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EA201690590A1 - ТЕРАПИЯ НЕДОСТАТОЧНОСТИ АРГИНИНОСУКЦИНАТ-СИНТЕТАЗЫ С ПОМОЩЬЮ мРНК - Google Patents

ТЕРАПИЯ НЕДОСТАТОЧНОСТИ АРГИНИНОСУКЦИНАТ-СИНТЕТАЗЫ С ПОМОЩЬЮ мРНК

Info

Publication number
EA201690590A1
EA201690590A1 EA201690590A EA201690590A EA201690590A1 EA 201690590 A1 EA201690590 A1 EA 201690590A1 EA 201690590 A EA201690590 A EA 201690590A EA 201690590 A EA201690590 A EA 201690590A EA 201690590 A1 EA201690590 A1 EA 201690590A1
Authority
EA
Eurasian Patent Office
Prior art keywords
mrna
lipids
synthetasis
argininosuccinate
insufficiency
Prior art date
Application number
EA201690590A
Other languages
English (en)
Inventor
Майкл Хартлейн
Франк Дероза
Лианн Смит
Original Assignee
Шир Хьюман Дженетик Терапис, Инк.
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=51905391&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=EA201690590(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Шир Хьюман Дженетик Терапис, Инк. filed Critical Шир Хьюман Дженетик Терапис, Инк.
Publication of EA201690590A1 publication Critical patent/EA201690590A1/ru

Links

Classifications

    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/53—Ligases (6)
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y603/00—Ligases forming carbon-nitrogen bonds (6.3)
    • C12Y603/04—Other carbon-nitrogen ligases (6.3.4)
    • C12Y603/04005—Argininosuccinate synthase (6.3.4.5)
    • 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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Microbiology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

В настоящем изобретении, среди прочего, предложены способы лечения недостаточности аргининсукцинатсинтазы (ASD), включающие введение нуждающемуся в лечении субъекту эффективных доз композиции, содержащей мРНК, кодирующую аргининсукцинатсинтазу (ASS1), с такими интервалами, что это приводит к снижению выраженности, степени тяжести или частоты появления, или к отсрочке проявления по меньшей мере одного симптома или признака ASD. В некоторых вариантах реализации изобретения мРНК инкапсулирована в липосому, содержащую один или более катионных липидов, один или более некатионных липидов, один или более липидов на основе холестерина и один или более ПЭГ-модифицированных липидов.
EA201690590A 2013-10-22 2014-10-22 ТЕРАПИЯ НЕДОСТАТОЧНОСТИ АРГИНИНОСУКЦИНАТ-СИНТЕТАЗЫ С ПОМОЩЬЮ мРНК EA201690590A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361894294P 2013-10-22 2013-10-22
PCT/US2014/061841 WO2015061500A1 (en) 2013-10-22 2014-10-22 Mrna therapy for argininosuccinate synthetase deficiency

Publications (1)

Publication Number Publication Date
EA201690590A1 true EA201690590A1 (ru) 2016-12-30

Family

ID=51905391

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201690590A EA201690590A1 (ru) 2013-10-22 2014-10-22 ТЕРАПИЯ НЕДОСТАТОЧНОСТИ АРГИНИНОСУКЦИНАТ-СИНТЕТАЗЫ С ПОМОЩЬЮ мРНК

Country Status (11)

Country Link
US (2) US11224642B2 (ru)
EP (3) EP3060303B1 (ru)
JP (4) JP6608815B2 (ru)
CN (1) CN106413811A (ru)
AU (2) AU2014340092B2 (ru)
BR (1) BR112016009014B1 (ru)
CA (1) CA2928188A1 (ru)
EA (1) EA201690590A1 (ru)
ES (2) ES2707966T3 (ru)
MX (1) MX2016005237A (ru)
WO (1) WO2015061500A1 (ru)

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WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
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