CA3147725A1 - Methods for the treatment of apoc3-related diseases and disorders - Google Patents

Methods for the treatment of apoc3-related diseases and disorders Download PDF

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Publication number
CA3147725A1
CA3147725A1 CA3147725A CA3147725A CA3147725A1 CA 3147725 A1 CA3147725 A1 CA 3147725A1 CA 3147725 A CA3147725 A CA 3147725A CA 3147725 A CA3147725 A CA 3147725A CA 3147725 A1 CA3147725 A1 CA 3147725A1
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CA
Canada
Prior art keywords
dose
apoc3
pharmaceutical composition
administered
apoc3 rnai
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.)
Pending
Application number
CA3147725A
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English (en)
French (fr)
Inventor
Bruce D. Given
James C. Hamilton
Stacey MELQUIST
Zhen Li
Rui ZHU
Tao Pei
Steven Kanner
So Wong
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 date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of CA3147725A1 publication Critical patent/CA3147725A1/en
Pending legal-status Critical Current

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • 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
    • 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
    • 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
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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
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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
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/353—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
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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)
  • 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)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3147725A 2019-08-05 2020-08-04 Methods for the treatment of apoc3-related diseases and disorders Pending CA3147725A1 (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 (1)

Publication Number Publication Date
CA3147725A1 true CA3147725A1 (en) 2021-02-11

Family

ID=74503223

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3147725A Pending CA3147725A1 (en) 2019-08-05 2020-08-04 Methods for the treatment of apoc3-related diseases and disorders

Country Status (9)

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201901841TA (en) * 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
AU2024280172A1 (en) 2023-06-01 2025-12-04 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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101383006B1 (ko) * 2009-02-10 2014-04-08 아마린 파마, 인크. 고중성지방혈증 치료 방법
MX390699B (es) * 2011-06-21 2025-03-21 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3).
EP2723758B1 (en) * 2011-06-21 2018-06-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof
JP2017535552A (ja) * 2014-11-17 2017-11-30 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
WO2018112320A1 (en) * 2016-12-16 2018-06-21 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
CN120330183A (zh) * 2017-06-02 2025-07-18 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
KR20240161202A (ko) * 2017-09-11 2024-11-12 애로우헤드 파마슈티컬스 인코포레이티드 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물
EP3719127A4 (en) * 2017-12-01 2021-10-20 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

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

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