JP2022535735A - 修飾ギャップマーオリゴヌクレオチドおよび使用方法 - Google Patents

修飾ギャップマーオリゴヌクレオチドおよび使用方法 Download PDF

Info

Publication number
JP2022535735A
JP2022535735A JP2021570299A JP2021570299A JP2022535735A JP 2022535735 A JP2022535735 A JP 2022535735A JP 2021570299 A JP2021570299 A JP 2021570299A JP 2021570299 A JP2021570299 A JP 2021570299A JP 2022535735 A JP2022535735 A JP 2022535735A
Authority
JP
Japan
Prior art keywords
aso
nucleosides
hbv
administered
asos
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2021570299A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022535735A5 (https=
Inventor
ベイゲルマン,レオニド
ケイ. パンディ,ラジェンドラ
クマル ラジワンシ,ヴィヴェク
ベルナルド スミス,デイヴィッド
ホン,ジン
Original Assignee
アリゴス セラピューティクス インコーポレイテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by アリゴス セラピューティクス インコーポレイテッド filed Critical アリゴス セラピューティクス インコーポレイテッド
Publication of JP2022535735A publication Critical patent/JP2022535735A/ja
Publication of JP2022535735A5 publication Critical patent/JP2022535735A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1131Non-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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/333Modified A
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/336Modified G
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021570299A 2019-05-31 2020-05-29 修飾ギャップマーオリゴヌクレオチドおよび使用方法 Pending JP2022535735A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962855793P 2019-05-31 2019-05-31
US62/855,793 2019-05-31
US201962937760P 2019-11-19 2019-11-19
US62/937,760 2019-11-19
US201962943532P 2019-12-04 2019-12-04
US62/943,532 2019-12-04
PCT/US2020/035212 WO2020243490A2 (en) 2019-05-31 2020-05-29 Modified gapmer oligonucleotides and methods of use

Publications (2)

Publication Number Publication Date
JP2022535735A true JP2022535735A (ja) 2022-08-10
JP2022535735A5 JP2022535735A5 (https=) 2023-05-31

Family

ID=72179172

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021570299A Pending JP2022535735A (ja) 2019-05-31 2020-05-29 修飾ギャップマーオリゴヌクレオチドおよび使用方法

Country Status (15)

Country Link
US (1) US11466274B2 (https=)
EP (1) EP3976056A2 (https=)
JP (1) JP2022535735A (https=)
KR (1) KR20220024192A (https=)
CN (1) CN113905744A (https=)
AU (1) AU2020282828A1 (https=)
BR (1) BR112021023488A2 (https=)
CA (1) CA3141874A1 (https=)
CO (1) CO2021018034A2 (https=)
IL (1) IL288259A (https=)
MX (1) MX2021014206A (https=)
PE (1) PE20220574A1 (https=)
SG (1) SG11202112741XA (https=)
TW (1) TW202113079A (https=)
WO (1) WO2020243490A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210189392A1 (en) * 2019-12-12 2021-06-24 Aligos Therapeutics, Inc. S-antigen transport inhibiting oligonucleotide polymers and methods
AU2021318943A1 (en) * 2020-07-27 2023-03-02 Aligos Therapeutics, Inc. HBV binding oligonucleotides and methods of use
TW202229554A (zh) * 2020-12-31 2022-08-01 中山大學附屬第一醫院 用於降低血管收縮素轉化酶2(ace2)之表現的寡核苷酸及其用於治療病毒感染的用途
US20250243491A1 (en) 2021-09-01 2025-07-31 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
CN118234862A (zh) 2021-09-08 2024-06-21 阿利戈斯治疗公司 经修饰的短干扰核酸分子(siNA)及其用途
TW202340468A (zh) 2022-01-10 2023-10-16 中國大陸商杭州浩博醫藥有限公司 B型肝炎病毒(hbv)表現之調節
KR20240158340A (ko) * 2022-03-17 2024-11-04 알리고스 테라퓨틱스 인코포레이티드 변형 갭머 올리고머 및 이의 사용 방법
WO2024182446A2 (en) 2023-02-28 2024-09-06 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
US20240352464A1 (en) 2023-03-07 2024-10-24 Aligos Therapeutics, Inc. Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
CN121175419A (zh) * 2023-04-14 2025-12-19 苏州炫景生物科技有限公司 抑制c3基因表达的化合物、药物组合物及其用途
US20250368996A1 (en) 2024-05-14 2025-12-04 Aligos Therapeutics, Inc. Modified antisense oligonucleotides for treating hepatitis b virus
CN120796266A (zh) * 2024-07-08 2025-10-17 北京悦康科创医药科技股份有限公司 一种降低hbv基因表达的反义寡核苷酸及其应用

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10503367A (ja) * 1994-07-28 1998-03-31 ジョージタウン ユニバーシティー B型肝炎ウイルス複製に対するアンチセンスオリゴヌクレオチド
JPH10510435A (ja) * 1995-07-13 1998-10-13 アイシス・ファーマシューティカルス・インコーポレーテッド B型肝炎ウイルス複製のアンチセンス阻害
JP2013507933A (ja) * 2009-10-16 2013-03-07 グラクソ グループ リミテッド Hbvアンチセンス阻害剤
JP2014513954A (ja) * 2011-04-21 2014-06-19 アイシス ファーマシューティカルズ, インコーポレーテッド B型肝炎ウイルス(hbv)発現の調節
WO2017216390A1 (en) * 2016-06-17 2017-12-21 F. Hoffmann-La Roche Ag Nucleic acid molecules for reduction of papd5 or papd7 mrna for treating hepatitis b infection
WO2018155450A1 (ja) * 2017-02-21 2018-08-30 国立大学法人大阪大学 アンチセンスオリゴ核酸
WO2018155451A1 (ja) * 2017-02-21 2018-08-30 国立大学法人大阪大学 核酸化合物およびオリゴヌクレオチド
WO2019076842A1 (en) * 2017-10-16 2019-04-25 F. Hoffmann-La Roche Ag NUCLEIC ACID MOLECULE FOR THE REDUCTION OF PAPD5 AND PAPD7 MRNA FOR THE TREATMENT OF HEPATITIS B INFECTION

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040127446A1 (en) 1992-05-14 2004-07-01 Lawrence Blatt Oligonucleotide mediated inhibition of hepatitis B virus and hepatitis C virus replication
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
WO2005009346A2 (en) 2003-06-24 2005-02-03 Mirus Corporation Inhibition of gene function by delivery of polynucleotide-based gene expression inhibitors to mammalian cells in vivo
EP2007888A2 (en) 2006-04-03 2008-12-31 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
CA2778171C (en) 2009-10-29 2017-07-25 Osaka University Bridged artificial nucleoside and nucleotide
EP2673361B1 (en) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2014043212A2 (en) 2012-09-11 2014-03-20 3M Innovative Properties Company Glass granule having a zoned structure
KR101707437B1 (ko) 2012-09-21 2017-02-16 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 구아니딘 가교를 갖는 인공 뉴클레오시드 및 올리고뉴클레오티드
CN104837996A (zh) 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
AU2014211406B2 (en) 2013-01-30 2019-07-18 Roche Innovation Center Copenhagen A/S LNA oligonucleotide carbohydrate conjugates
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
WO2015042447A1 (en) 2013-09-20 2015-03-26 Isis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
CN106103463B (zh) 2014-02-18 2018-11-30 国立大学法人大阪大学 桥连型核苷和核苷酸
GB201408623D0 (en) * 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
CN106795200B (zh) 2014-10-10 2020-06-19 豪夫迈·罗氏有限公司 Galnac亚磷酰胺、其核酸缀合物及其用途
WO2016079183A1 (en) 2014-11-19 2016-05-26 Roche Innovation Center Copenhagen A/S Lna chiral phosphorothioates
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
PE20211783A1 (es) 2018-11-08 2021-09-08 Aligos Therapeutics Inc Polimeros oligonucleotidos que inhiben el transporte de antigenos y metodos
MX2021009717A (es) 2019-02-14 2021-09-14 Aligos Therapeutics Inc Sulfonamidas biciclicas.

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10503367A (ja) * 1994-07-28 1998-03-31 ジョージタウン ユニバーシティー B型肝炎ウイルス複製に対するアンチセンスオリゴヌクレオチド
JPH10510435A (ja) * 1995-07-13 1998-10-13 アイシス・ファーマシューティカルス・インコーポレーテッド B型肝炎ウイルス複製のアンチセンス阻害
JP2013507933A (ja) * 2009-10-16 2013-03-07 グラクソ グループ リミテッド Hbvアンチセンス阻害剤
JP2014513954A (ja) * 2011-04-21 2014-06-19 アイシス ファーマシューティカルズ, インコーポレーテッド B型肝炎ウイルス(hbv)発現の調節
WO2017216390A1 (en) * 2016-06-17 2017-12-21 F. Hoffmann-La Roche Ag Nucleic acid molecules for reduction of papd5 or papd7 mrna for treating hepatitis b infection
WO2018155450A1 (ja) * 2017-02-21 2018-08-30 国立大学法人大阪大学 アンチセンスオリゴ核酸
WO2018155451A1 (ja) * 2017-02-21 2018-08-30 国立大学法人大阪大学 核酸化合物およびオリゴヌクレオチド
WO2019076842A1 (en) * 2017-10-16 2019-04-25 F. Hoffmann-La Roche Ag NUCLEIC ACID MOLECULE FOR THE REDUCTION OF PAPD5 AND PAPD7 MRNA FOR THE TREATMENT OF HEPATITIS B INFECTION

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
THE NEW ENGLAND JOURNAL OF MEDICINE, vol. 334, no. 17, JPN6023004527, 1996, pages 1135 - 1138, ISSN: 0005480292 *

Also Published As

Publication number Publication date
WO2020243490A3 (en) 2021-01-21
TW202113079A (zh) 2021-04-01
US20200385735A1 (en) 2020-12-10
CA3141874A1 (en) 2020-12-03
KR20220024192A (ko) 2022-03-03
IL288259A (en) 2022-01-01
BR112021023488A2 (pt) 2022-01-18
SG11202112741XA (en) 2021-12-30
MX2021014206A (es) 2022-01-06
AU2020282828A1 (en) 2022-01-27
EP3976056A2 (en) 2022-04-06
US11466274B2 (en) 2022-10-11
CO2021018034A2 (es) 2022-04-19
CN113905744A (zh) 2022-01-07
PE20220574A1 (es) 2022-04-20
WO2020243490A2 (en) 2020-12-03

Similar Documents

Publication Publication Date Title
JP2022535735A (ja) 修飾ギャップマーオリゴヌクレオチドおよび使用方法
JP7792371B2 (ja) GalNAc誘導体
TWI769177B (zh) 經修飾之寡核苷酸及使用方法
KR20210090217A (ko) S-항원 수송 저해 올리고뉴클레오타이드 중합체 및 방법
JP5559159B2 (ja) 修飾オリゴヌクレオチドを使用するhrp−3の阻害
JP2026069791A (ja) Hbvの処置のためのhbvを標的とする治療用オリゴヌクレオチドとtlr7アゴニストとの医薬組合せ
KR20240101580A (ko) Hbv 치료를 위한 약학 조합물
JP2025509552A (ja) 修飾ギャップマーオリゴマー及びその使用方法
HK40036011A (en) Galnac derivatives
HK40110728A (zh) 用於治疗hbv的药物组合

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230523

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230523

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240514

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20241210