JP7169995B2 - Ｈｔｒａ１発現を調節するためのアンチセンスオリゴヌクレオチド - Google Patents

Ｈｔｒａ１発現を調節するためのアンチセンスオリゴヌクレオチド Download PDF

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JP7169995B2
JP7169995B2 JP2019566236A JP2019566236A JP7169995B2 JP 7169995 B2 JP7169995 B2 JP 7169995B2 JP 2019566236 A JP2019566236 A JP 2019566236A JP 2019566236 A JP2019566236 A JP 2019566236A JP 7169995 B2 JP7169995 B2 JP 7169995B2
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pharmaceutically acceptable
htra1
oligonucleotide
nucleosides
acceptable salt
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JP2020521491A (ja
JP2020521491A5 (enExample
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サンチェス，ルーベン・アルバレス
イアコーネ，ロベルト
ハーゲドルン，ペーター
カムラ，スサネ
オトセン，セーアン
トラウスタソン，シンドリ
フルレブッシュ，ハイディ・リュ
ピーダスン，リュケ
ベレラ，マルコ
ディークマン，アンドレアス
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F Hoffmann La Roche AG
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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
    • C12N15/1137—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 against enzymes
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21108—HtrA2 peptidase (3.4.21.108)
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    • C12N2310/30—Chemical structure
    • 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
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2019566236A 2017-06-01 2018-05-30 Ｈｔｒａ１発現を調節するためのアンチセンスオリゴヌクレオチド Active JP7169995B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
EP17173964.2 2017-06-01
EP17173964 2017-06-01
EP17209407.0 2017-12-21
EP17209535 2017-12-21
EP17209407 2017-12-21
EP17209535.8 2017-12-21
PCT/EP2018/064221 WO2018220034A1 (en) 2017-06-01 2018-05-30 Antisense oligonucleotides for modulating htra1 expression

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JP2020521491A JP2020521491A (ja) 2020-07-27
JP2020521491A5 JP2020521491A5 (enExample) 2021-07-26
JP7169995B2 true JP7169995B2 (ja) 2022-11-11

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US (3) US20190055564A1 (enExample)
EP (1) EP3630976A1 (enExample)
JP (1) JP7169995B2 (enExample)
KR (1) KR20200015608A (enExample)
CN (1) CN110691849A (enExample)
AU (1) AU2018277219A1 (enExample)
BR (1) BR112019025290A2 (enExample)
CA (1) CA3062590A1 (enExample)
CL (1) CL2019003337A1 (enExample)
CO (1) CO2019013317A2 (enExample)
CR (1) CR20190543A (enExample)
IL (1) IL271039A (enExample)
MA (1) MA49278A (enExample)
MX (1) MX2019014187A (enExample)
PE (1) PE20191845A1 (enExample)
PH (1) PH12019502636A1 (enExample)
RU (1) RU2019143004A (enExample)
TW (1) TW201907008A (enExample)
WO (1) WO2018220034A1 (enExample)

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RU2019101298A (ru) 2016-07-01 2020-08-03 Ф. Хоффманн-Ля Рош Аг Антисмысловые олигонуклеотиды для модулирования экспрессии htra1
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
KR102414152B1 (ko) * 2020-08-20 2022-06-29 (주)지놈오피니언 연령 관련 황반변성 진단용 바이오마커 및 이의 용도
JPWO2022092326A1 (enExample) * 2020-10-30 2022-05-05

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WO2009006460A1 (en) 2007-07-02 2009-01-08 Alcon Research, Ltd. Rnai-mediated inhibition of htra1 for treatment of macular degeneration
WO2012038668A1 (fr) 2010-09-23 2012-03-29 Universite De Caen Basse Normandie Procédé d'obtention in vitro ou ex vivo de chondrocytes articulaires différenciés et leurs utilisations

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Publication number Publication date
CN110691849A (zh) 2020-01-14
US20200157546A1 (en) 2020-05-21
RU2019143004A (ru) 2021-07-09
TW201907008A (zh) 2019-02-16
AU2018277219A1 (en) 2019-12-19
MA49278A (fr) 2020-04-08
CA3062590A1 (en) 2018-12-06
JP2020521491A (ja) 2020-07-27
IL271039A (en) 2020-01-30
WO2018220034A1 (en) 2018-12-06
CO2019013317A2 (es) 2020-01-17
KR20200015608A (ko) 2020-02-12
US20220042022A1 (en) 2022-02-10
PH12019502636A1 (en) 2020-06-08
BR112019025290A2 (pt) 2020-06-23
MX2019014187A (es) 2020-01-27
EP3630976A1 (en) 2020-04-08
US20190055564A1 (en) 2019-02-21
PE20191845A1 (es) 2019-12-31
CL2019003337A1 (es) 2020-05-15
CR20190543A (es) 2020-02-10

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