TW201831684A - 用於cccDNA之寡核苷酸標靶策略 - Google Patents

用於cccDNA之寡核苷酸標靶策略 Download PDF

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Publication number
TW201831684A
TW201831684A TW106139190A TW106139190A TW201831684A TW 201831684 A TW201831684 A TW 201831684A TW 106139190 A TW106139190 A TW 106139190A TW 106139190 A TW106139190 A TW 106139190A TW 201831684 A TW201831684 A TW 201831684A
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Taiwan
Prior art keywords
oligonucleotide
hbv
nucleotide
seq
formula
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TW106139190A
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English (en)
Chinese (zh)
Inventor
薛爾蓋 格里亞茲諾夫
梅根 菲茨杰拉德
安緹莎 史托契瓦
勁 洪
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美商艾洛斯生物製藥公司
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Publication of TW201831684A publication Critical patent/TW201831684A/zh

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1131—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 viruses
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW106139190A 2016-11-11 2017-11-13 用於cccDNA之寡核苷酸標靶策略 TW201831684A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662420801P 2016-11-11 2016-11-11
US62/420,801 2016-11-11
US201762558770P 2017-09-14 2017-09-14
US62/558,770 2017-09-14

Publications (1)

Publication Number Publication Date
TW201831684A true TW201831684A (zh) 2018-09-01

Family

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Family Applications (1)

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TW106139190A TW201831684A (zh) 2016-11-11 2017-11-13 用於cccDNA之寡核苷酸標靶策略

Country Status (10)

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US (1) US20180179542A1 (enExample)
EP (1) EP3538654A1 (enExample)
JP (1) JP2019533472A (enExample)
KR (1) KR20190076050A (enExample)
CN (1) CN110234763A (enExample)
AU (1) AU2017356221A1 (enExample)
CA (1) CA3043637A1 (enExample)
IL (1) IL266525A (enExample)
TW (1) TW201831684A (enExample)
WO (1) WO2018089914A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202002149XA (en) * 2017-09-14 2020-04-29 Janssen Biopharma Inc Galnac derivatives
WO2019240504A1 (en) * 2018-06-12 2019-12-19 Am Sciences Co., Ltd. Modified oligonucleotides for inhibition of target gene expression
WO2019240503A1 (ko) * 2018-06-12 2019-12-19 주식회사 에이엠사이언스 B형 간염 예방 또는 치료용 조성물
JP2023506540A (ja) * 2019-12-19 2023-02-16 エフ. ホフマン-ラ ロシュ エージー. B型肝炎ウイルス感染を処置するためのscamp3阻害剤の使用
WO2021122921A1 (en) * 2019-12-19 2021-06-24 F. Hoffmann-La Roche Ag Use of cops3 inhibitors for treating hepatitis b virus infection
CN113493855B (zh) * 2020-03-19 2023-12-26 首都医科大学附属北京佑安医院 一种基于RAA-CRISPR-cas13a检测HBV cccDNA的试剂盒
CA3187388A1 (en) * 2020-07-27 2022-02-03 Jin Hong Hbv binding oligonucleotides and methods of use
JP2023538630A (ja) * 2020-08-21 2023-09-08 エフ. ホフマン-ラ ロシュ アーゲー B型肝炎ウイルス感染症を処置するためのa1cf阻害剤の使用
CN118667808B (zh) * 2023-08-22 2025-04-18 浙江柏拉阿图医药科技有限公司 双‐Gap修饰寡核苷酸及其在抗乙型肝炎和丁型肝炎病毒中的应用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999001579A1 (en) 1997-07-01 1999-01-14 Isis Pharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
AR028149A1 (es) * 1999-07-08 2003-04-30 Innogenetics Nv Deteccion de la resistencia a los farmacos contra la hepatitis b
HK1049339B (en) * 1999-09-10 2008-06-27 Geron Corporation Oligonucleotide n3'-p5' thiophosphoramidates: their synthesis and use
WO2006069064A2 (en) * 2004-12-22 2006-06-29 Nucleonics, Inc. Conserved hbv and hcv sequences useful for gene silencing
SI3505528T1 (sl) * 2011-04-21 2021-04-30 Glaxo Group Limited Modulacija izražanja virusa hepatitisa B (HBV)
PL2992098T3 (pl) * 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
TWI769565B (zh) * 2014-11-10 2022-07-01 美商阿尼拉製藥公司 B型肝炎病毒(HBV)iRNA組成物及其用途方法
CN107683336A (zh) * 2015-05-06 2018-02-09 贝尼泰克生物制药有限公司 用于治疗乙型肝炎病毒(hbv)感染的试剂及其用途

Also Published As

Publication number Publication date
CN110234763A (zh) 2019-09-13
WO2018089914A1 (en) 2018-05-17
KR20190076050A (ko) 2019-07-01
US20180179542A1 (en) 2018-06-28
AU2017356221A1 (en) 2019-05-30
IL266525A (en) 2019-07-31
EP3538654A1 (en) 2019-09-18
CA3043637A1 (en) 2018-05-17
JP2019533472A (ja) 2019-11-21

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