EP4735596A2 - Traitement antisens - Google Patents

Traitement antisens

Info

Publication number
EP4735596A2
EP4735596A2 EP24739664.1A EP24739664A EP4735596A2 EP 4735596 A2 EP4735596 A2 EP 4735596A2 EP 24739664 A EP24739664 A EP 24739664A EP 4735596 A2 EP4735596 A2 EP 4735596A2
Authority
EP
European Patent Office
Prior art keywords
tdp
atg4b
disease
ranbp1
compound
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
EP24739664.1A
Other languages
German (de)
English (en)
Inventor
Chris Sibley
Aida CARDONA-ALBERICH
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.)
University of Edinburgh
Original Assignee
University of Edinburgh
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 University of Edinburgh filed Critical University of Edinburgh
Publication of EP4735596A2 publication Critical patent/EP4735596A2/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
    • 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
    • C12N2310/11—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)

Abstract

La divulgation concerne des procédés, des composés (molécules) et/ou des compositions pour le traitement et/ou la prévention de maladies et/ou d'états pathologiques causés par, où auxquels contribuent, un mauvais épissage dépendant de la protéine de liaison à l'ADN TAR 43 (TDP-43) de ses gènes cibles en aval.
EP24739664.1A 2023-06-29 2024-06-26 Traitement antisens Pending EP4735596A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB2309922.9A GB202309922D0 (en) 2023-06-29 2023-06-29 Antisense treatment
PCT/GB2024/051634 WO2025003660A2 (fr) 2023-06-29 2024-06-26 Traitement antisens

Publications (1)

Publication Number Publication Date
EP4735596A2 true EP4735596A2 (fr) 2026-05-06

Family

ID=87557004

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24739664.1A Pending EP4735596A2 (fr) 2023-06-29 2024-06-26 Traitement antisens

Country Status (6)

Country Link
EP (1) EP4735596A2 (fr)
KR (1) KR20260032585A (fr)
CN (1) CN121420062A (fr)
AU (1) AU2024309963A1 (fr)
GB (1) GB202309922D0 (fr)
WO (1) WO2025003660A2 (fr)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10962551B2 (en) * 2015-06-17 2021-03-30 The Johns Hopkins University TDP-43 in degenerative disease
WO2022018155A1 (fr) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucléotides lna pour la modulation d'épissage de stmn2
EP4320236A1 (fr) * 2021-04-06 2024-02-14 Maze Therapeutics, Inc. Compositions et méthodes de traitement de la protéinopathie tdp-43

Also Published As

Publication number Publication date
CN121420062A (zh) 2026-01-27
GB202309922D0 (en) 2023-08-16
WO2025003660A3 (fr) 2025-03-06
AU2024309963A9 (en) 2025-12-11
KR20260032585A (ko) 2026-03-09
WO2025003660A2 (fr) 2025-01-02
AU2024309963A1 (en) 2025-11-27

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