KR20230104652A - 변형된 siRNA를 포함하는 표적화된 접합체 - Google Patents

변형된 siRNA를 포함하는 표적화된 접합체 Download PDF

Info

Publication number
KR20230104652A
KR20230104652A KR1020237018346A KR20237018346A KR20230104652A KR 20230104652 A KR20230104652 A KR 20230104652A KR 1020237018346 A KR1020237018346 A KR 1020237018346A KR 20237018346 A KR20237018346 A KR 20237018346A KR 20230104652 A KR20230104652 A KR 20230104652A
Authority
KR
South Korea
Prior art keywords
salt
compound
group
alkyl
chloro
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
KR1020237018346A
Other languages
English (en)
Korean (ko)
Inventor
오웬 엠. 달리
에이미 씨. 에이치. 리
미쉘 제이. 소피아
에밀리 피. 타이
Original Assignee
아뷰터스 바이오파마 코포레이션
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 아뷰터스 바이오파마 코포레이션 filed Critical 아뷰터스 바이오파마 코포레이션
Publication of KR20230104652A publication Critical patent/KR20230104652A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • 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
    • 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
    • C12N2310/31—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
    • C12N2310/322—2'-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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

Landscapes

  • 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)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
KR1020237018346A 2020-11-06 2021-11-05 변형된 siRNA를 포함하는 표적화된 접합체 Pending KR20230104652A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063110837P 2020-11-06 2020-11-06
US63/110,837 2020-11-06
PCT/US2021/058232 WO2022098990A1 (en) 2020-11-06 2021-11-05 Targeted conjugates comprising modified sirna

Publications (1)

Publication Number Publication Date
KR20230104652A true KR20230104652A (ko) 2023-07-10

Family

ID=81457390

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237018346A Pending KR20230104652A (ko) 2020-11-06 2021-11-05 변형된 siRNA를 포함하는 표적화된 접합체

Country Status (11)

Country Link
US (1) US20240052349A1 (https=)
EP (1) EP4240369A4 (https=)
JP (1) JP2023548295A (https=)
KR (1) KR20230104652A (https=)
CN (1) CN116472063A (https=)
AU (1) AU2021376390A1 (https=)
CA (1) CA3199757A1 (https=)
IL (1) IL302530A (https=)
MX (1) MX2023005138A (https=)
TW (1) TW202233243A (https=)
WO (1) WO2022098990A1 (https=)

Families Citing this family (3)

* 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
WO2024099316A1 (zh) * 2022-11-08 2024-05-16 南京明德新药研发有限公司 含七元杂环的四价缀合基团及其应用
WO2026061476A1 (zh) * 2024-09-19 2026-03-26 长春金赛药业有限责任公司 一种多簇连接子、其制备方法及应用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050222064A1 (en) * 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
EP2563922A1 (en) * 2010-04-26 2013-03-06 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
CA2996722A1 (en) * 2015-07-17 2017-01-26 Arcturus Therapeutics, Inc. Compositions and agents against hepatitis b virus and uses thereof
CA3020585A1 (en) * 2016-04-11 2017-10-19 Arbutus Biopharma Corporation Targeted nucleic acid conjugate compositions
MX2019012280A (es) * 2017-04-11 2020-01-23 Arbutus Biopharma Corp Composiciones dirigidas.
US11324820B2 (en) * 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
JP7473472B2 (ja) * 2017-10-17 2024-04-23 アローヘッド ファーマシューティカルズ インコーポレイテッド アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物
MA55375A (fr) * 2019-03-20 2022-01-26 Arbutus Biopharma Corp Méthodes thérapeutiques de traitement de l'hépatite b

Also Published As

Publication number Publication date
US20240052349A1 (en) 2024-02-15
MX2023005138A (es) 2023-06-23
JP2023548295A (ja) 2023-11-16
CN116472063A (zh) 2023-07-21
WO2022098990A1 (en) 2022-05-12
AU2021376390A1 (en) 2023-06-22
CA3199757A1 (en) 2022-05-12
EP4240369A1 (en) 2023-09-13
IL302530A (en) 2023-07-01
EP4240369A4 (en) 2025-04-23
TW202233243A (zh) 2022-09-01
AU2021376390A9 (en) 2024-10-10

Similar Documents

Publication Publication Date Title
JP7530927B2 (ja) ５’－シクロ－ホスホン酸修飾ヌクレオチド
CN112105625B (zh) 核苷酸前体、核苷酸类似物以及含其的寡聚化合物
CN113874373A (zh) 用于治疗乙型肝炎的治疗方法
JP2023145620A (ja) オリゴヌクレオチド組成物及びその使用方法
KR20230104652A (ko) 변형된 siRNA를 포함하는 표적화된 접합체
JP7754524B2 (ja) Ｂ型肝炎ウイルス遺伝子発現を抑制するｒｎａ阻害剤及びその適用
EP3518934A1 (en) Inhibitors of adenosine 5'-nucleotidase
AU2022295990A1 (en) Methods and compositions for targeting pd-l1
JP2022546055A (ja) 化合物、薬物複合体及びその調製方法と使用
KR20230030056A (ko) Pd-l1을 표적화하기 위한 방법 및 조성물
CN109312342A (zh) 靶向核酸偶联物组合物
CN116368146A (zh) 脱唾液酸糖蛋白受体的新型配体
JP2023505434A (ja) 肝線維症を治療するためのコンジュゲート及び方法
JP7668032B2 (ja) 先端巨大症を処置するためのコンジュゲート及び方法

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20230531

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20241105

Comment text: Request for Examination of Application