ES2961686T3 - Modulación de la expresión de apolipoproteína C-III (APOCIII) en poblaciones deficientes en lipoproteína lipasa (LPLD) - Google Patents

Modulación de la expresión de apolipoproteína C-III (APOCIII) en poblaciones deficientes en lipoproteína lipasa (LPLD) Download PDF

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Publication number
ES2961686T3
ES2961686T3 ES18179911T ES18179911T ES2961686T3 ES 2961686 T3 ES2961686 T3 ES 2961686T3 ES 18179911 T ES18179911 T ES 18179911T ES 18179911 T ES18179911 T ES 18179911T ES 2961686 T3 ES2961686 T3 ES 2961686T3
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Prior art keywords
apociii
specific
certain embodiments
modified
inhibitor
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Spanish (es)
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Veronica J Alexander
Nicholas J Viney
Joseph L Witztum
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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
    • 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
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • 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
    • 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
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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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]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
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  • Biotechnology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
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  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
ES18179911T 2013-02-14 2014-02-14 Modulación de la expresión de apolipoproteína C-III (APOCIII) en poblaciones deficientes en lipoproteína lipasa (LPLD) Active ES2961686T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361764969P 2013-02-14 2013-02-14
US201361880779P 2013-09-20 2013-09-20

Publications (1)

Publication Number Publication Date
ES2961686T3 true ES2961686T3 (es) 2024-03-13

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ID=51354689

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ES18179911T Active ES2961686T3 (es) 2013-02-14 2014-02-14 Modulación de la expresión de apolipoproteína C-III (APOCIII) en poblaciones deficientes en lipoproteína lipasa (LPLD)
ES14751357.6T Active ES2680599T3 (es) 2013-02-14 2014-02-14 Modulación de la expresión de apolipoproteína C-III (ApoCIII) en poblaciones deficientes en lipoproteína lipasa (LPLD)

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ES14751357.6T Active ES2680599T3 (es) 2013-02-14 2014-02-14 Modulación de la expresión de apolipoproteína C-III (ApoCIII) en poblaciones deficientes en lipoproteína lipasa (LPLD)

Country Status (16)

Country Link
US (4) US9593333B2 (cg-RX-API-DMAC7.html)
EP (2) EP3400947B1 (cg-RX-API-DMAC7.html)
JP (3) JP6313789B2 (cg-RX-API-DMAC7.html)
KR (2) KR20200123263A (cg-RX-API-DMAC7.html)
CN (2) CN104994884B (cg-RX-API-DMAC7.html)
AU (3) AU2014216137B2 (cg-RX-API-DMAC7.html)
BR (1) BR112015019515A8 (cg-RX-API-DMAC7.html)
CA (2) CA2901119C (cg-RX-API-DMAC7.html)
DK (1) DK2956176T3 (cg-RX-API-DMAC7.html)
ES (2) ES2961686T3 (cg-RX-API-DMAC7.html)
IL (2) IL240338B (cg-RX-API-DMAC7.html)
MX (2) MX368155B (cg-RX-API-DMAC7.html)
NZ (1) NZ631119A (cg-RX-API-DMAC7.html)
RU (2) RU2018121529A (cg-RX-API-DMAC7.html)
WO (1) WO2014127268A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201505628B (cg-RX-API-DMAC7.html)

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EP3011028B1 (en) * 2013-06-21 2019-06-12 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
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WO2018193427A1 (en) 2017-04-21 2018-10-25 Staten Biotechnology B.V. Anti-apoc3 antibodies and methods of use thereof
JP2020522510A (ja) 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
US10538583B2 (en) 2017-10-31 2020-01-21 Staten Biotechnology B.V. Anti-APOC3 antibodies and compositions thereof
CN111315772A (zh) 2017-10-31 2020-06-19 斯塔顿生物技术有限公司 抗apoc3抗体及其使用方法
US12391942B2 (en) 2018-05-11 2025-08-19 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US20240033280A1 (en) * 2020-10-02 2024-02-01 Ionis Pharmaceuticals, Inc. Methods for reducing apociii expression
CN116814621B (zh) * 2022-08-05 2025-09-09 厦门甘宝利生物医药有限公司 一种抑制apoc3基因表达的rna抑制剂及其应用
WO2025110984A1 (en) * 2023-11-21 2025-05-30 Hydrogene Therapeutics, Inc. Compositions and methods for treating wilson's disease

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