KR20240141197A - 병원성 fus 변이체와 관련된 질환을 치료하기 위한 조성물 및 방법 - Google Patents

병원성 fus 변이체와 관련된 질환을 치료하기 위한 조성물 및 방법 Download PDF

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Publication number
KR20240141197A
KR20240141197A KR1020247028814A KR20247028814A KR20240141197A KR 20240141197 A KR20240141197 A KR 20240141197A KR 1020247028814 A KR1020247028814 A KR 1020247028814A KR 20247028814 A KR20247028814 A KR 20247028814A KR 20240141197 A KR20240141197 A KR 20240141197A
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fus
antisense oligonucleotide
mrna
disease
subject
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KR1020247028814A
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Korean (ko)
Inventor
리타 메지니
패트릭 안토니 아카리
로렌 루이스 플린
Original Assignee
페론 인스티튜트 포 뉴롤로지컬 앤드 트랜스레이셔널 사이언스 리미티드
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Priority claimed from AU2022900200A external-priority patent/AU2022900200A0/en
Application filed by 페론 인스티튜트 포 뉴롤로지컬 앤드 트랜스레이셔널 사이언스 리미티드 filed Critical 페론 인스티튜트 포 뉴롤로지컬 앤드 트랜스레이셔널 사이언스 리미티드
Publication of KR20240141197A publication Critical patent/KR20240141197A/ko
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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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    • C07—ORGANIC CHEMISTRY
    • 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
    • A61K2121/00—Preparations for use in therapy
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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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • 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
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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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
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  • Biotechnology (AREA)
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  • Epidemiology (AREA)
  • 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)
KR1020247028814A 2022-02-02 2023-02-02 병원성 fus 변이체와 관련된 질환을 치료하기 위한 조성물 및 방법 Pending KR20240141197A (ko)

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 (1)

Publication Number Publication Date
KR20240141197A true KR20240141197A (ko) 2024-09-25

Family

ID=87553059

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247028814A Pending KR20240141197A (ko) 2022-02-02 2023-02-02 병원성 fus 변이체와 관련된 질환을 치료하기 위한 조성물 및 방법

Country Status (8)

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

Also Published As

Publication number Publication date
WO2023147630A1 (en) 2023-08-10
AU2023215555A1 (en) 2024-08-15
EP4473111A1 (en) 2024-12-11
JP2025507279A (ja) 2025-03-18
CN118871584A (zh) 2024-10-29
CA3243618A1 (en) 2023-08-10
US20250382612A1 (en) 2025-12-18

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PA0105 International application

Patent event date: 20240827

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application