JP2025500412A - 血友病患者において患者報告アウトカムを改善するのに使用するためのフィツシラン - Google Patents

血友病患者において患者報告アウトカムを改善するのに使用するためのフィツシラン Download PDF

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Publication number
JP2025500412A
JP2025500412A JP2024537896A JP2024537896A JP2025500412A JP 2025500412 A JP2025500412 A JP 2025500412A JP 2024537896 A JP2024537896 A JP 2024537896A JP 2024537896 A JP2024537896 A JP 2024537896A JP 2025500412 A JP2025500412 A JP 2025500412A
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JP
Japan
Prior art keywords
fitusiran
qol
hemophilia
patient
questionnaire
Prior art date
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Pending
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JP2024537896A
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English (en)
Japanese (ja)
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JP2025500412A5 (https=
Inventor
ショーナ・アンデション
ホセ・バルテルト－ホファー
プロナベッシュ・ダズマハパトラ
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Genzyme Corp
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Genzyme Corp
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of JP2025500412A publication Critical patent/JP2025500412A/ja
Publication of JP2025500412A5 publication Critical patent/JP2025500412A5/ja
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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/7056—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing five-membered rings with nitrogen as a ring hetero atom
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024537896A 2021-12-22 2022-12-22 血友病患者において患者報告アウトカムを改善するのに使用するためのフィツシラン Pending JP2025500412A (ja)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US202163293070P 2021-12-22 2021-12-22
US63/293,070 2021-12-22
US202263305550P 2022-02-01 2022-02-01
US63/305,550 2022-02-01
US202263335535P 2022-04-27 2022-04-27
US63/335,535 2022-04-27
US202263359717P 2022-07-08 2022-07-08
US63/359,717 2022-07-08
US202263382223P 2022-11-03 2022-11-03
US63/382,223 2022-11-03
US202263386489P 2022-12-07 2022-12-07
US63/386,489 2022-12-07
PCT/US2022/082213 WO2023122713A1 (en) 2021-12-22 2022-12-22 Fitusiran for use in improving patient-reported outcome in hemophilia patients

Publications (2)

Publication Number Publication Date
JP2025500412A true JP2025500412A (ja) 2025-01-09
JP2025500412A5 JP2025500412A5 (https=) 2026-01-05

Family

ID=85227438

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024537896A Pending JP2025500412A (ja) 2021-12-22 2022-12-22 血友病患者において患者報告アウトカムを改善するのに使用するためのフィツシラン

Country Status (10)

Country Link
US (1) US20250064842A1 (https=)
EP (1) EP4452280A1 (https=)
JP (1) JP2025500412A (https=)
KR (1) KR20240138572A (https=)
AU (1) AU2022419588A1 (https=)
CA (1) CA3243297A1 (https=)
IL (1) IL313677A (https=)
MX (1) MX2024007894A (https=)
TW (1) TW202342065A (https=)
WO (1) WO2023122713A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025171279A1 (en) * 2024-02-09 2025-08-14 Genzyme Corporation Treatment of hemophilia with fitusiran

Family Cites Families (4)

* 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
WO2022120291A1 (en) * 2020-12-06 2022-06-09 Genzyme Corporation Treatment of hemophilia with fitusiran

Also Published As

Publication number Publication date
TW202342065A (zh) 2023-11-01
AU2022419588A1 (en) 2024-08-01
MX2024007894A (es) 2024-08-20
US20250064842A1 (en) 2025-02-27
EP4452280A1 (en) 2024-10-30
WO2023122713A1 (en) 2023-06-29
KR20240138572A (ko) 2024-09-20
IL313677A (en) 2024-08-01
CA3243297A1 (en) 2023-06-29

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