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
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—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
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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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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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
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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
    • 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
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    • 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Dermatology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (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)

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

Publications (2)

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

Family

ID=74503223

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020324974A Active AU2020324974B2 (en) 2019-08-05 2020-08-04 Methods for the treatment of APOC3-related diseases and disorders

Country Status (10)

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6989521B2 (ja) * 2016-09-02 2022-01-05 アローヘッド ファーマシューティカルズ インコーポレイテッド 標的化リガンド
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
EP4706646A1 (en) 2023-06-01 2026-03-11 Sunshine Lake Pharma Co., Ltd. Novel double-stranded sirna, conjugate thereof, and use thereof
KR20260032888A (ko) * 2024-08-30 2026-03-10 올릭스 주식회사 APOC3 유전자를 표적으로 하는 RNAi 제제 및 이의 용도

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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ES2768091T3 (es) * 2009-02-10 2020-06-19 Amarin Pharmaceuticals Ie Ltd Uso del éster etílico del ácido eicosapentaenoico para tratar la hipertrigliceridemia
EP3444348A1 (en) 2011-06-21 2019-02-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
KR20230084331A (ko) * 2011-06-21 2023-06-12 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
CA2968114A1 (en) * 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
AU2017376950B2 (en) * 2016-12-16 2024-02-22 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing TTR-associated diseases using transthyretin (TTR) iRNA compositions
EP3630788A4 (en) 2017-06-02 2021-04-28 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE
US11660347B2 (en) * 2017-12-01 2023-05-30 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing same, preparation method, and use thereof

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

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