KR102776167B1 - 감소된 신장 청소율을 갖는 다중결합 올리고뉴클레오티드 - Google Patents

감소된 신장 청소율을 갖는 다중결합 올리고뉴클레오티드 Download PDF

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KR102776167B1
KR102776167B1 KR1020247000303A KR20247000303A KR102776167B1 KR 102776167 B1 KR102776167 B1 KR 102776167B1 KR 1020247000303 A KR1020247000303 A KR 1020247000303A KR 20247000303 A KR20247000303 A KR 20247000303A KR 102776167 B1 KR102776167 B1 KR 102776167B1
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oligonucleotide
multimeric
multimeric oligonucleotide
oligonucleotides
stranded
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KR20240010750A (ko
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조나단 마일즈 브라운
한스-피터 포른로셔
크리스틴 케이 에이치 노이먼
필립 해드위거
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엠펙 엘에이, 엘.엘.씨.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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    • C12N2320/30—Special therapeutic applications
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Bioinformatics & Cheminformatics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
KR1020247000303A 2017-02-06 2018-02-06 감소된 신장 청소율을 갖는 다중결합 올리고뉴클레오티드 Active KR102776167B1 (ko)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201762455231P 2017-02-06 2017-02-06
US62/455,231 2017-02-06
US201762522363P 2017-06-20 2017-06-20
US62/522,363 2017-06-20
US201762561853P 2017-09-22 2017-09-22
US62/561,853 2017-09-22
PCT/US2018/017062 WO2018145086A1 (en) 2017-02-06 2018-02-06 Multimeric oligonucleotides having decreased kidney clearance
KR1020197023090A KR102623311B1 (ko) 2017-02-06 2018-02-06 감소된 신장 청소율을 갖는 다중결합 올리고뉴클레오티드

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KR20240010750A KR20240010750A (ko) 2024-01-24
KR102776167B1 true KR102776167B1 (ko) 2025-03-05

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KR1020197023090A Active KR102623311B1 (ko) 2017-02-06 2018-02-06 감소된 신장 청소율을 갖는 다중결합 올리고뉴클레오티드

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US (2) US11078484B2 (enExample)
EP (1) EP3576752A4 (enExample)
JP (1) JP7183167B2 (enExample)
KR (2) KR102776167B1 (enExample)
CN (1) CN110248665A (enExample)
AU (1) AU2018215684B2 (enExample)
CA (1) CA3051480A1 (enExample)
IL (2) IL307726A (enExample)
MX (1) MX2019009347A (enExample)
SG (1) SG11201906728TA (enExample)
WO (1) WO2018145086A1 (enExample)

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