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WO2025003660A3 - Antisense treatment - Google Patents

Antisense treatment Download PDF

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Publication number
WO2025003660A3
WO2025003660A3 PCT/GB2024/051634 GB2024051634W WO2025003660A3 WO 2025003660 A3 WO2025003660 A3 WO 2025003660A3 GB 2024051634 W GB2024051634 W GB 2024051634W WO 2025003660 A3 WO2025003660 A3 WO 2025003660A3
Authority
WO
WIPO (PCT)
Prior art keywords
antisense treatment
treatment
tdp
contributed
splicing
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
PCT/GB2024/051634
Other languages
French (fr)
Other versions
WO2025003660A2 (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
Priority to CN202480043501.XA priority Critical patent/CN121420062A/en
Priority to KR1020267002947A priority patent/KR20260032585A/en
Priority to AU2024309963A priority patent/AU2024309963A1/en
Publication of WO2025003660A2 publication Critical patent/WO2025003660A2/en
Publication of WO2025003660A3 publication Critical patent/WO2025003660A3/en
Anticipated expiration legal-status Critical
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)
  • Biophysics (AREA)
  • Microbiology (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)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)

Abstract

The disclosure provides methods, compounds (molecules) and/or compositions for the treatment and/or prevention of diseases and/or conditions caused or contributed to by TAR DNA-binding protein 43 (TDP-43) dependent mis-splicing of its downstream target genes.
PCT/GB2024/051634 2023-06-29 2024-06-26 Antisense treatment Pending WO2025003660A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
CN202480043501.XA CN121420062A (en) 2023-06-29 2024-06-26 Antisense therapy
KR1020267002947A KR20260032585A (en) 2023-06-29 2024-06-26 antisense therapy
AU2024309963A AU2024309963A1 (en) 2023-06-29 2024-06-26 Antisense treatment

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB2309922.9 2023-06-29
GBGB2309922.9A GB202309922D0 (en) 2023-06-29 2023-06-29 Antisense treatment

Publications (2)

Publication Number Publication Date
WO2025003660A2 WO2025003660A2 (en) 2025-01-02
WO2025003660A3 true WO2025003660A3 (en) 2025-03-06

Family

ID=87557004

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2024/051634 Pending WO2025003660A2 (en) 2023-06-29 2024-06-26 Antisense treatment

Country Status (5)

Country Link
KR (1) KR20260032585A (en)
CN (1) CN121420062A (en)
AU (1) AU2024309963A1 (en)
GB (1) GB202309922D0 (en)
WO (1) WO2025003660A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016205615A1 (en) * 2015-06-17 2016-12-22 The Johns Hopkins University Tdp-43 in degenerative disease
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
WO2022216759A1 (en) * 2021-04-06 2022-10-13 Maze Therapeutics, Inc. Compositions and methods for treating tdp-43 proteinopathy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016205615A1 (en) * 2015-06-17 2016-12-22 The Johns Hopkins University Tdp-43 in degenerative disease
WO2022018155A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Lna oligonucleotides for splice modulation of stmn2
WO2022216759A1 (en) * 2021-04-06 2022-10-13 Maze Therapeutics, Inc. Compositions and methods for treating tdp-43 proteinopathy

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
BROWN ANNA-LEIGH ET AL: "TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A", NATURE,, vol. 603, no. 7899, 23 February 2022 (2022-02-23), pages 131 - 137, XP037707129, DOI: 10.1038/S41586-022-04436-3 *
CAO MAIZE C. ET AL: "A panel of TDP-43-regulated splicing events verify loss of TDP-43 function in amyotrophic lateral sclerosis brain tissue", BIORXIV, 11 May 2023 (2023-05-11), XP093220174, Retrieved from the Internet <URL:https://www.biorxiv.org/content/10.1101/2023.02.03.527079v2.full.pdf> DOI: 10.1101/2023.02.03.527079 *
JONATHAN P. LING ET AL: "TDP-43 repression of nonconserved cryptic exons is compromised in ALS-FTD", SCIENCE, vol. 349, no. 6248, 7 August 2015 (2015-08-07), US, pages 650 - 655, XP055727592, ISSN: 0036-8075, DOI: 10.1126/science.aab0983 *
LING JONATHAN P. ET AL: "Supplementary Material: TDP-43 repression of nonconserved cryptic exons is compromised in ALS-FTD", SCIENCE, 7 August 2015 (2015-08-07), XP093220542, Retrieved from the Internet <URL:https://www.science.org/doi/suppl/10.1126/science.aab0983/suppl_file/ling-sm.pdf> DOI: 10.1126/science.aab0983 *
PARK NA YEON ET AL: "Post-Translational Modifications of ATG4B in the Regulation of Autophagy", CELLS, vol. 11, no. 8, 13 April 2022 (2022-04-13), pages 1330, XP093220187, ISSN: 2073-4409, DOI: 10.3390/cells11081330 *
PERRONE BENEDETTA ET AL: "Alternative Splicing of ALS Genes: Misregulation and Potential Therapies", CELLULAR AND MOLECULAR NEUROBIOLOGY, SPRINGER NEW YORK, US, vol. 40, no. 1, 5 August 2019 (2019-08-05), pages 1 - 14, XP036976756, ISSN: 0272-4340, [retrieved on 20190805], DOI: 10.1007/S10571-019-00717-0 *
TORRES PASCUAL ET AL: "Cryptic exon splicing function of TARDBP interacts with autophagy in nervous tissue", AUTOPHAGY, vol. 14, no. 8, 28 July 2018 (2018-07-28), US, pages 1398 - 1403, XP093220582, ISSN: 1554-8627, DOI: 10.1080/15548627.2018.1474311 *
TORRES PASCUAL ET AL: "Supplemental material: Cryptic exon splicing function of TARDBP interacts with autophagy in nervous tissue", 28 July 2018 (2018-07-28), XP093220590, Retrieved from the Internet <URL:https://www.tandfonline.com/doi/full/10.1080/15548627.2018.1474311#> *

Also Published As

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

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