KR20220163960A - 변경된 짧은 개재성 핵산(siNA) 분자 및 이의 용도 - Google Patents

변경된 짧은 개재성 핵산(siNA) 분자 및 이의 용도 Download PDF

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KR20220163960A
KR20220163960A KR1020227034100A KR20227034100A KR20220163960A KR 20220163960 A KR20220163960 A KR 20220163960A KR 1020227034100 A KR1020227034100 A KR 1020227034100A KR 20227034100 A KR20227034100 A KR 20227034100A KR 20220163960 A KR20220163960 A KR 20220163960A
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nucleotides
nucleotide sequence
nucleotide
sina
methyl
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Korean (ko)
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레오니드 베이겔만
비벡 쿠마르 라즈완시
마르쿠스 호스바흐
라젠드라 케이 판디
진 홍
락스맨 엘테푸
사울 마르티네즈 몬테로
코스타 엔 틸라니 에스 드
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알리고스 테라퓨틱스 인코포레이티드
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Publication of KR20220163960A publication Critical patent/KR20220163960A/ko
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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
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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
    • C12N15/1131—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 viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • A61P31/20—Antivirals for DNA viruses
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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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2320/30—Special therapeutic applications
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  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Cosmetics (AREA)
KR1020227034100A 2020-03-06 2021-03-05 변경된 짧은 개재성 핵산(siNA) 분자 및 이의 용도 Pending KR20220163960A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062986150P 2020-03-06 2020-03-06
US62/986,150 2020-03-06
US202063109196P 2020-11-03 2020-11-03
US63/109,196 2020-11-03
PCT/US2021/021199 WO2021178885A1 (en) 2020-03-06 2021-03-05 Modified short interfering nucleic acid (sina) molecules and uses thereof

Publications (1)

Publication Number Publication Date
KR20220163960A true KR20220163960A (ko) 2022-12-12

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US (4) US20220364096A1 (enExample)
EP (1) EP4121533A1 (enExample)
JP (2) JP2023515664A (enExample)
KR (1) KR20220163960A (enExample)
CN (1) CN115485384A (enExample)
AU (1) AU2021230601A1 (enExample)
BR (1) BR112022017287A2 (enExample)
CA (1) CA3170466A1 (enExample)
CL (1) CL2022002379A1 (enExample)
CO (1) CO2022014258A2 (enExample)
GE (2) GEP20257742B (enExample)
IL (1) IL296045A (enExample)
MX (1) MX2022011012A (enExample)
PE (1) PE20221578A1 (enExample)
PH (1) PH12022552163A1 (enExample)
TW (1) TW202208625A (enExample)
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US20220364096A1 (en) 2022-11-17
JP2023515664A (ja) 2023-04-13
US20220177888A1 (en) 2022-06-09
PE20221578A1 (es) 2022-10-06
IL296045A (en) 2022-10-01
JP2026102645A (ja) 2026-06-23
CO2022014258A2 (es) 2022-12-30
US20230365970A1 (en) 2023-11-16
WO2021178885A1 (en) 2021-09-10
CL2022002379A1 (es) 2023-07-28
EP4121533A1 (en) 2023-01-25
US12129469B2 (en) 2024-10-29
CA3170466A1 (en) 2021-09-10
PH12022552163A1 (en) 2024-01-29
MX2022011012A (es) 2022-10-07
CN115485384A (zh) 2022-12-16
US20230332153A1 (en) 2023-10-19
AU2021230601A2 (en) 2022-10-20
BR112022017287A2 (pt) 2022-11-16
GEP20257742B (en) 2025-03-10
TW202208625A (zh) 2022-03-01
GEAP202416053A (en) 2024-10-10
AU2021230601A1 (en) 2022-10-06
US11549110B2 (en) 2023-01-10

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