AU2019377275A1 - Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a) - Google Patents

Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a) Download PDF

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Publication number
AU2019377275A1
AU2019377275A1 AU2019377275A AU2019377275A AU2019377275A1 AU 2019377275 A1 AU2019377275 A1 AU 2019377275A1 AU 2019377275 A AU2019377275 A AU 2019377275A AU 2019377275 A AU2019377275 A AU 2019377275A AU 2019377275 A1 AU2019377275 A1 AU 2019377275A1
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Australia
Prior art keywords
patient
compound
administration
plasma
death
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Abandoned
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AU2019377275A
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English (en)
Inventor
Anastasia LESOGOR
Qing SHAO
Tom Thuren
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Novartis AG
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Novartis AG
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Filing date
Publication date
Application filed by Novartis AG filed Critical Novartis AG
Publication of AU2019377275A1 publication Critical patent/AU2019377275A1/en
Priority to AU2023210550A priority Critical patent/AU2023210550A1/en
Priority to AU2025267467A priority patent/AU2025267467A1/en
Abandoned legal-status Critical Current

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    • 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
    • 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
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2019377275A 2018-11-09 2019-11-08 Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a) Abandoned AU2019377275A1 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2023210550A AU2023210550A1 (en) 2018-11-09 2023-07-31 METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(a)
AU2025267467A AU2025267467A1 (en) 2018-11-09 2025-11-14 METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(a)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862758323P 2018-11-09 2018-11-09
US62/758,323 2018-11-09
US201962874459P 2019-07-15 2019-07-15
US62/874,459 2019-07-15
PCT/IB2019/059632 WO2020095274A1 (en) 2018-11-09 2019-11-08 Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a)

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2023210550A Division AU2023210550A1 (en) 2018-11-09 2023-07-31 METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(a)

Publications (1)

Publication Number Publication Date
AU2019377275A1 true AU2019377275A1 (en) 2021-03-25

Family

ID=68582078

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2019377275A Abandoned AU2019377275A1 (en) 2018-11-09 2019-11-08 Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a)
AU2023210550A Abandoned AU2023210550A1 (en) 2018-11-09 2023-07-31 METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(a)
AU2025267467A Pending AU2025267467A1 (en) 2018-11-09 2025-11-14 METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(a)

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2023210550A Abandoned AU2023210550A1 (en) 2018-11-09 2023-07-31 METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(a)
AU2025267467A Pending AU2025267467A1 (en) 2018-11-09 2025-11-14 METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(a)

Country Status (9)

Country Link
US (2) US20210395737A1 (enExample)
EP (1) EP3877523A1 (enExample)
JP (3) JP2022512911A (enExample)
KR (2) KR20210091703A (enExample)
CN (1) CN113286885A (enExample)
AU (3) AU2019377275A1 (enExample)
CA (1) CA3111382A1 (enExample)
IL (1) IL281147A (enExample)
WO (1) WO2020095274A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020150473A2 (en) 2019-01-18 2020-07-23 Dogma Therapeutics, Inc. Pcsk9 inhibitors and methods of use thereof
BR112021013924A2 (pt) 2019-01-18 2021-09-21 Astrazeneca Ab Inibidores de pcsk9 e métodos de uso do mesmo

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
US7259150B2 (en) 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
WO2003014397A1 (en) 2001-08-09 2003-02-20 Biomedlab Corporation Probe for detection of enteric virus detection kit and method for enteric virus with the same
RU2624028C2 (ru) 2012-05-24 2017-06-30 Ионис Фармасьютикалз, Инк. Способы и композиции для модулирования экспрессии аполипопротеина (а)
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
WO2015168589A2 (en) * 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating angiopoietin-like 3 expression
AU2016349625B2 (en) 2015-11-06 2022-07-07 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
LT4119569T (lt) * 2015-11-06 2024-09-25 Ionis Pharmaceuticals, Inc. Konjuguoti priešprasmiai junginiai, skirti panaudoti terapijoje

Also Published As

Publication number Publication date
WO2020095274A1 (en) 2020-05-14
AU2025267467A1 (en) 2025-12-04
AU2023210550A1 (en) 2023-08-24
JP2022512911A (ja) 2022-02-07
IL281147A (en) 2021-04-29
US20260049310A1 (en) 2026-02-19
CA3111382A1 (en) 2020-05-14
JP2026021335A (ja) 2026-02-10
KR20260037169A (ko) 2026-03-17
CN113286885A (zh) 2021-08-20
US20210395737A1 (en) 2021-12-23
EP3877523A1 (en) 2021-09-15
JP2024081645A (ja) 2024-06-18
KR20210091703A (ko) 2021-07-22

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