AU2021296786A1 - Methods and compositions for treating hemophilia - Google Patents

Methods and compositions for treating hemophilia Download PDF

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Publication number
AU2021296786A1
AU2021296786A1 AU2021296786A AU2021296786A AU2021296786A1 AU 2021296786 A1 AU2021296786 A1 AU 2021296786A1 AU 2021296786 A AU2021296786 A AU 2021296786A AU 2021296786 A AU2021296786 A AU 2021296786A AU 2021296786 A1 AU2021296786 A1 AU 2021296786A1
Authority
AU
Australia
Prior art keywords
patient
fitusiran
hemophilia
inhibitors
qol
Prior art date
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
Application number
AU2021296786A
Other languages
English (en)
Inventor
Shauna Andersson
Pronabesh Dasmahapatra
Baisong Mei
Qifeng Yu
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
Original Assignee
Genzyme Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of AU2021296786A1 publication Critical patent/AU2021296786A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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

Landscapes

  • 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)
AU2021296786A 2020-06-22 2021-06-22 Methods and compositions for treating hemophilia Pending AU2021296786A1 (en)

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
AU2021296786A1 true AU2021296786A1 (en) 2023-02-23

Family

ID=76943130

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021296786A Pending AU2021296786A1 (en) 2020-06-22 2021-06-22 Methods and compositions for treating hemophilia

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
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
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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