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WO2024165568A3 - Inhibiteurs d'expression et/ou de fonction - Google Patents

Inhibiteurs d'expression et/ou de fonction 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
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English (en)
Other versions
WO2024165568A2 (fr
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/ko
Priority to EP24703562.9A priority patent/EP4662314A2/fr
Priority to AU2024218976A priority patent/AU2024218976A1/en
Publication of WO2024165568A2 publication Critical patent/WO2024165568A2/fr
Publication of WO2024165568A3 publication Critical patent/WO2024165568A3/fr
Priority to MX2025009139A priority patent/MX2025009139A/es
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • 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

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  • 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

La présente invention concerne des inhibiteurs, et des compositions contenant des inhibiteurs, ainsi que leurs utilisations dans le traitement ou la prévention de la dégénérescence maculaire liée à l'âge (DMLA).
PCT/EP2024/052927 2023-02-06 2024-02-06 Inhibiteurs d'expression et/ou de fonction Ceased WO2024165568A2 (fr)

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 (ko) 2023-02-06 2024-02-06 발현 및/또는 기능의 억제제
EP24703562.9A EP4662314A2 (fr) 2023-02-06 2024-02-06 Inhibiteurs d'expression et/ou de fonction
AU2024218976A AU2024218976A1 (en) 2023-02-06 2024-02-06 Inhibitors of expression and/or function
MX2025009139A MX2025009139A (es) 2023-02-06 2025-08-05 Inhibidores de la expresion y/o funcion

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 (fr) 2024-08-15
WO2024165568A3 true WO2024165568A3 (fr) 2024-10-17

Family

ID=89845439

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2024/052927 Ceased WO2024165568A2 (fr) 2023-02-06 2024-02-06 Inhibiteurs d'expression et/ou de fonction

Country Status (6)

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

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012005898A2 (fr) * 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Transcriptome de cellules d'ovaire de hamster chinois (cho), arnsi correspondants et utilisations de ceux-ci
WO2012177639A2 (fr) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Biotraitement et bioproduction à l'aide de lignées de cellules aviaires
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)
WO2019215330A1 (fr) * 2018-05-10 2019-11-14 The University Of Manchester Méthodes d'évaluation de la dégénérescence maculaire
WO2022058447A1 (fr) * 2020-09-16 2022-03-24 The University Of Manchester Test du « complémentome »
WO2022162155A1 (fr) * 2021-01-30 2022-08-04 E-Therapeutics Plc Acides nucléiques contenant des nucléotides abasiques
WO2022248651A2 (fr) * 2021-05-27 2022-12-01 Complement Therapeutics Limited Acides nucléiques inhibiteurs pour protéines de la famille du facteur h

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 (fr) * 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Transcriptome de cellules d'ovaire de hamster chinois (cho), arnsi correspondants et utilisations de ceux-ci
WO2012177639A2 (fr) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Biotraitement et bioproduction à l'aide de lignées de cellules aviaires
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)
WO2019215330A1 (fr) * 2018-05-10 2019-11-14 The University Of Manchester Méthodes d'évaluation de la dégénérescence maculaire
WO2022058447A1 (fr) * 2020-09-16 2022-03-24 The University Of Manchester Test du « complémentome »
WO2022162155A1 (fr) * 2021-01-30 2022-08-04 E-Therapeutics Plc Acides nucléiques contenant des nucléotides abasiques
WO2022248651A2 (fr) * 2021-05-27 2022-12-01 Complement Therapeutics Limited Acides nucléiques inhibiteurs pour protéines de la famille du facteur h

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 (fr) 2024-08-15
EP4662314A2 (fr) 2025-12-17
AU2024218976A1 (en) 2025-09-11
IL322516A (en) 2025-10-01
MX2025009139A (es) 2025-10-01
KR20250157547A (ko) 2025-11-04

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