CN115942940A - 治疗血友病的方法及组合物 - Google Patents

治疗血友病的方法及组合物 Download PDF

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Publication number
CN115942940A
CN115942940A CN202180044790.1A CN202180044790A CN115942940A CN 115942940 A CN115942940 A CN 115942940A CN 202180044790 A CN202180044790 A CN 202180044790A CN 115942940 A CN115942940 A CN 115942940A
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CN
China
Prior art keywords
patient
hemophilia
qol
optionally
fituslan
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Pending
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CN202180044790.1A
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English (en)
Chinese (zh)
Inventor
梅柏松
S·安德森
俞旗峰
P·达斯玛哈帕特拉
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Genzyme Corp
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Genzyme Corp
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Publication of CN115942940A publication Critical patent/CN115942940A/zh
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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)
CN202180044790.1A 2020-06-22 2021-06-22 治疗血友病的方法及组合物 Pending CN115942940A (zh)

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
CN115942940A true CN115942940A (zh) 2023-04-07

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ID=76943130

Family Applications (1)

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CN202180044790.1A Pending CN115942940A (zh) 2020-06-22 2021-06-22 治疗血友病的方法及组合物

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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=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (3)

* 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108601795A (zh) * 2015-12-07 2018-09-28 建新公司 用于治疗serpinc1相关病症的方法和组合物
CN111093771A (zh) * 2017-07-10 2020-05-01 建新公司 在患有血友病的受试者中治疗出血事件的方法和组合物

Family Cites Families (1)

* 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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108601795A (zh) * 2015-12-07 2018-09-28 建新公司 用于治疗serpinc1相关病症的方法和组合物
CN111093771A (zh) * 2017-07-10 2020-05-01 建新公司 在患有血友病的受试者中治疗出血事件的方法和组合物

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
JIMENEZ-YUSTE VICTOR ET AL: ""Practical considerations for nonfactor-replacement therapies in the treatment of haemophilia with inhibitors"", HAEMOPHILIA, vol. 27, no. 3, 1 May 2021 (2021-05-01), pages 340 - 350 *
JOHN PASI ET AL: ""Targeting of Anti thrombin in Hemophilia A or B with RNAi Therapy"", THE NEW ENGLAND JOURNAL OF MEDICINE, vol. 377, no. 9, 31 August 2017 (2017-08-31), pages 819 - 828 *
MIESBACH WOLFGANG ET AL: ""Current and Future Options of Haemophilia A Treatments"", EXPERT OPINION ON BIOLOGICAL THERAPY, 1 April 2021 (2021-04-01), pages 1 - 8, XP055841466, DOI: 10.1080/14712598.2021.1908993 *
PIPE STEVEN ET AL: ""Fitusiran, an RNAi Therapeutic Targeting Anti thrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events"", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, vol. 134, 13 November 2019 (2019-11-13), pages 1138 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
WO2021262695A1 (en) 2021-12-30
KR20230027277A (ko) 2023-02-27
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
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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