MA49395A - Polynucléotides codant pour le facteur viii de coagulation - Google Patents

Polynucléotides codant pour le facteur viii de coagulation

Info

Publication number
MA49395A
MA49395A MA049395A MA49395A MA49395A MA 49395 A MA49395 A MA 49395A MA 049395 A MA049395 A MA 049395A MA 49395 A MA49395 A MA 49395A MA 49395 A MA49395 A MA 49395A
Authority
MA
Morocco
Prior art keywords
polynucleotides
factor viii
coagulation factor
viii coding
coding
Prior art date
Application number
MA049395A
Other languages
English (en)
Inventor
Paolo Martini
Carol H Miao
Raj Rajendran
Original Assignee
Modernatx Inc
Seattle Childrens Hospital D/B/A Seattle Childrens Res Institute
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 Modernatx Inc, Seattle Childrens Hospital D/B/A Seattle Childrens Res Institute filed Critical Modernatx Inc
Publication of MA49395A publication Critical patent/MA49395A/fr

Links

Classifications

    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Organic Chemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA049395A 2017-06-14 2018-06-13 Polynucléotides codant pour le facteur viii de coagulation MA49395A (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762519827P 2017-06-14 2017-06-14
US201762589914P 2017-11-22 2017-11-22
US201862663839P 2018-04-27 2018-04-27

Publications (1)

Publication Number Publication Date
MA49395A true MA49395A (fr) 2020-04-22

Family

ID=63013083

Family Applications (1)

Application Number Title Priority Date Filing Date
MA049395A MA49395A (fr) 2017-06-14 2018-06-13 Polynucléotides codant pour le facteur viii de coagulation

Country Status (4)

Country Link
US (1) US20200268666A1 (fr)
EP (1) EP3638292A1 (fr)
MA (1) MA49395A (fr)
WO (1) WO2018232006A1 (fr)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45052A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille
EP4288104A4 (fr) * 2021-02-02 2025-03-26 Ramot at Tel-Aviv University Ltd. Lipides appropriés pour l'administration d'acides nucléiques
US20240279649A1 (en) * 2021-05-11 2024-08-22 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Gene editing for expression of functional factor viii for the treatment of hemophilia
CN115745819B (zh) * 2021-09-03 2025-06-10 广州谷森制药有限公司 阳离子脂质化合物
NL2030022B1 (en) * 2021-12-03 2023-06-21 Wisdom Pharmaceutical Co Ltd SYNTHESIS OF mRNA TRANSMITTER
WO2025045247A1 (fr) * 2023-08-31 2025-03-06 Geneditbio Limited Acides nucléiques codant pour des protéines associées à crispr et leurs utilisations

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