AU2020324974B2 - Methods for the treatment of APOC3-related diseases and disorders - Google Patents

Methods for the treatment of APOC3-related diseases and disorders

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Publication number
AU2020324974B2
AU2020324974B2 AU2020324974A AU2020324974A AU2020324974B2 AU 2020324974 B2 AU2020324974 B2 AU 2020324974B2 AU 2020324974 A AU2020324974 A AU 2020324974A AU 2020324974 A AU2020324974 A AU 2020324974A AU 2020324974 B2 AU2020324974 B2 AU 2020324974B2
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AU
Australia
Prior art keywords
apoc3
dose
drug substance
apoc3 rnai
administered
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2020324974A
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English (en)
Other versions
AU2020324974A1 (en
Inventor
Bruce D. Given
James C. Hamilton
Steven Kanner
Zhen Li
Stacey MELQUIST
Tao Pei
So Wong
Rui ZHU
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.)
Arrowhead Pharmaceuticals Inc
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Arrowhead Pharmaceuticals Inc
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Publication of AU2020324974A1 publication Critical patent/AU2020324974A1/en
Application granted granted Critical
Publication of AU2020324974B2 publication Critical patent/AU2020324974B2/en
Priority to AU2026208045A priority Critical patent/AU2026208045A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
    • 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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/31—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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Dermatology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2020324974A 2019-08-05 2020-08-04 Methods for the treatment of APOC3-related diseases and disorders Active AU2020324974B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2026208045A AU2026208045A1 (en) 2019-08-05 2026-07-23 Methods For The Treatment Of APOC3-Reiated Diseases And Disorders

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962883046P 2019-08-05 2019-08-05
US62/883,046 2019-08-05
US201962936559P 2019-11-17 2019-11-17
US62/936,559 2019-11-17
US202062970613P 2020-02-05 2020-02-05
US62/970,613 2020-02-05
PCT/US2020/044879 WO2021026150A1 (en) 2019-08-05 2020-08-04 Methods for the treatment of apoc3-related diseases and disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2026208045A Division AU2026208045A1 (en) 2019-08-05 2026-07-23 Methods For The Treatment Of APOC3-Reiated Diseases And Disorders

Publications (2)

Publication Number Publication Date
AU2020324974A1 AU2020324974A1 (en) 2022-02-24
AU2020324974B2 true AU2020324974B2 (en) 2026-04-30

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AU2020324974A Active AU2020324974B2 (en) 2019-08-05 2020-08-04 Methods for the treatment of APOC3-related diseases and disorders
AU2026208045A Pending AU2026208045A1 (en) 2019-08-05 2026-07-23 Methods For The Treatment Of APOC3-Reiated Diseases And Disorders

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AU2026208045A Pending AU2026208045A1 (en) 2019-08-05 2026-07-23 Methods For The Treatment Of APOC3-Reiated Diseases And Disorders

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US (1) US20220152079A1 (enExample)
EP (1) EP4010075A4 (enExample)
JP (2) JP7829478B2 (enExample)
KR (1) KR20220044551A (enExample)
CN (1) CN114555188A (enExample)
AU (2) AU2020324974B2 (enExample)
CA (1) CA3147725A1 (enExample)
MX (1) MX2022001460A (enExample)
TW (1) TW202120104A (enExample)
WO (1) WO2021026150A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20170056B1 (ar) * 2016-09-02 2021-08-17 Arrowhead Pharmaceuticals Inc مركبات ترابطية مستهدفة
CN114934074A (zh) * 2022-06-01 2022-08-23 北京大学 一种ApoC3基因敲除仓鼠模型的构建方法
WO2024245381A1 (zh) 2023-06-01 2024-12-05 广东东阳光药业股份有限公司 一种新型的双链siRNA、其缀合物及其用途
WO2026049536A1 (en) * 2024-08-30 2026-03-05 Olix Pharmaceuticals, Inc. RNAi AGENT TARGETING APOC3 GENE AND USE THEREOF

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019051402A1 (en) * 2017-09-11 2019-03-14 Arrowhead Pharmaceuticals, Inc. RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)

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RU2505292C2 (ru) * 2009-02-10 2014-01-27 Амарин Фармасьютикалз Айрлэнд Лимитед Способы лечения гипертриглицеридемии
CA2839573A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
EP2723756B1 (en) * 2011-06-21 2020-03-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
TWI865997B (zh) * 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
CN111164091B (zh) 2017-06-02 2025-01-07 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
CN118291456A (zh) * 2017-12-01 2024-07-05 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019051402A1 (en) * 2017-09-11 2019-03-14 Arrowhead Pharmaceuticals, Inc. RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
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ANROOPB NAIR ET AL.: "A simple practice guide for dose conversion between animals and human", JOURNAL OF BASIC AND CLINICAL PHARMACY, vol. 7, no. 2, 1 May 2016 (2016-05-01), India, pages 27, DOI: 10.4103/0976-0105.177703 *
Yu, R. Z., et al., Molecular therapy. Nucleic acids, (2015), vol. 4, no. 1, e218. https://doi.org/10.1038/mtna.2014.69 *

Also Published As

Publication number Publication date
TW202120104A (zh) 2021-06-01
CN114555188A (zh) 2022-05-27
JP7829478B2 (ja) 2026-03-13
AU2026208045A1 (en) 2026-08-13
JP2022543136A (ja) 2022-10-07
US20220152079A1 (en) 2022-05-19
KR20220044551A (ko) 2022-04-08
MX2022001460A (es) 2022-02-22
EP4010075A4 (en) 2023-11-15
WO2021026150A1 (en) 2021-02-11
JP2025061882A (ja) 2025-04-11
CA3147725A1 (en) 2021-02-11
EP4010075A1 (en) 2022-06-15
AU2020324974A1 (en) 2022-02-24

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