CN116348125A - 降低apociii表达的方法 - Google Patents

降低apociii表达的方法 Download PDF

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Publication number
CN116348125A
CN116348125A CN202180066569.6A CN202180066569A CN116348125A CN 116348125 A CN116348125 A CN 116348125A CN 202180066569 A CN202180066569 A CN 202180066569A CN 116348125 A CN116348125 A CN 116348125A
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Prior art keywords
weeks
less
therapeutically effective
effective amount
administering
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Pending
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CN202180066569.6A
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English (en)
Chinese (zh)
Inventor
S·齐米卡斯
V·J·亚历山大
S·T·克罗克
R·S·吉里
B·P·莫妮亚
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/05—Dipeptides
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
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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
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
    • C12N2310/35—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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Biochemistry (AREA)
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  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
CN202180066569.6A 2020-10-02 2021-09-24 降低apociii表达的方法 Pending CN116348125A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063087095P 2020-10-02 2020-10-02
US63/087,095 2020-10-02
PCT/US2021/052001 WO2022072244A1 (en) 2020-10-02 2021-09-24 Methods for reducing apociii expression

Publications (1)

Publication Number Publication Date
CN116348125A true CN116348125A (zh) 2023-06-27

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Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180066569.6A Pending CN116348125A (zh) 2020-10-02 2021-09-24 降低apociii表达的方法

Country Status (10)

Country Link
US (1) US20240033280A1 (https=)
EP (1) EP4221720A4 (https=)
JP (1) JP2023543898A (https=)
KR (1) KR20230079394A (https=)
CN (1) CN116348125A (https=)
AU (1) AU2021353849A1 (https=)
BR (1) BR112023004012A2 (https=)
IL (1) IL300882A (https=)
MX (1) MX2023003855A (https=)
WO (1) WO2022072244A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (en) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
AU2023372971A1 (en) * 2022-11-02 2025-05-15 Ionis Pharmaceuticals, Inc. Methods for modulating complement factor b expression
WO2024194686A2 (en) 2023-03-17 2024-09-26 Oxitope Pharma B.V. Anti-phosphocholine antibodies and methods of use thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190046555A1 (en) * 2015-11-06 2019-02-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7598227B2 (en) * 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
AU2014216137B2 (en) * 2013-02-14 2018-05-10 Ionis Pharmaceuticals, Inc. Modulation of Apolipoprotein C-III (ApoCIII) expression in lipoprotein lipase deficient (LPLD) populations
JP2018511555A (ja) * 2015-02-27 2018-04-26 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． リポジストロフィー集団におけるアポリポタンパク質Ｃ−ＩＩＩ（ＡｐｏＣＩＩＩ）発現の調節

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190046555A1 (en) * 2015-11-06 2019-02-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
OLUWAYEMISI ESAN等: "Volanesorsen in the Treatment of Familial Chylomicronemia Syndrome or Hypertriglyceridaemia: Design, Development and Place in Therapy", 《DRUG DESIGN, DEVELOPMENT AND THERAPY》, vol. 14, 6 July 2020 (2020-07-06), pages 2623 - 2636, XP093190614, DOI: 10.2147/DDDT.S224771 *
VERONICA J. ALEXANDER等: "N-acetyl galactosamine-conjugated antisense drug to APOC3mRNA, triglycerides and atherogenic lipoprotein levels", 《EUROPEAN HEART JOURNAL》, vol. 40, 24 April 2019 (2019-04-24), pages 2785 *

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Publication number Publication date
KR20230079394A (ko) 2023-06-07
WO2022072244A1 (en) 2022-04-07
AU2021353849A1 (en) 2023-03-23
EP4221720A4 (en) 2024-11-13
US20240033280A1 (en) 2024-02-01
EP4221720A1 (en) 2023-08-09
MX2023003855A (es) 2023-04-14
IL300882A (en) 2023-04-01
AU2021353849A8 (en) 2023-03-30
JP2023543898A (ja) 2023-10-18
BR112023004012A2 (pt) 2023-04-25

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