JP2025509242A - Ｇｎａｑ及びｇｎａ１１駆動疾患の治療方法 - Google Patents

Ｇｎａｑ及びｇｎａ１１駆動疾患の治療方法 Download PDF

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JP2025509242A
JP2025509242A JP2024553192A JP2024553192A JP2025509242A JP 2025509242 A JP2025509242 A JP 2025509242A JP 2024553192 A JP2024553192 A JP 2024553192A JP 2024553192 A JP2024553192 A JP 2024553192A JP 2025509242 A JP2025509242 A JP 2025509242A
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acid molecule
nucleic acid
sequence
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JP2025509242A5 (https=
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ベロニカ キンズラー
ダビデ ツェッキン
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Francis Crick Institute Ltd
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Francis Crick Institute Ltd
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Priority claimed from GBGB2203164.5A external-priority patent/GB202203164D0/en
Priority claimed from GBGB2203233.8A external-priority patent/GB202203233D0/en
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Publication of JP2025509242A publication Critical patent/JP2025509242A/ja
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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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Dermatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024553192A 2022-03-07 2023-03-07 Ｇｎａｑ及びｇｎａ１１駆動疾患の治療方法 Pending JP2025509242A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB2203164.5 2022-03-07
GBGB2203164.5A GB202203164D0 (en) 2022-03-07 2022-03-07 Methods of treatment of gnaq and gna11 mosaicism
GBGB2203233.8A GB202203233D0 (en) 2022-03-08 2022-03-08 Methods of treatment of GNAQ and GNA11 driven disease
GB2203233.8 2022-03-08
PCT/EP2023/055817 WO2023170109A1 (en) 2022-03-07 2023-03-07 Methods of treatment of gnaq and gna11 driven disease

Publications (2)

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JP2025509242A true JP2025509242A (ja) 2025-04-11
JP2025509242A5 JP2025509242A5 (https=) 2026-03-16

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JP2024553192A Pending JP2025509242A (ja) 2022-03-07 2023-03-07 Ｇｎａｑ及びｇｎａ１１駆動疾患の治療方法

Country Status (5)

Country Link
US (1) US20250230435A1 (https=)
EP (1) EP4490290A1 (https=)
JP (1) JP2025509242A (https=)
AU (1) AU2023232895A1 (https=)
WO (1) WO2023170109A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025245193A1 (en) * 2024-05-21 2025-11-27 The University Of North Carolina At Chapel Hill Methods and compositions for inhibition of mutant gnaq and gna11

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2746514C (en) * 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
US10544461B2 (en) * 2013-04-16 2020-01-28 The Johns Hopkins University Diagnostic and prognostic test for sturge-weber syndrome, klippel-trenaunay-weber syndrome, and port-wine stains (PWSS)
CN105218668B (zh) * 2015-10-30 2020-03-24 山东农业大学 马耳他型布氏杆菌的EF-Tu蛋白单克隆抗体MAb及其制备方法与应用
US20210403864A1 (en) * 2018-11-06 2021-12-30 Memorial Sloan Kettering Cancer Center Methods and compositions for treating activated g-alpha q mutant cancers or melanocytic malignancies

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AU2023232895A1 (en) 2024-08-29
US20250230435A1 (en) 2025-07-17
EP4490290A1 (en) 2025-01-15
WO2023170109A1 (en) 2023-09-14

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