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WO2025125576A3 - Inhibiteurs d'expression et/ou de fonction - Google Patents

Inhibiteurs d'expression et/ou de fonction

Info

Publication number
WO2025125576A3
WO2025125576A3 PCT/EP2024/086234 EP2024086234W WO2025125576A3 WO 2025125576 A3 WO2025125576 A3 WO 2025125576A3 EP 2024086234 W EP2024086234 W EP 2024086234W WO 2025125576 A3 WO2025125576 A3 WO 2025125576A3
Authority
WO
WIPO (PCT)
Prior art keywords
inhibitors
expression
function
disease
disorder associated
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
PCT/EP2024/086234
Other languages
English (en)
Other versions
WO2025125576A2 (fr
Inventor
Natalie PURSELL
Alan VICTOR WHITMORE
Sophia MALANDRAKI-MILLER
Ines DE SANTIAGO
Gaye SAGINC GIANNOUSTAS
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.)
E Therapeutics PLC
Original Assignee
E Therapeutics PLC
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 PCT/EP2024/052934 external-priority patent/WO2024165571A2/fr
Application filed by E Therapeutics PLC filed Critical E Therapeutics PLC
Priority to PCT/EP2024/088248 priority Critical patent/WO2025133348A1/fr
Publication of WO2025125576A2 publication Critical patent/WO2025125576A2/fr
Publication of WO2025125576A3 publication Critical patent/WO2025125576A3/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap 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
    • C12N2310/32—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
    • C12N2310/32—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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne des inhibiteurs et des compositions contenant des inhibiteurs et leurs utilisations dans le traitement ou la prévention d'une maladie ou d'un trouble métabolique, tels qu'une maladie ou un trouble métabolique associés à la stéatose hépatique non alcoolique (NAFLD), et/ou l'obésité et/ou une maladie ou un trouble associés à l'adipogenèse.
PCT/EP2024/086234 2023-12-15 2024-12-13 Inhibiteurs d'expression et/ou de fonction Pending WO2025125576A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
PCT/EP2024/088248 WO2025133348A1 (fr) 2023-12-22 2024-12-20 Inhibiteurs d'expression et/ou de fonction

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP23217288.2 2023-12-15
EP23217288 2023-12-15
PCT/EP2024/052934 WO2024165571A2 (fr) 2023-02-06 2024-02-06 Inhibiteurs d'expression et/ou de fonction
EPPCT/EP2024/052934 2024-02-06

Publications (2)

Publication Number Publication Date
WO2025125576A2 WO2025125576A2 (fr) 2025-06-19
WO2025125576A3 true WO2025125576A3 (fr) 2025-08-14

Family

ID=94128391

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2024/086234 Pending WO2025125576A2 (fr) 2023-12-15 2024-12-13 Inhibiteurs d'expression et/ou de fonction

Country Status (1)

Country Link
WO (1) WO2025125576A2 (fr)

Citations (6)

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KR20150086442A (ko) * 2014-01-17 2015-07-28 주식회사 바이오인프라 ANT2 siRNA를 함유하는 HER2 표적 항암제에 대한 내성 극복을 위한 조성물
WO2019051402A1 (fr) * 2017-09-11 2019-03-14 Arrowhead Pharmaceuticals, Inc. Agents d'interférence arn et compositions destinés à inhiber l'expression de l'apolipoprotéine c-iii (apoc3)
WO2024165571A2 (fr) * 2023-02-06 2024-08-15 E-Therapeutics Plc Inhibiteurs d'expression et/ou de fonction

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