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WO2025085810A3 - Antisense oligomers for treatment of centronuclear myopathies - Google Patents

Antisense oligomers for treatment of centronuclear myopathies Download PDF

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Publication number
WO2025085810A3
WO2025085810A3 PCT/US2024/052063 US2024052063W WO2025085810A3 WO 2025085810 A3 WO2025085810 A3 WO 2025085810A3 US 2024052063 W US2024052063 W US 2024052063W WO 2025085810 A3 WO2025085810 A3 WO 2025085810A3
Authority
WO
WIPO (PCT)
Prior art keywords
treatment
antisense oligomers
dnm2
subject
pharmaceutically acceptable
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/US2024/052063
Other languages
French (fr)
Other versions
WO2025085810A2 (en
Inventor
Noa LIBERMAN-ISAKOV
Ryan Oliver
Craig Mcintosh
Stephen Donald Wilton
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.)
Murdoch University
Sarepta Therapeutics Inc
Original Assignee
Murdoch University
Sarepta 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 Murdoch University, Sarepta Therapeutics Inc filed Critical Murdoch University
Publication of WO2025085810A2 publication Critical patent/WO2025085810A2/en
Publication of WO2025085810A3 publication Critical patent/WO2025085810A3/en
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/3513—Protein; Peptide
    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Provided herein are oligonucleotides/oligomers, peptide-oligonucleotide-conjugates, and targeting sequences complementary to a target region within a pre-mRNA of the human dynamin 2 (DNM2) gene. Also provided herein are methods of treating a disease or disorder associated with aberrant expression of DNM2 in a subject in need thereof, comprising administering to the subject the antisense oligomer, or a pharmaceutically acceptable salt thereof, or pharmaceutical composition comprising the antisense oligomer, or a pharmaceutically acceptable salt thereof.
PCT/US2024/052063 2023-10-18 2024-10-18 Antisense oligomers for treatment of centronuclear myopathies Pending WO2025085810A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363591174P 2023-10-18 2023-10-18
US63/591,174 2023-10-18

Publications (2)

Publication Number Publication Date
WO2025085810A2 WO2025085810A2 (en) 2025-04-24
WO2025085810A3 true WO2025085810A3 (en) 2025-05-30

Family

ID=93455982

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/052063 Pending WO2025085810A2 (en) 2023-10-18 2024-10-18 Antisense oligomers for treatment of centronuclear myopathies

Country Status (1)

Country Link
WO (1) WO2025085810A2 (en)

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WO2018115477A1 (en) * 2016-12-23 2018-06-28 Universite De Strasbourg Dynamin 2 inhibitor for the treatment of myotonic dystrophy
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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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WO2018115477A1 (en) * 2016-12-23 2018-06-28 Universite De Strasbourg Dynamin 2 inhibitor for the treatment of myotonic dystrophy
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CN111020710A (en) * 2018-10-10 2020-04-17 珠海铂华生物工程有限公司 ctDNA high-throughput detection of hematopoietic and lymphoid tissue tumors
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