RU2018121529A - МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD) - Google Patents

МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD) Download PDF

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RU2018121529A
RU2018121529A RU2018121529A RU2018121529A RU2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A
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apociii
compound
use according
stranded
interfering rna
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RU2018121529A
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English (en)
Russian (ru)
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Вероника Дж. АЛЕКСАНДЕР
Николас Дж. ВИНЕЙ
Джозеф Л. УИТЗТУМ
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Ионис Фармасьютикалз, Инк.
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Application filed by Ионис Фармасьютикалз, Инк. filed Critical Ионис Фармасьютикалз, Инк.
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
RU2018121529A 2013-02-14 2014-02-14 МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD) RU2018121529A (ru)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361764969P 2013-02-14 2013-02-14
US61/764,969 2013-02-14
US201361880779P 2013-09-20 2013-09-20
US61/880,779 2013-09-20

Related Parent Applications (1)

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RU2015139055A Division RU2661781C2 (ru) 2013-02-14 2014-02-14 Модулирование экспрессии аполипопротеина с-iii (аросiii) у людей с дефицитом липопротеинлипазы (lpld)

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RU2018121529A true RU2018121529A (ru) 2019-03-06

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RU2015139055A RU2661781C2 (ru) 2013-02-14 2014-02-14 Модулирование экспрессии аполипопротеина с-iii (аросiii) у людей с дефицитом липопротеинлипазы (lpld)
RU2018121529A RU2018121529A (ru) 2013-02-14 2014-02-14 МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD)

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RU2015139055A RU2661781C2 (ru) 2013-02-14 2014-02-14 Модулирование экспрессии аполипопротеина с-iii (аросiii) у людей с дефицитом липопротеинлипазы (lpld)

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US (4) US9593333B2 (OSRAM)
EP (2) EP3400947B1 (OSRAM)
JP (3) JP6313789B2 (OSRAM)
KR (2) KR20200123263A (OSRAM)
CN (2) CN104994884B (OSRAM)
AU (3) AU2014216137B2 (OSRAM)
BR (1) BR112015019515A8 (OSRAM)
CA (2) CA2901119C (OSRAM)
DK (1) DK2956176T3 (OSRAM)
ES (2) ES2961686T3 (OSRAM)
IL (2) IL240338B (OSRAM)
MX (2) MX368155B (OSRAM)
NZ (1) NZ631119A (OSRAM)
RU (2) RU2661781C2 (OSRAM)
WO (1) WO2014127268A2 (OSRAM)
ZA (1) ZA201505628B (OSRAM)

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IL300882A (en) * 2020-10-02 2023-04-01 Ionis Pharmaceuticals Inc Methods for reducing APOCIII expression
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