AU2025205193A1 - Double-Stranded RNAi Agents For Targeting And Regulating HBV Gene Expression, And A Use Thereof - Google Patents

Double-Stranded RNAi Agents For Targeting And Regulating HBV Gene Expression, And A Use Thereof

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Publication number
AU2025205193A1
AU2025205193A1 AU2025205193A AU2025205193A AU2025205193A1 AU 2025205193 A1 AU2025205193 A1 AU 2025205193A1 AU 2025205193 A AU2025205193 A AU 2025205193A AU 2025205193 A AU2025205193 A AU 2025205193A AU 2025205193 A1 AU2025205193 A1 AU 2025205193A1
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AU
Australia
Prior art keywords
strand
sequence
double
modified
lipid
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
AU2025205193A
Other languages
English (en)
Inventor
Zhongcai GAO
Zeao HUANG
Gengshen SONG
Zhikang TIAN
Yucheng Wu
Kai XIONG
Shuo YANG
Yang Yang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Hangzhou Tianlong Pharmaceutical Co Ltd
Original Assignee
Hangzhou Tianlong Pharmaceutical Co Ltd
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
Priority claimed from CN202510037618.3A external-priority patent/CN119752912B/zh
Application filed by Hangzhou Tianlong Pharmaceutical Co Ltd filed Critical Hangzhou Tianlong Pharmaceutical Co Ltd
Publication of AU2025205193A1 publication Critical patent/AU2025205193A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2025205193A 2024-07-08 2025-07-07 Double-Stranded RNAi Agents For Targeting And Regulating HBV Gene Expression, And A Use Thereof Pending AU2025205193A1 (en)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
CN202410909405 2024-07-08
CN2024109094050 2024-07-08
CN202510037618.3A CN119752912B (zh) 2024-07-08 2025-01-09 靶向调控HBV基因表达的siRNA及其应用
CN2025100376183 2025-01-09
CN202510039613.4A CN119799706B (zh) 2024-07-08 2025-01-09 靶向调控HBV基因表达的siRNA及其应用
CN2025100393920 2025-01-09
CN2025100396134 2025-01-09
CN202510039392.0A CN119799705B (zh) 2024-07-08 2025-01-09 靶向调控HBV基因表达的siRNA及其应用

Publications (1)

Publication Number Publication Date
AU2025205193A1 true AU2025205193A1 (en) 2026-01-29

Family

ID=96343514

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2025205193A Pending AU2025205193A1 (en) 2024-07-08 2025-07-07 Double-Stranded RNAi Agents For Targeting And Regulating HBV Gene Expression, And A Use Thereof

Country Status (6)

Country Link
EP (1) EP4678747A3 (https=)
JP (1) JP2026009862A (https=)
KR (1) KR20260008676A (https=)
AU (1) AU2025205193A1 (https=)
CA (1) CA3279332A1 (https=)
IL (1) IL322012A (https=)

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KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
IL292159B1 (en) 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA compositions and methods of using them
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
EP4186888B1 (en) 2017-03-15 2025-11-26 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
JP7625512B2 (ja) * 2018-08-13 2025-02-03 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｂ型肝炎ウイルス（ＨＢＶ）ｄｓＲＮＡ物質組成物およびその使用方法
WO2020163747A1 (en) * 2019-02-07 2020-08-13 Arrowhead Pharmaceuticals, Inc. Rnai agents for hepatitis b virus infection
MA55375A (fr) * 2019-03-20 2022-01-26 Arbutus Biopharma Corp Méthodes thérapeutiques de traitement de l'hépatite b
JP7455336B2 (ja) * 2019-03-26 2024-03-26 富士フイルム株式会社 Ｂ型肝炎ウイルスタンパク質の産生を阻害する医薬組成物およびスクリーニング方法
PH12021552870A1 (en) * 2019-05-13 2022-03-21 Vir Biotechnology Inc Compositions and methods for treating hepatitis b virus (hbv) infection
WO2021110148A1 (zh) * 2019-12-06 2021-06-10 南京明德新药研发有限公司 siRNA缀合物、双链siRNA缀合物及其盐和应用
TW202221122A (zh) * 2020-08-05 2022-06-01 瑞士商赫孚孟拉羅股份公司 Ｂ型肝炎患者之寡核苷酸治療
EP4223876A4 (en) * 2020-09-30 2025-12-10 Fujifilm Corp DOUBLE-STRANDED RNA THAT INHIBITS THE PRODUCTION OF A HEPATITIS B VIRUS PROTEIN, AND PHARMACEUTICAL COMPOSITION
US20240124879A1 (en) * 2020-12-18 2024-04-18 Olix Pharmaceuticals, Inc. Rnai agent for inhibiting hbv expression and use thereof
CA3235672A1 (en) * 2021-11-29 2023-06-01 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of hepatitis b virus (hbv) protein
CN114044741B (zh) 2022-01-13 2022-04-15 北京悦康科创医药科技股份有限公司 一种阳离子脂质化合物、包含其的组合物及用途
CN115745820B (zh) 2023-01-05 2023-04-28 北京悦康科创医药科技股份有限公司 长效低毒的新型阳离子脂质化合物及其组合物
CN115677518B (zh) 2023-01-05 2023-04-14 北京悦康科创医药科技股份有限公司 用于递送核酸的可电离阳离子脂质化合物和组合物及用途
CN115784921B (zh) 2023-02-08 2023-05-05 北京悦康科创医药科技股份有限公司 肝外靶向高效低毒的阳离子脂质化合物及其组合物
CN116854754B (zh) 2023-09-01 2023-12-12 北京悦康科创医药科技股份有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物

Also Published As

Publication number Publication date
CA3279332A1 (en) 2026-03-01
EP4678747A3 (en) 2026-03-25
KR20260008676A (ko) 2026-01-16
JP2026009862A (ja) 2026-01-21
IL322012A (en) 2026-02-01
EP4678747A2 (en) 2026-01-14

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