JP2023505434A - 肝線維症を治療するためのコンジュゲート及び方法 - Google Patents

肝線維症を治療するためのコンジュゲート及び方法 Download PDF

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JP2023505434A
JP2023505434A JP2022532614A JP2022532614A JP2023505434A JP 2023505434 A JP2023505434 A JP 2023505434A JP 2022532614 A JP2022532614 A JP 2022532614A JP 2022532614 A JP2022532614 A JP 2022532614A JP 2023505434 A JP2023505434 A JP 2023505434A
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formula
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sirna
alkyl
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JP2023505434A5 (https=
Inventor
ヘイズ，ジェイムズ
ジェイ ホーランド，リチャード
ウッド，マーク
ディー マーティン，アラン
エサウ，クリスティーン
シュヴァルツ，マルグリット
イエ，シン
ホイ ラム リー，アリス
ジャスティン パセツカ，クリストファー
クロウ，デイヴィッド
タイラー，スティーヴン
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Genevant Sciences GmbH
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Genevant Sciences GmbH
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/01—Oxidoreductases acting on the CH-OH group of donors (1.1) with NAD+ or NADP+ as acceptor (1.1.1)
    • C12Y101/01051—3 (or 17)-Beta-hydroxysteroid dehydrogenase (1.1.1.51)
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
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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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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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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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
JP2022532614A 2019-12-06 2020-12-07 肝線維症を治療するためのコンジュゲート及び方法 Pending JP2023505434A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962944963P 2019-12-06 2019-12-06
US62/944,963 2019-12-06
PCT/US2020/063617 WO2021113820A1 (en) 2019-12-06 2020-12-07 Conjugates and methods for treating liver fibrosis

Publications (2)

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JP2023505434A true JP2023505434A (ja) 2023-02-09
JP2023505434A5 JP2023505434A5 (https=) 2023-12-13

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JP2022532614A Pending JP2023505434A (ja) 2019-12-06 2020-12-07 肝線維症を治療するためのコンジュゲート及び方法

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US (1) US20220387600A1 (https=)
EP (1) EP4069276A4 (https=)
JP (1) JP2023505434A (https=)
KR (1) KR20220112788A (https=)
CN (1) CN115884984A (https=)
AU (1) AU2020398708A1 (https=)
BR (1) BR112022010613A2 (https=)
CA (1) CA3163911A1 (https=)
IL (1) IL293560A (https=)
MX (1) MX2022006846A (https=)
NZ (1) NZ788988A (https=)
WO (1) WO2021113820A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3020585A1 (en) * 2016-04-11 2017-10-19 Arbutus Biopharma Corporation Targeted nucleic acid conjugate compositions
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
WO2024175062A1 (zh) * 2023-02-23 2024-08-29 大睿生物医药科技(上海)有限公司 调控KHK基因活性的dsRNA分子
US20240352464A1 (en) * 2023-03-07 2024-10-24 Aligos Therapeutics, Inc. Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
CN119219716A (zh) * 2023-06-30 2024-12-31 上海维申医药有限公司 新型GalNAc的靶向递送片段及其制备和应用
WO2025255972A1 (zh) * 2024-06-13 2025-12-18 广州必贝特医药股份有限公司 用于靶向递送寡核苷酸药物的化合物、缀合物及其应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017177326A1 (en) * 2016-04-11 2017-10-19 Protiva Biotherapeutics, Inc. Targeted nucleic acid conjugate compositions
WO2018191278A2 (en) * 2017-04-11 2018-10-18 Arbutus Biopharma Corporation Targeted compositions
WO2019183164A1 (en) * 2018-03-21 2019-09-26 Regeneron Pharmaceuticals, Inc. 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019051257A2 (en) * 2017-09-11 2019-03-14 Arbutus Biopharma Corporation METHODS OF TREATING HEPATITIS B TYPE INFECTIONS
EP3873530A4 (en) * 2018-11-02 2022-11-09 Genevant Sciences GmbH THERAPEUTIC PROCESSES

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017177326A1 (en) * 2016-04-11 2017-10-19 Protiva Biotherapeutics, Inc. Targeted nucleic acid conjugate compositions
WO2018191278A2 (en) * 2017-04-11 2018-10-18 Arbutus Biopharma Corporation Targeted compositions
WO2019183164A1 (en) * 2018-03-21 2019-09-26 Regeneron Pharmaceuticals, Inc. 17β-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF

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Publication number Publication date
IL293560A (en) 2022-08-01
NZ788988A (en) 2026-02-27
US20220387600A1 (en) 2022-12-08
EP4069276A4 (en) 2025-10-08
EP4069276A1 (en) 2022-10-12
BR112022010613A2 (pt) 2022-08-16
CN115884984A (zh) 2023-03-31
CA3163911A1 (en) 2021-06-10
AU2020398708A1 (en) 2022-07-14
WO2021113820A1 (en) 2021-06-10
KR20220112788A (ko) 2022-08-11
MX2022006846A (es) 2022-10-21

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