521276 A JP2018521276 A JP 2018521276A JP 2018521276 A JP2018521276 A JP 2018521276A JP 2018531037 A JP2018531037 A JP 2018531037A
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nucleic acid
acid molecule
cell
modified
lncrna
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JP2018531037A6 (ja
JP2018531037A5 (OSRAM
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カーディア，ジェームズ
ブロック，カレン，ジー．
ヴァンデサンペール，ジョーク，ヘドヴィク
ペール，ゲール ヴァン
ペール，ゲール ヴァン
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Biogazelle NV
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Biogazelle NV
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Publication of JP2018531037A5 publication Critical patent/JP2018531037A5/ja
Priority to JP2022000077A priority Critical patent/JP2022061991A/ja
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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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
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    • C12N2310/334—Modified C
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/335—Modified T or U
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2018521276A 2015-10-19 2016-10-19 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物 Ceased JP2018531037A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022000077A JP2022061991A (ja) 2015-10-19 2022-01-04 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562243565P 2015-10-19 2015-10-19
US62/243,565 2015-10-19
PCT/US2016/057608 WO2017070151A1 (en) 2015-10-19 2016-10-19 Reduced size self-delivering nucleic acid compounds targeting long non-coding rna

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JP2022000077A Division JP2022061991A (ja) 2015-10-19 2022-01-04 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物

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JP2018531037A true JP2018531037A (ja) 2018-10-25
JP2018531037A6 JP2018531037A6 (ja) 2018-12-13
JP2018531037A5 JP2018531037A5 (OSRAM) 2020-03-05

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JP2018521276A Ceased JP2018531037A (ja) 2015-10-19 2016-10-19 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物
JP2022000077A Pending JP2022061991A (ja) 2015-10-19 2022-01-04 長い非コードｒｎａを標的とする減少したサイズの自己送達型核酸化合物

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US (1) US11021707B2 (OSRAM)
EP (1) EP3365446A4 (OSRAM)
JP (2) JP2018531037A (OSRAM)
CN (1) CN109563509B (OSRAM)
CA (1) CA3002744A1 (OSRAM)
WO (1) WO2017070151A1 (OSRAM)

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US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
KR20180044433A (ko) 2010-03-24 2018-05-02 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 ｒｎａ 간섭
CA2794187C (en) 2010-03-24 2020-07-14 Rxi Pharmaceuticals Corporation Rna interference in ocular indications
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