CN120022289A - 血管生成素样7（angptl7）相关疾病的治疗 - Google Patents

血管生成素样7（angptl7）相关疾病的治疗 Download PDF

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Publication number
CN120022289A
CN120022289A CN202411735471.7A CN202411735471A CN120022289A CN 120022289 A CN120022289 A CN 120022289A CN 202411735471 A CN202411735471 A CN 202411735471A CN 120022289 A CN120022289 A CN 120022289A
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sequence
sense strand
antisense strand
sirna
oligonucleotide
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Inventor
奥马里·戈特斯曼
香农·布鲁斯
保罗·布斯克
布赖恩·加耶斯
大卫·路易斯
大卫·罗泽马
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Eperik Co
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Eperik Co
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    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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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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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Ophthalmology & Optometry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
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  • Biochemistry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN202411735471.7A 2019-05-24 2020-05-21 血管生成素样7（angptl7）相关疾病的治疗 Pending CN120022289A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201962852813P 2019-05-24 2019-05-24
US62/852,813 2019-05-24
US201962881906P 2019-08-01 2019-08-01
US62/881,906 2019-08-01
CN202080053392.1A CN114375194B (zh) 2019-05-24 2020-05-21 血管生成素样7（angptl7）相关疾病的治疗
PCT/US2020/034063 WO2020242896A2 (en) 2019-05-24 2020-05-21 Treatment of angiopoietin like 7 (angptl7) related diseases

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CN120022289A true CN120022289A (zh) 2025-05-23

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CN202080053392.1A Active CN114375194B (zh) 2019-05-24 2020-05-21 血管生成素样7（angptl7）相关疾病的治疗

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US (2) US10941404B2 (https=)
EP (1) EP3976059A4 (https=)
JP (2) JP2022535717A (https=)
KR (1) KR20220024153A (https=)
CN (2) CN120022289A (https=)
AU (1) AU2020285639A1 (https=)
CA (1) CA3140917A1 (https=)
WO (1) WO2020242896A2 (https=)

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EP3976059A4 (en) 2024-09-11
US12378556B2 (en) 2025-08-05
AU2020285639A1 (en) 2021-12-23
WO2020242896A3 (en) 2021-02-04
KR20220024153A (ko) 2022-03-03
EP3976059A2 (en) 2022-04-06
US10941404B2 (en) 2021-03-09
CN114375194A (zh) 2022-04-19
US20210163950A1 (en) 2021-06-03
CA3140917A1 (en) 2020-12-03
JP2022535717A (ja) 2022-08-10
CN114375194B (zh) 2024-12-13
WO2020242896A2 (en) 2020-12-03
US20200399640A1 (en) 2020-12-24
JP2025128091A (ja) 2025-09-02

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