KR20200026960A - 혈우병을 갖는 대상체에서 출혈 사건을 치료하기 위한 방법 및 조성물 - Google Patents

혈우병을 갖는 대상체에서 출혈 사건을 치료하기 위한 방법 및 조성물 Download PDF

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KR20200026960A
KR20200026960A KR1020207003461A KR20207003461A KR20200026960A KR 20200026960 A KR20200026960 A KR 20200026960A KR 1020207003461 A KR1020207003461 A KR 1020207003461A KR 20207003461 A KR20207003461 A KR 20207003461A KR 20200026960 A KR20200026960 A KR 20200026960A
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nucleotides
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hemophilia
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아킨 아킨크
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젠자임 코포레이션
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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020207003461A 2017-07-10 2018-07-10 혈우병을 갖는 대상체에서 출혈 사건을 치료하기 위한 방법 및 조성물 Ceased KR20200026960A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020257035288A KR20250159269A (ko) 2017-07-10 2018-07-10 혈우병을 갖는 대상체에서 출혈 사건을 치료하기 위한 방법 및 조성물

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
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
PCT/US2018/041400 WO2019014187A1 (en) 2017-07-10 2018-07-10 METHODS AND COMPOSITIONS FOR TREATING A BLEEDING EVENT IN A SUBJECT WITH HEMOPHILIA

Related Child Applications (1)

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KR1020257035288A Division KR20250159269A (ko) 2017-07-10 2018-07-10 혈우병을 갖는 대상체에서 출혈 사건을 치료하기 위한 방법 및 조성물

Publications (1)

Publication Number Publication Date
KR20200026960A true KR20200026960A (ko) 2020-03-11

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KR1020207003461A Ceased KR20200026960A (ko) 2017-07-10 2018-07-10 혈우병을 갖는 대상체에서 출혈 사건을 치료하기 위한 방법 및 조성물
KR1020257035288A Pending KR20250159269A (ko) 2017-07-10 2018-07-10 혈우병을 갖는 대상체에서 출혈 사건을 치료하기 위한 방법 및 조성물

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Country Status (17)

Country Link
US (2) US20200163987A1 (https=)
EP (3) EP3651852B1 (https=)
JP (3) JP2020530442A (https=)
KR (2) KR20200026960A (https=)
CN (1) CN111093771A (https=)
AU (2) AU2018301801A1 (https=)
BR (1) BR112020000400A2 (https=)
CA (1) CA3069150A1 (https=)
CO (1) CO2020001354A2 (https=)
IL (1) IL271646A (https=)
MX (1) MX2025007819A (https=)
NZ (1) NZ761103A (https=)
PY (1) PY1855437A (https=)
SG (1) SG11201912243PA (https=)
TW (2) TW202434265A (https=)
UY (1) UY37803A (https=)
WO (1) WO2019014187A1 (https=)

Cited By (2)

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KR20230027675A (ko) 2021-08-19 2023-02-28 (주) 바텍에스이씨 액체 누설에 의한 폭발 위험도 산출 장치 및 방법
KR20230027676A (ko) 2021-08-19 2023-02-28 (주) 바텍에스이씨 폭발 위험도 산출 장치 및 방법

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WO2022076835A1 (en) * 2020-10-09 2022-04-14 Dyax Corp. Methods and apparatuses for modeling adamts13 and von willebrand factor interactions
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WO2022120291A1 (en) 2020-12-06 2022-06-09 Genzyme Corporation Treatment of hemophilia with fitusiran
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
US20240000744A1 (en) 2022-06-08 2024-01-04 Genzyme Corporation Treatment of Hemophilia with Fitusiran
IL317438A (en) 2022-06-08 2025-02-01 Genzyme Corp FITUSIRAN for the treatment of hemophilia A and B in pediatric patients
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EP4611846A1 (en) 2022-10-31 2025-09-10 Sanofi Plunger for expelling a drug, drug delivery device, rear sub-assembly and corresponding methods
CN120500361A (zh) 2022-10-31 2025-08-15 赛诺菲 具有反馈元件的药物递送装置以及用于向药物递送装置的用户提供关于剂量分配过程的反馈的方法
JP2025537695A (ja) 2022-11-02 2025-11-20 ジェンザイム・コーポレーション トロンビン－アンチトロンビンをモデリングするためのシステム及び方法
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