JP2025507279A - 病原性ｆｕｓバリアントに関連する疾患を処置するための組成物および方法 - Google Patents

病原性ｆｕｓバリアントに関連する疾患を処置するための組成物および方法 Download PDF

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JP2025507279A
JP2025507279A JP2024545792A JP2024545792A JP2025507279A JP 2025507279 A JP2025507279 A JP 2025507279A JP 2024545792 A JP2024545792 A JP 2024545792A JP 2024545792 A JP2024545792 A JP 2024545792A JP 2025507279 A JP2025507279 A JP 2025507279A
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fus
mrna
antisense oligonucleotide
disease
aons
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JP2025507279A5 (https=
Inventor
リタ メイジニ，
パトリック アンソニー アッカーリー，
ローレン ルイーズ フリン，
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ペロン インスティテュート フォー ニューロロジカル アンド トランスレーショナル サイエンス リミテッド
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • 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
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    • C12N2320/00—Applications; Uses
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    • C12N2320/34—Allele or polymorphism specific uses

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  • Bioinformatics & Cheminformatics (AREA)
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  • Biotechnology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Biophysics (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2024545792A 2022-02-02 2023-02-02 病原性ｆｕｓバリアントに関連する疾患を処置するための組成物および方法 Pending JP2025507279A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2022900200A AU2022900200A0 (en) 2022-02-02 Compositions and Methods for Treating Diseases Associated with Pathogenic FUS Variants
AU2022900200 2022-02-02
PCT/AU2023/050067 WO2023147630A1 (en) 2022-02-02 2023-02-02 Compositions and methods for treating diseases associated with pathogenic fus variants

Publications (2)

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JP2025507279A true JP2025507279A (ja) 2025-03-18
JP2025507279A5 JP2025507279A5 (https=) 2025-12-17

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ID=87553059

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JP2024545792A Pending JP2025507279A (ja) 2022-02-02 2023-02-02 病原性ｆｕｓバリアントに関連する疾患を処置するための組成物および方法

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Country Link
US (1) US20250382612A1 (https=)
EP (1) EP4473111A1 (https=)
JP (1) JP2025507279A (https=)
KR (1) KR20240141197A (https=)
CN (1) CN118871584A (https=)
AU (1) AU2023215555A1 (https=)
CA (1) CA3243618A1 (https=)
WO (1) WO2023147630A1 (https=)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050244851A1 (en) * 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US20140349938A1 (en) * 2011-06-03 2014-11-27 President And Fellows Of Harvard College Methods of diagnosing and treating amyotrophic lateral sclerosis
HRP20250468T1 (hr) * 2019-05-28 2025-07-18 Ionis Pharmaceuticals. Inc. SPOJEVI I METODE ZA SMANJENJE POKAZATELJA FUS-a

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Publication number Publication date
KR20240141197A (ko) 2024-09-25
WO2023147630A1 (en) 2023-08-10
AU2023215555A1 (en) 2024-08-15
EP4473111A1 (en) 2024-12-11
CN118871584A (zh) 2024-10-29
CA3243618A1 (en) 2023-08-10
US20250382612A1 (en) 2025-12-18

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