TW202345873A - 調節ｓｃａｐ活性之組合物及方法 - Google Patents

調節ｓｃａｐ活性之組合物及方法 Download PDF

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Publication number
TW202345873A
TW202345873A TW112113992A TW112113992A TW202345873A TW 202345873 A TW202345873 A TW 202345873A TW 112113992 A TW112113992 A TW 112113992A TW 112113992 A TW112113992 A TW 112113992A TW 202345873 A TW202345873 A TW 202345873A
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Taiwan
Prior art keywords
seq
nucleotides
antisense strand
oligonucleotide
sense strand
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TW112113992A
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English (en)
Chinese (zh)
Inventor
鮑伯 戴爾 布朗
亨立克 Ｔ 杜迪克
鄭成文
妮可 亞歷克西斯 施皮格爾曼
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美商戴瑟納製藥股份有限公司
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Publication of TW202345873A publication Critical patent/TW202345873A/zh

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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/50—Physical structure
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    • C12N2320/30—Special therapeutic applications
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  • Plant Pathology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Heart & Thoracic Surgery (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW112113992A 2022-04-15 2023-04-14 調節ｓｃａｐ活性之組合物及方法 TW202345873A (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263363091P 2022-04-15 2022-04-15
US63/363,091 2022-04-15

Publications (1)

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TW202345873A true TW202345873A (zh) 2023-12-01

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US (1) US20230374522A1 (https=)
EP (1) EP4508218A1 (https=)
JP (1) JP2025512445A (https=)
KR (1) KR20250005108A (https=)
CN (1) CN119013402A (https=)
AR (1) AR129073A1 (https=)
AU (1) AU2023254798A1 (https=)
CA (1) CA3245064A1 (https=)
IL (1) IL315878A (https=)
MX (1) MX2024012729A (https=)
TW (1) TW202345873A (https=)
WO (1) WO2023201043A1 (https=)

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MX2024012729A (es) 2024-11-08
JP2025512445A (ja) 2025-04-17
CA3245064A1 (en) 2023-10-19
IL315878A (en) 2024-11-01
KR20250005108A (ko) 2025-01-09
WO2023201043A1 (en) 2023-10-19
AR129073A1 (es) 2024-07-10
AU2023254798A1 (en) 2024-09-19
EP4508218A1 (en) 2025-02-19
US20230374522A1 (en) 2023-11-23
CN119013402A (zh) 2024-11-22

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