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WO2024165571A3 - Inhibitors of expression and/or function - Google Patents

Inhibitors of expression and/or function Download PDF

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Publication number
WO2024165571A3
WO2024165571A3 PCT/EP2024/052934 EP2024052934W WO2024165571A3 WO 2024165571 A3 WO2024165571 A3 WO 2024165571A3 EP 2024052934 W EP2024052934 W EP 2024052934W WO 2024165571 A3 WO2024165571 A3 WO 2024165571A3
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.)
Ceased
Application number
PCT/EP2024/052934
Other languages
French (fr)
Other versions
WO2024165571A2 (en
Inventor
Alan Victor WHITMORE
Sophia MALANDRAKI-MILLER
Ines DE SANTIAGO
Natalie PURSELL
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
Application filed by E Therapeutics PLC filed Critical E Therapeutics PLC
Priority to EP24703564.5A priority Critical patent/EP4662315A2/en
Priority to AU2024217327A priority patent/AU2024217327A1/en
Priority to KR1020257029426A priority patent/KR20250162632A/en
Priority to IL322517A priority patent/IL322517A/en
Publication of WO2024165571A2 publication Critical patent/WO2024165571A2/en
Publication of WO2024165571A3 publication Critical patent/WO2024165571A3/en
Priority to PCT/EP2024/086234 priority patent/WO2025125576A2/en
Priority to PCT/EP2024/088248 priority patent/WO2025133348A1/en
Priority to MX2025009171A priority patent/MX2025009171A/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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
    • 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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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

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

Abstract

The present invention relates to inhibitors, and compositions containing inhibitors, and uses of the same in the treatment or prevention of a metabolic disease or disorder, such as a metabolic disease or disorder associated with non-alcoholic fatty liver disease (NAFLD), and/or obesity and/or a disease or disorder associated with adipogenesis.
PCT/EP2024/052934 2023-02-06 2024-02-06 Inhibitors of expression and/or function Ceased WO2024165571A2 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
EP24703564.5A EP4662315A2 (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
AU2024217327A AU2024217327A1 (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
KR1020257029426A KR20250162632A (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
IL322517A IL322517A (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
PCT/EP2024/086234 WO2025125576A2 (en) 2023-12-15 2024-12-13 Inhibitors of expression and/or function
PCT/EP2024/088248 WO2025133348A1 (en) 2023-12-22 2024-12-20 Inhibitors of expression and/or function
MX2025009171A MX2025009171A (en) 2023-02-06 2025-08-05 Inhibitors of expression and/or function

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
EP23155094.8 2023-02-06
EP23155094 2023-02-06
EP23217288.2 2023-12-15
EP23217288 2023-12-15
EP23219939.8 2023-12-22
EP23219939 2023-12-22

Publications (2)

Publication Number Publication Date
WO2024165571A2 WO2024165571A2 (en) 2024-08-15
WO2024165571A3 true WO2024165571A3 (en) 2024-10-10

Family

ID=89845100

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2024/052934 Ceased WO2024165571A2 (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function

Country Status (1)

Country Link
WO (1) WO2024165571A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025125576A2 (en) * 2023-12-15 2025-06-19 E-Therapeutics Plc Inhibitors of expression and/or function
WO2025133348A1 (en) * 2023-12-22 2025-06-26 E-Therapeutics Plc Inhibitors of expression and/or function

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