IL240338B - Modulation of apolipoprotein apociii) c–iii) expression in populations with lipoprotein lipase deficiency (lpld) - Google Patents

Modulation of apolipoprotein apociii) c–iii) expression in populations with lipoprotein lipase deficiency (lpld)

Info

Publication number
IL240338B
IL240338B IL240338A IL24033815A IL240338B IL 240338 B IL240338 B IL 240338B IL 240338 A IL240338 A IL 240338A IL 24033815 A IL24033815 A IL 24033815A IL 240338 B IL240338 B IL 240338B
Authority
IL
Israel
Prior art keywords
lpld
apociii
apolipoprotein
populations
modulation
Prior art date
Application number
IL240338A
Other languages
English (en)
Hebrew (he)
Other versions
IL240338A0 (en
Original Assignee
Ionis Pharmaceuticals Inc
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=51354689&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=IL240338(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of IL240338A0 publication Critical patent/IL240338A0/en
Publication of IL240338B publication Critical patent/IL240338B/en

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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
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • 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
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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
    • 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
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    • 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
    • C—CHEMISTRY; METALLURGY
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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 ===---===
    • C—CHEMISTRY; METALLURGY
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/35—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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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
IL240338A 2013-02-14 2015-08-03 Modulation of apolipoprotein apociii) c–iii) expression in populations with lipoprotein lipase deficiency (lpld) IL240338B (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361764969P 2013-02-14 2013-02-14
US201361880779P 2013-09-20 2013-09-20
PCT/US2014/016546 WO2014127268A2 (en) 2013-02-14 2014-02-14 Modulation of apolipoprotein c-iii (apociii) expression in lipoprotein lipase deficient (lpld) populations

Publications (2)

Publication Number Publication Date
IL240338A0 IL240338A0 (en) 2015-09-24
IL240338B true IL240338B (en) 2020-06-30

Family

ID=51354689

Family Applications (2)

Application Number Title Priority Date Filing Date
IL240338A IL240338B (en) 2013-02-14 2015-08-03 Modulation of apolipoprotein apociii) c–iii) expression in populations with lipoprotein lipase deficiency (lpld)
IL275246A IL275246A (en) 2013-02-14 2020-06-09 Modulation of apolipoprotein C–III (APOCIII) expression in populations with lipoprotein lipase deficiency (LPLD)

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL275246A IL275246A (en) 2013-02-14 2020-06-09 Modulation of apolipoprotein C–III (APOCIII) expression in populations with lipoprotein lipase deficiency (LPLD)

Country Status (16)

Country Link
US (4) US9593333B2 (OSRAM)
EP (2) EP2956176B1 (OSRAM)
JP (3) JP6313789B2 (OSRAM)
KR (2) KR20200123263A (OSRAM)
CN (2) CN108743943A (OSRAM)
AU (3) AU2014216137B2 (OSRAM)
BR (1) BR112015019515A8 (OSRAM)
CA (2) CA3170716A1 (OSRAM)
DK (1) DK2956176T3 (OSRAM)
ES (2) ES2680599T3 (OSRAM)
IL (2) IL240338B (OSRAM)
MX (2) MX368155B (OSRAM)
NZ (1) NZ631119A (OSRAM)
RU (2) RU2018121529A (OSRAM)
WO (1) WO2014127268A2 (OSRAM)
ZA (1) ZA201505628B (OSRAM)

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Publication number Priority date Publication date Assignee Title
CN107854478A (zh) * 2011-04-27 2018-03-30 Ionis制药公司 载脂蛋白ciii（apociii）表达的调节
RU2018121529A (ru) * 2013-02-14 2019-03-06 Ионис Фармасьютикалз, Инк. МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD)
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EP3270931A4 (en) * 2015-02-27 2018-10-03 Ionis Pharmaceuticals, Inc. Modulation of apolipoprotein c-iii (apociii) expression in lipodystrophy populations
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CN120884604A (zh) 2017-06-02 2025-11-04 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
KR20200074975A (ko) 2017-10-31 2020-06-25 스태튼 바이오테크놀로지 비.브이. 항-ApoC3 항체 및 이의 사용 방법
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TWI844541B (zh) 2018-05-11 2024-06-11 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其使用方法
WO2022072244A1 (en) * 2020-10-02 2022-04-07 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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