TW201907008A - 用於調節htra1表現之反義寡核苷酸 - Google Patents

用於調節htra1表現之反義寡核苷酸 Download PDF

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Publication number
TW201907008A
TW201907008A TW107118609A TW107118609A TW201907008A TW 201907008 A TW201907008 A TW 201907008A TW 107118609 A TW107118609 A TW 107118609A TW 107118609 A TW107118609 A TW 107118609A TW 201907008 A TW201907008 A TW 201907008A
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Taiwan
Prior art keywords
oligonucleotide
htra1
nucleosides
seq
disease
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TW107118609A
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English (en)
Chinese (zh)
Inventor
魯本 阿凡瑞茲 山查茲
羅伯特 伊康
彼得 哈吉朵恩
蘇珊 卡麥樂
索倫 歐特森
辛德瑞 陶史迪生
海蒂 萊 胡朵布希
萊可 佩得森
馬可 巴雷拉
安德魯斯 迪克曼
Original Assignee
瑞士商赫孚孟拉羅股份公司
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Publication of TW201907008A publication Critical patent/TW201907008A/zh

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21108HtrA2 peptidase (3.4.21.108)
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/35Nature of the modification
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
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  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Ophthalmology & Optometry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
TW107118609A 2017-06-01 2018-05-31 用於調節htra1表現之反義寡核苷酸 TW201907008A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
EP17173964 2017-06-01
??17173964.2 2017-06-01
??17209407.0 2017-12-21
??17209535.8 2017-12-21
EP17209535 2017-12-21
EP17209407 2017-12-21

Publications (1)

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TW201907008A true TW201907008A (zh) 2019-02-16

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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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* Cited by examiner, † Cited by third party
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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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KR100414936B1 (ko) 1997-09-12 2004-01-13 엑시콘 에이/에스 이환 및 삼환 뉴클레오시드, 뉴클레오타이드 및올리고뉴클레오타이드 동족체
WO2000008134A2 (en) * 1998-08-03 2000-02-17 Novartis Ag HUMAN HtrA SERINE PROTEASE
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Also Published As

Publication number Publication date
CN110691849A (zh) 2020-01-14
US20200157546A1 (en) 2020-05-21
RU2019143004A (ru) 2021-07-09
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
JP7169995B2 (ja) 2022-11-11
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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