EP4713027A1 - Procédés et compositions pour moduler la méthylation d'un gène cible - Google Patents

Procédés et compositions pour moduler la méthylation d'un gène cible

Info

Publication number
EP4713027A1
EP4713027A1 EP24734281.9A EP24734281A EP4713027A1 EP 4713027 A1 EP4713027 A1 EP 4713027A1 EP 24734281 A EP24734281 A EP 24734281A EP 4713027 A1 EP4713027 A1 EP 4713027A1
Authority
EP
European Patent Office
Prior art keywords
expression
dna
days
weeks
repressor
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
EP24734281.9A
Other languages
German (de)
English (en)
Inventor
Chevaun Danielle MORRISON-SMITH
Jeremiah D. FARELLI
Justin Chen
Charles W. O'DONNELL
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.)
Omega Therapeutics Inc
Original Assignee
Omega 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 Omega Therapeutics Inc filed Critical Omega Therapeutics Inc
Publication of EP4713027A1 publication Critical patent/EP4713027A1/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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric

Landscapes

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

Abstract

La présente divulgation concerne des procédés pour augmenter la méthylation de l'ADN d'un site dans une région du génome comprenant un gène cible, par exemple, à l'aide d'un répresseur d'expression, ou d'un acide nucléique codant pour le répresseur d'expression, comprenant une fraction de ciblage d'ADN qui se lie à une séquence cible dans la région et un domaine effecteur qui méthylate l'ADN (par exemple, une ADN méthyltransférase). Des systèmes comprenant au moins deux répresseurs d'expression sont également divulgués. Les compositions peuvent être utilisées, par exemple, pour diminuer l'expression du gène cible dans une cellule ou un sujet, par exemple, pour le traitement d'une affection associée à l'expression du gène cible.
EP24734281.9A 2023-05-16 2024-05-16 Procédés et compositions pour moduler la méthylation d'un gène cible Pending EP4713027A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363502581P 2023-05-16 2023-05-16
PCT/US2024/029576 WO2024238726A1 (fr) 2023-05-16 2024-05-16 Procédés et compositions pour moduler la méthylation d'un gène cible

Publications (1)

Publication Number Publication Date
EP4713027A1 true EP4713027A1 (fr) 2026-03-25

Family

ID=91585645

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24734281.9A Pending EP4713027A1 (fr) 2023-05-16 2024-05-16 Procédés et compositions pour moduler la méthylation d'un gène cible

Country Status (3)

Country Link
EP (1) EP4713027A1 (fr)
CN (1) CN121772943A (fr)
WO (1) WO2024238726A1 (fr)

Family Cites Families (60)

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Also Published As

Publication number Publication date
WO2024238726A1 (fr) 2024-11-21
CN121772943A (zh) 2026-03-31

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