UY37803A - Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia - Google Patents

Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia

Info

Publication number
UY37803A
UY37803A UY0001037803A UY37803A UY37803A UY 37803 A UY37803 A UY 37803A UY 0001037803 A UY0001037803 A UY 0001037803A UY 37803 A UY37803 A UY 37803A UY 37803 A UY37803 A UY 37803A
Authority
UY
Uruguay
Prior art keywords
compositions
hemophilia
subject
methods
treat
Prior art date
Application number
UY0001037803A
Other languages
English (en)
Spanish (es)
Inventor
Akin Akinc
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 UY37803A publication Critical patent/UY37803A/es

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
    • 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
    • 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
    • C12N2310/10—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Hematology (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)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
UY0001037803A 2017-07-10 2018-07-10 Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia UY37803A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762530518P 2017-07-10 2017-07-10
US201762599223P 2017-12-15 2017-12-15
US201862614111P 2018-01-05 2018-01-05
US201862673424P 2018-05-18 2018-05-18

Publications (1)

Publication Number Publication Date
UY37803A true UY37803A (es) 2019-02-28

Family

ID=63036452

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001037803A UY37803A (es) 2017-07-10 2018-07-10 Métodos y composiciones para tratar un evento de sangrado en un sujeto que padece hemofilia

Country Status (15)

Country Link
US (2) US20200163987A1 (enExample)
EP (2) EP3651852A1 (enExample)
JP (3) JP2020530442A (enExample)
KR (2) KR20200026960A (enExample)
CN (1) CN111093771A (enExample)
AU (2) AU2018301801A1 (enExample)
BR (1) BR112020000400A2 (enExample)
CA (1) CA3069150A1 (enExample)
CO (1) CO2020001354A2 (enExample)
IL (1) IL271646A (enExample)
MX (1) MX2025007819A (enExample)
SG (1) SG11201912243PA (enExample)
TW (2) TW202434265A (enExample)
UY (1) UY37803A (enExample)
WO (1) WO2019014187A1 (enExample)

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TW202216172A (zh) * 2020-06-22 2022-05-01 美商健臻公司 治療血友病之方法及組成物
WO2022076835A1 (en) * 2020-10-09 2022-04-14 Dyax Corp. Methods and apparatuses for modeling adamts13 and von willebrand factor interactions
IL303452A (en) * 2020-12-06 2023-08-01 Genzyme Corp Treatment of hemophilia with fitusiran
WO2022120291A1 (en) * 2020-12-06 2022-06-09 Genzyme Corporation Treatment of hemophilia with fitusiran
KR102826316B1 (ko) 2021-08-19 2025-06-27 (주) 바텍에스이씨 액체 누설에 의한 폭발 위험도 산출 장치 및 방법
KR102755621B1 (ko) 2021-08-19 2025-01-21 (주) 바텍에스이씨 폭발 위험도 산출 장치 및 방법
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
JP2025519491A (ja) 2022-06-08 2025-06-26 ジェンザイム・コーポレーション 小児患者における血友病ａ及びｂの処置のためのフィツシラン
EP4611848A1 (en) 2022-10-31 2025-09-10 Sanofi Device body for a drug delivery device, assembly for a drug delivery device and drug delivery device
CN120187476A (zh) 2022-10-31 2025-06-20 赛诺菲 容器固持器以及包括该容器固持器的药物递送装置
EP4611854A1 (en) 2022-10-31 2025-09-10 Sanofi Drug delivery device having a two-part user indicator
EP4611853A1 (en) 2022-10-31 2025-09-10 Sanofi Assembly for a drug delivery device
WO2024094706A1 (en) 2022-10-31 2024-05-10 Sanofi Audible indicator, indicator holder and method of assembling an audible indicator
EP4611850A1 (en) 2022-10-31 2025-09-10 Sanofi Arrangement for a drug delivery device, drug delivery device and method for assembly
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
EP4611852A1 (en) 2022-10-31 2025-09-10 Sanofi Front sub-assembly for a drug delivery device
EP4611846A1 (en) 2022-10-31 2025-09-10 Sanofi Plunger for expelling a drug, drug delivery device, rear sub-assembly and corresponding methods
WO2024094700A1 (en) 2022-10-31 2024-05-10 Sanofi Method for assembling an assembly for a drug delivery device and drug delivery device.
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WO2024251793A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
WO2024251800A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
WO2024251795A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
WO2024251802A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device and method for decoupling a drug administration element and at least one part of a drug delivery device
WO2024251797A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
WO2024251792A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
WO2024251794A1 (en) 2023-06-05 2024-12-12 Sanofi Piercing closure assistance member, drug delivery device and method
WO2024251796A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device having two drug delivery arrangements
WO2024251798A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device
WO2024251799A1 (en) 2023-06-05 2024-12-12 Sanofi Self-driven medicament container and medicament delivery device
WO2024251801A1 (en) 2023-06-05 2024-12-12 Sanofi Drug delivery device with laterally flexible needle sleeve
WO2025059635A1 (en) 2023-09-15 2025-03-20 Genzyme Corporation Treatment of hemophilia a with fitusiran
WO2025162927A1 (en) 2024-01-29 2025-08-07 Sanofi Purifying device, system and method for purifying a needle for delivering a medicament
WO2025162928A1 (en) 2024-01-29 2025-08-07 Sanofi Needle shroud unlock device for a medicament delivery device
WO2025162926A1 (en) 2024-01-29 2025-08-07 Sanofi Needle arrangement, drug delivery device comprising the needle arrangement and method for operating the drug delivery device
WO2025171279A1 (en) 2024-02-09 2025-08-14 Genzyme Corporation Treatment of hemophilia with fitusiran
WO2025212516A1 (en) 2024-04-01 2025-10-09 Genzyme Corporation Treatment of factor x deficiency with fitusiran
CN118557756B (zh) * 2024-05-24 2025-04-29 华中科技大学同济医学院附属协和医院 一种基于aav的遗传性抗凝血酶缺乏的基因疗法

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