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WO2024165568A3 - Inhibitors of expression and/or function - Google Patents

Inhibitors of expression and/or function Download PDF

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Publication number
WO2024165568A3
WO2024165568A3 PCT/EP2024/052927 EP2024052927W WO2024165568A3 WO 2024165568 A3 WO2024165568 A3 WO 2024165568A3 EP 2024052927 W EP2024052927 W EP 2024052927W WO 2024165568 A3 WO2024165568 A3 WO 2024165568A3
Authority
WO
WIPO (PCT)
Prior art keywords
inhibitors
expression
function
amd
age
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/EP2024/052927
Other languages
French (fr)
Other versions
WO2024165568A2 (en
Inventor
Alan Victor WHITMORE
Christian TENDENG
Natalie PURSELL
Damian ELLE
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 IL322516A priority Critical patent/IL322516A/en
Priority to KR1020257029424A priority patent/KR20250157547A/en
Priority to EP24703562.9A priority patent/EP4662314A2/en
Priority to AU2024218976A priority patent/AU2024218976A1/en
Publication of WO2024165568A2 publication Critical patent/WO2024165568A2/en
Publication of WO2024165568A3 publication Critical patent/WO2024165568A3/en
Priority to MX2025009139A priority patent/MX2025009139A/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
    • C12N2310/32—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
    • C12N2310/343—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
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (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 age-related macular degeneration (AMD).
PCT/EP2024/052927 2023-02-06 2024-02-06 Inhibitors of expression and/or function Ceased WO2024165568A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
IL322516A IL322516A (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
KR1020257029424A KR20250157547A (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
EP24703562.9A EP4662314A2 (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
AU2024218976A AU2024218976A1 (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
MX2025009139A MX2025009139A (en) 2023-02-06 2025-08-05 Inhibitors of expression and/or function

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP23155091 2023-02-06
EP23155091.4 2023-02-06
EP23206777.7 2023-10-30
EP23206777 2023-10-30

Publications (2)

Publication Number Publication Date
WO2024165568A2 WO2024165568A2 (en) 2024-08-15
WO2024165568A3 true WO2024165568A3 (en) 2024-10-17

Family

ID=89845439

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2024/052927 Ceased WO2024165568A2 (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function

Country Status (6)

Country Link
EP (1) EP4662314A2 (en)
KR (1) KR20250157547A (en)
AU (1) AU2024218976A1 (en)
IL (1) IL322516A (en)
MX (1) MX2025009139A (en)
WO (1) WO2024165568A2 (en)

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012005898A2 (en) * 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
WO2019051402A1 (en) * 2017-09-11 2019-03-14 Arrowhead Pharmaceuticals, Inc. Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
WO2019215330A1 (en) * 2018-05-10 2019-11-14 The University Of Manchester Methods for assessing macular degeneration
WO2022058447A1 (en) * 2020-09-16 2022-03-24 The University Of Manchester Complementome assay
WO2022162155A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
WO2022248651A2 (en) * 2021-05-27 2022-12-01 Complement Therapeutics Limited Inhibitory nucleic acids for factor h family proteins

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 (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012005898A2 (en) * 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
WO2019051402A1 (en) * 2017-09-11 2019-03-14 Arrowhead Pharmaceuticals, Inc. Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
WO2019215330A1 (en) * 2018-05-10 2019-11-14 The University Of Manchester Methods for assessing macular degeneration
WO2022058447A1 (en) * 2020-09-16 2022-03-24 The University Of Manchester Complementome assay
WO2022162155A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
WO2022248651A2 (en) * 2021-05-27 2022-12-01 Complement Therapeutics Limited Inhibitory nucleic acids for factor h family proteins

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS: "Safety Data Sheet: Complement Factor H-related 4/CFHR4 Antibody (640212) [Biotin]", 26 June 2021 (2021-06-26), XP093158195, Retrieved from the Internet <URL:https://aero.bio-techne.com/en-us/sds/pdf/fab5980b?_ga=2.237152771.2085111282.1714635023-1692302889.1714635023> *
CIPRIANI VALENTINA ET AL: "Increased circulating levels of factor H-related protein 4 are strongly associated with age-related macular degeneration", NATURE COMMUNICATIONS, vol. 11, no. 1, 7 February 2020 (2020-02-07), XP055852430, Retrieved from the Internet <URL:https://www.nature.com/articles/s41467-020-14499-3.pdf> DOI: 10.1038/s41467-020-14499-3 *
DE JONG SARAH ET AL: "Implications of genetic variation in the complement system in age-related macular degeneration", PROGRESS IN RETINAL AND EYE RESEARCH, OXFORD, GB, vol. 84, 19 February 2021 (2021-02-19), XP086777844, ISSN: 1350-9462, [retrieved on 20210219], DOI: 10.1016/J.PRETEYERES.2021.100952 *
LI MING-HSIN ET AL: "Ligand Characteristics Important to Avidity Interactions of Multivalent Nanoparticles", BIOCONJUGATE CHEMISTRY, vol. 28, no. 6, 11 April 2017 (2017-04-11), US, pages 1649 - 1657, XP055917014, ISSN: 1043-1802, DOI: 10.1021/acs.bioconjchem.7b00098 *
LORÉS-MOTTA LAURA ET AL: "Common haplotypes at the CFH locus and low-frequency variants in CFHR2 and CFHR5 associate with systemic FHR concentrations and age-related macular degeneration", THE AMERICAN JOURNAL OF HUMAN GENETICS, AMERICAN SOCIETY OF HUMAN GENETICS , CHICAGO , IL, US, vol. 108, no. 8, 13 July 2021 (2021-07-13), pages 1367 - 1384, XP086722183, ISSN: 0002-9297, [retrieved on 20210713], DOI: 10.1016/J.AJHG.2021.06.002 *
SUSETTE LAUWEN: "Analysis of hemopexin plasma levels in patients with age-related macular degeneration", MOLECULAR VISION, vol. 28, 31 December 2022 (2022-12-31), US, pages 536 - 543, XP093158420, ISSN: 1090-0535 *

Also Published As

Publication number Publication date
WO2024165568A2 (en) 2024-08-15
EP4662314A2 (en) 2025-12-17
AU2024218976A1 (en) 2025-09-11
IL322516A (en) 2025-10-01
MX2025009139A (en) 2025-10-01
KR20250157547A (en) 2025-11-04

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