JP2023540429A - てんかんを治療するための方法及び組成物 - Google Patents

てんかんを治療するための方法及び組成物 Download PDF

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Publication number
JP2023540429A
JP2023540429A JP2023501422A JP2023501422A JP2023540429A JP 2023540429 A JP2023540429 A JP 2023540429A JP 2023501422 A JP2023501422 A JP 2023501422A JP 2023501422 A JP2023501422 A JP 2023501422A JP 2023540429 A JP2023540429 A JP 2023540429A
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seq
sequence
nucleic acid
sequence identity
contiguous nucleotides
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JP2023540429A5 (https=
Inventor
ヴァレリー クレペル，
クリストフ ミュル，
セリーヌ ボアロー，
セヴェリーヌ デフォルジュ，
オリヴィエ ダノス，
アンドリュー マーサー，
リチャード ポーター，
エイプリル アール． ティープ，
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Aix Marseille Universite
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
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Aix Marseille Universite
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
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Publication of JP2023540429A publication Critical patent/JP2023540429A/ja
Publication of JP2023540429A5 publication Critical patent/JP2023540429A5/ja
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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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
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  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
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  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2023501422A 2020-07-10 2021-07-09 てんかんを治療するための方法及び組成物 Pending JP2023540429A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063050742P 2020-07-10 2020-07-10
US63/050,742 2020-07-10
US202163137669P 2021-01-14 2021-01-14
US63/137,669 2021-01-14
US202163185699P 2021-05-07 2021-05-07
US63/185,699 2021-05-07
PCT/US2021/041089 WO2022011262A1 (en) 2020-07-10 2021-07-09 Methods and compositions for treating epilepsy

Publications (2)

Publication Number Publication Date
JP2023540429A true JP2023540429A (ja) 2023-09-25
JP2023540429A5 JP2023540429A5 (https=) 2024-07-19

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JP2023501422A Pending JP2023540429A (ja) 2020-07-10 2021-07-09 てんかんを治療するための方法及び組成物

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US (1) US20240018524A1 (https=)
EP (1) EP4179091A1 (https=)
JP (1) JP2023540429A (https=)
KR (1) KR20230050336A (https=)
CN (1) CN116113697A (https=)
AU (1) AU2021305665A1 (https=)
BR (1) BR112023000428A2 (https=)
CA (1) CA3177613A1 (https=)
IL (1) IL299771A (https=)
WO (1) WO2022011262A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240218397A1 (en) * 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
US20230039652A1 (en) * 2021-07-09 2023-02-09 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
WO2024054850A1 (en) * 2022-09-06 2024-03-14 The Trustees Of Princeton University Rna-based compositions and methods of use thereof
TW202430228A (zh) * 2022-10-10 2024-08-01 法商尤尼克爾法國公司 治療癲癇之方法及組成物

Citations (3)

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WO2005124343A2 (en) * 2004-06-16 2005-12-29 Galapagos N.V. Methods for modulating bone tissue formation, orthogenic agents and pharmaceutical compositions
WO2015036618A1 (en) * 2013-09-16 2015-03-19 INSERM (Institut National de la Santé et de la Recherche Médicale) Method and pharmaceutical composition for use in the treatment of epilepsy
US20180064748A1 (en) * 2015-03-27 2018-03-08 Yeda Research And Development Co. Ltd. Methods of treating motor neuron diseases

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