KR20230050336A - 뇌전증을 치료하기 위한 방법과 조성물 - Google Patents

뇌전증을 치료하기 위한 방법과 조성물 Download PDF

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KR20230050336A
KR20230050336A KR1020237004876A KR20237004876A KR20230050336A KR 20230050336 A KR20230050336 A KR 20230050336A KR 1020237004876 A KR1020237004876 A KR 1020237004876A KR 20237004876 A KR20237004876 A KR 20237004876A KR 20230050336 A KR20230050336 A KR 20230050336A
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seq
sequence
nucleic acid
sequence identity
contiguous nucleotides
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KR1020237004876A
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Korean (ko)
Inventor
발레리 크레펠
크리스토프 뮬
셀린 부알로
세브린 드포르주
올리비에 다노스
앤드류 머서
리처드 포터
에이프릴 알. 테페
Original Assignee
인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸)
위니베르시떼 덱스-마르세이유
유니베르시떼 드 보르도
리젠엑스바이오 인크.
상뜨르 나쇼날 드 라 러쉐르쉬 샹띠피끄
콜리브 테라퓨틱스
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Publication of KR20230050336A publication Critical patent/KR20230050336A/ko
Pending legal-status Critical Current

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    • 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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • 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
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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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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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
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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
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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    • 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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  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020237004876A 2020-07-10 2021-07-09 뇌전증을 치료하기 위한 방법과 조성물 Pending KR20230050336A (ko)

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

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KR20230050336A true KR20230050336A (ko) 2023-04-14

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KR1020237004876A Pending KR20230050336A (ko) 2020-07-10 2021-07-09 뇌전증을 치료하기 위한 방법과 조성물

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

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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

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