EP4728075A2 - Compositions et procédés de modulation de l'apolipoprotéine e - Google Patents

Compositions et procédés de modulation de l'apolipoprotéine e

Info

Publication number
EP4728075A2
EP4728075A2 EP24824221.6A EP24824221A EP4728075A2 EP 4728075 A2 EP4728075 A2 EP 4728075A2 EP 24824221 A EP24824221 A EP 24824221A EP 4728075 A2 EP4728075 A2 EP 4728075A2
Authority
EP
European Patent Office
Prior art keywords
nucleic acid
isolated nucleic
apoe
seq
subject
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
EP24824221.6A
Other languages
German (de)
English (en)
Inventor
Asa Abeliovich
Herve Rhinn
Laura Heckman
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.)
Leal Therapeutics Inc
Original Assignee
Leal Therapeutics Inc
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 Leal Therapeutics Inc filed Critical Leal Therapeutics Inc
Publication of EP4728075A2 publication Critical patent/EP4728075A2/fr
Pending 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
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/322—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
    • C12N2310/341—Gapmers, i.e. of the type ===---===

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

Des aspects de la divulgation concernent des compositions et des procédés de modulation de niveaux, de transcription, d'épissage et/ou de traduction d'un ou de plusieurs transcrits d'ARN (p. ex. des transcrits d'ARNm) dans une cellule ou un sujet. La divulgation est basée, en partie, sur des acides nucléiques isolés qui se lient à des transcrits d'ARNm de gènes impliqués dans des maladies et des troubles neurodégénératifs, par exemple un gène d'apolipoprotéine E (APOE), qui code pour une protéine impliquée dans l'homéostasie lipidique et la formation de plaque amyloïde dans le SNC. Dans certains modes de réalisation, les compositions de la divulgation sont utiles pour traiter des maladies ou des troubles neurodégénératifs, tels que la maladie d'Alzheimer (AD) ou la démence à corps de Lewy.
EP24824221.6A 2023-06-16 2024-06-14 Compositions et procédés de modulation de l'apolipoprotéine e Pending EP4728075A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202363508831P 2023-06-16 2023-06-16
US202363588136P 2023-10-05 2023-10-05
US202463556391P 2024-02-21 2024-02-21
PCT/US2024/034006 WO2024259232A2 (fr) 2023-06-16 2024-06-14 Compositions et procédés de modulation de l'apolipoprotéine e

Publications (1)

Publication Number Publication Date
EP4728075A2 true EP4728075A2 (fr) 2026-04-22

Family

ID=93852762

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24824221.6A Pending EP4728075A2 (fr) 2023-06-16 2024-06-14 Compositions et procédés de modulation de l'apolipoprotéine e

Country Status (2)

Country Link
EP (1) EP4728075A2 (fr)
WO (1) WO2024259232A2 (fr)

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021178374A2 (fr) * 2020-03-05 2021-09-10 Synerk Inc. Composés et procédés de réduction de l'expression de l'apoe

Also Published As

Publication number Publication date
WO2024259232A3 (fr) 2025-04-17
WO2024259232A2 (fr) 2024-12-19

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