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
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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]
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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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    • C12N2330/50—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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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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Country Link
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)

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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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