WO2024023267A3 - Nucleic acid compounds - Google Patents

Nucleic acid compounds Download PDF

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Publication number
WO2024023267A3
WO2024023267A3 PCT/EP2023/070927 EP2023070927W WO2024023267A3 WO 2024023267 A3 WO2024023267 A3 WO 2024023267A3 EP 2023070927 W EP2023070927 W EP 2023070927W WO 2024023267 A3 WO2024023267 A3 WO 2024023267A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
acid compounds
compounds
present
methods
Prior art date
Application number
PCT/EP2023/070927
Other languages
French (fr)
Other versions
WO2024023267A2 (en
Inventor
Amy MCCARTHY
Graham CRAGGS
James LONGDEN
Ines DE SANTIAGO
Duncan Brown
Ahmad Ali MORTAZAVI
Viviana MANNELLA
Muthusamy Jayaraman
Alexandre DEBACKER
Adrian James MOGG
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
Publication of WO2024023267A2 publication Critical patent/WO2024023267A2/en
Publication of WO2024023267A3 publication Critical patent/WO2024023267A3/en

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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
    • C12N15/1137—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 enzymes
    • 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 N.A.
    • 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
    • C12N2310/321—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
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01038—Beta-N-acetylglucosaminylglycopeptide beta-1,4-galactosyltransferase (2.4.1.38)

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

Abstract

The present invention provides novel nucleic acid compound suitable for therapeutic use. Additionally, the present invention provides methods of making these compounds, as well as methods of using such compounds for the treatment of various diseases and conditions.
PCT/EP2023/070927 2022-07-27 2023-07-27 Nucleic acid compounds WO2024023267A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202263369619P 2022-07-27 2022-07-27
US63/369,619 2022-07-27
EP23155113 2023-02-06
EP23155113.6 2023-02-06

Publications (2)

Publication Number Publication Date
WO2024023267A2 WO2024023267A2 (en) 2024-02-01
WO2024023267A3 true WO2024023267A3 (en) 2024-03-07

Family

ID=87553928

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2023/070927 WO2024023267A2 (en) 2022-07-27 2023-07-27 Nucleic acid compounds

Country Status (1)

Country Link
WO (1) WO2024023267A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009062199A1 (en) * 2007-11-09 2009-05-14 Fox Chase Cancer Center EGFR/NEDD9/TGF-β LNTERACTOME AND METHODS OF USE THEREOF FOR THE IDENTIFICATION OF AGENTS HAVING EFFICACY IN THE TREATMENT OF HYPERPROLIFERATIVE DISORDERS
WO2009079452A2 (en) * 2007-12-14 2009-06-25 The Brigham And Women's Hospital, Inc. Treatment and prevention of hiv infection
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11560563B2 (en) 2018-04-05 2023-01-24 Silence Therapeutics Gmbh SiRNAs with vinylphosphonate at the 5′ end of the antisense strand

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009062199A1 (en) * 2007-11-09 2009-05-14 Fox Chase Cancer Center EGFR/NEDD9/TGF-β LNTERACTOME AND METHODS OF USE THEREOF FOR THE IDENTIFICATION OF AGENTS HAVING EFFICACY IN THE TREATMENT OF HYPERPROLIFERATIVE DISORDERS
WO2009079452A2 (en) * 2007-12-14 2009-06-25 The Brigham And Women's Hospital, Inc. Treatment and prevention of hiv infection
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DE VITIS CLAUDIA ET AL: "B4GALT1 Is a New Candidate to Maintain the Stemness of Lung Cancer Stem Cells", JOURNAL OF CLINICAL MEDICINE, vol. 8, no. 11, 9 November 2019 (2019-11-09), pages 1928, XP093105092, DOI: 10.3390/jcm8111928 *
ZHOU HUIMIN ET AL: "B4GALT1 gene knockdown inhibits the hedgehog pathway and reverses multidrug resistance in the human leukemia K562/adriamycin-resistant cell line", IUBMB LIFE, vol. 64, no. 11, 29 November 2012 (2012-11-29), Hoboken, USA, pages 889 - 900, XP093101801, ISSN: 1521-6543, DOI: 10.1002/iub.1080 *

Also Published As

Publication number Publication date
WO2024023267A2 (en) 2024-02-01

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