CN118871584A - 用于治疗与致病性fus变体相关的疾病的组合物和方法 - Google Patents

用于治疗与致病性fus变体相关的疾病的组合物和方法 Download PDF

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Publication number
CN118871584A
CN118871584A CN202380027775.5A CN202380027775A CN118871584A CN 118871584 A CN118871584 A CN 118871584A CN 202380027775 A CN202380027775 A CN 202380027775A CN 118871584 A CN118871584 A CN 118871584A
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China
Prior art keywords
fus
antisense oligonucleotide
mrna
aon
disease
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Pending
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CN202380027775.5A
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English (en)
Chinese (zh)
Inventor
R·梅吉尼
P·A·阿卡里
L·L·弗林
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Perrin Institute Of Neurology And Translational Sciences Ltd
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Perrin Institute Of Neurology And Translational Sciences Ltd
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Publication date
Priority claimed from AU2022900200A external-priority patent/AU2022900200A0/en
Application filed by Perrin Institute Of Neurology And Translational Sciences Ltd filed Critical Perrin Institute Of Neurology And Translational Sciences Ltd
Publication of CN118871584A publication Critical patent/CN118871584A/zh
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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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
    • 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
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    • 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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    • C12N2310/00—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
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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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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
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  • Biophysics (AREA)
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  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • 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)
CN202380027775.5A 2022-02-02 2023-02-02 用于治疗与致病性fus变体相关的疾病的组合物和方法 Pending CN118871584A (zh)

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
CN118871584A true CN118871584A (zh) 2024-10-29

Family

ID=87553059

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380027775.5A Pending CN118871584A (zh) 2022-02-02 2023-02-02 用于治疗与致病性fus变体相关的疾病的组合物和方法

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
KR20240141197A (ko) 2024-09-25
WO2023147630A1 (en) 2023-08-10
AU2023215555A1 (en) 2024-08-15
EP4473111A1 (en) 2024-12-11
JP2025507279A (ja) 2025-03-18
CA3243618A1 (en) 2023-08-10
US20250382612A1 (en) 2025-12-18

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