TWI843703B - 於患有血友病之個體中治療出血事件之方法及組成物 - Google Patents

於患有血友病之個體中治療出血事件之方法及組成物 Download PDF

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TWI843703B
TWI843703B TW107123784A TW107123784A TWI843703B TW I843703 B TWI843703 B TW I843703B TW 107123784 A TW107123784 A TW 107123784A TW 107123784 A TW107123784 A TW 107123784A TW I843703 B TWI843703 B TW I843703B
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effective amount
sense strand
bleeding
patient
medically effective
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TW201920670A (zh
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艾金 阿肯
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美商健贊公司
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/36Blood coagulation or fibrinolysis factors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
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    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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TW107123784A 2017-07-10 2018-07-10 於患有血友病之個體中治療出血事件之方法及組成物 TWI843703B (zh)

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US201762530518P 2017-07-10 2017-07-10
US62/530,518 2017-07-10
US201762599223P 2017-12-15 2017-12-15
US62/599,223 2017-12-15
US201862614111P 2018-01-05 2018-01-05
US62/614,111 2018-01-05
US201862673424P 2018-05-18 2018-05-18
US62/673,424 2018-05-18

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TW201920670A TW201920670A (zh) 2019-06-01
TWI843703B true TWI843703B (zh) 2024-06-01

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TW107123784A TWI843703B (zh) 2017-07-10 2018-07-10 於患有血友病之個體中治療出血事件之方法及組成物

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US (2) US20200163987A1 (enExample)
EP (2) EP3651852A1 (enExample)
JP (3) JP2020530442A (enExample)
KR (2) KR20250159269A (enExample)
CN (1) CN111093771A (enExample)
AU (2) AU2018301801A1 (enExample)
BR (1) BR112020000400A2 (enExample)
CA (1) CA3069150A1 (enExample)
CO (1) CO2020001354A2 (enExample)
IL (1) IL271646A (enExample)
MX (1) MX2025007819A (enExample)
SG (1) SG11201912243PA (enExample)
TW (2) TW202434265A (enExample)
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TW202342065A (zh) 2021-12-22 2023-11-01 美商健臻公司 治療血友病的方法和組成物
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