KR20230027277A - 혈우병을 치료하기 위한 방법 및 조성물 - Google Patents

혈우병을 치료하기 위한 방법 및 조성물 Download PDF

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Publication number
KR20230027277A
KR20230027277A KR1020237002631A KR20237002631A KR20230027277A KR 20230027277 A KR20230027277 A KR 20230027277A KR 1020237002631 A KR1020237002631 A KR 1020237002631A KR 20237002631 A KR20237002631 A KR 20237002631A KR 20230027277 A KR20230027277 A KR 20230027277A
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KR
South Korea
Prior art keywords
patient
hemophilia
pitusiran
inhibitor
qol
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
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KR1020237002631A
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English (en)
Korean (ko)
Inventor
바이송 메이
샤우나 앤더슨
치펑 위
프로나베쉬 다스마하파트라
Original Assignee
젠자임 코포레이션
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Application filed by 젠자임 코포레이션 filed Critical 젠자임 코포레이션
Publication of KR20230027277A publication Critical patent/KR20230027277A/ko
Pending legal-status Critical Current

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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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Rheumatology (AREA)
  • Hematology (AREA)
  • Dermatology (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
KR1020237002631A 2020-06-22 2021-06-22 혈우병을 치료하기 위한 방법 및 조성물 Pending KR20230027277A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063042390P 2020-06-22 2020-06-22
US63/042,390 2020-06-22
PCT/US2021/038445 WO2021262695A1 (en) 2020-06-22 2021-06-22 Methods and compositions for treating hemophilia

Publications (1)

Publication Number Publication Date
KR20230027277A true KR20230027277A (ko) 2023-02-27

Family

ID=76943130

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237002631A Pending KR20230027277A (ko) 2020-06-22 2021-06-22 혈우병을 치료하기 위한 방법 및 조성물

Country Status (12)

Country Link
US (2) US20210393669A1 (https=)
EP (1) EP4168018A1 (https=)
JP (1) JP2023531679A (https=)
KR (1) KR20230027277A (https=)
CN (1) CN115942940A (https=)
AU (1) AU2021296786A1 (https=)
BR (1) BR112022026265A2 (https=)
CA (1) CA3188137A1 (https=)
IL (1) IL299295A (https=)
MX (1) MX2023000164A (https=)
TW (1) TW202216172A (https=)
WO (1) WO2021262695A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240075001A1 (en) * 2022-05-24 2024-03-07 Darren Rubin Methods to reduce hemorrhage and mortality on the battlefield and applications for trauma patients
US20240000744A1 (en) * 2022-06-08 2024-01-04 Genzyme Corporation Treatment of Hemophilia with Fitusiran
CN118460655B (zh) * 2024-07-10 2025-01-28 凯莱英医药集团（天津）股份有限公司 抑制FXI基因表达的siRNA的制备方法
WO2026012399A1 (zh) * 2024-07-10 2026-01-15 正大天晴药业集团股份有限公司 靶向serpinc1的双链核糖核酸

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
KR20180095843A (ko) 2015-12-07 2018-08-28 젠자임 코포레이션 Serpinc1-연관 장애의 치료를 위한 방법 및 조성물
TW202434265A (zh) * 2017-07-10 2024-09-01 美商健贊公司 於患有血友病之個體中治療出血事件之方法及組成物

Also Published As

Publication number Publication date
WO2021262695A1 (en) 2021-12-30
BR112022026265A2 (pt) 2023-01-17
JP2023531679A (ja) 2023-07-25
MX2023000164A (es) 2023-07-06
TW202216172A (zh) 2022-05-01
AU2021296786A1 (en) 2023-02-23
CN115942940A (zh) 2023-04-07
IL299295A (en) 2023-02-01
CA3188137A1 (en) 2021-12-30
US20260014188A1 (en) 2026-01-15
US20210393669A1 (en) 2021-12-23
EP4168018A1 (en) 2023-04-26

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Date Code Title Description
PA0105 International application

Patent event date: 20230120

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20240612

Comment text: Request for Examination of Application