JP7829478B2 - Apoc3関連疾患および障害の処置のための方法 - Google Patents

Apoc3関連疾患および障害の処置のための方法

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Publication number
JP7829478B2
JP7829478B2 JP2022506919A JP2022506919A JP7829478B2 JP 7829478 B2 JP7829478 B2 JP 7829478B2 JP 2022506919 A JP2022506919 A JP 2022506919A JP 2022506919 A JP2022506919 A JP 2022506919A JP 7829478 B2 JP7829478 B2 JP 7829478B2
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apoc3
dose
approximately
pharmaceutical composition
apoc3 rnai
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JP2022543136A5 (OSRAM
JP2022543136A (ja
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ブルース ディー. ギブン,
ジェイムズ シー. ハミルトン,
ステイシー メルクイスト,
ゼン リ,
ルイ ジュー,
タオ ペイ,
スティーブン カナー,
ソ ウォン,
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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
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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
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P3/06—Antihyperlipidemics
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    • 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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    • C12N2310/30—Chemical structure
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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
    • 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
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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
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    • 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)
JP2022506919A 2019-08-05 2020-08-04 Apoc3関連疾患および障害の処置のための方法 Active JP7829478B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025012194A JP2025061882A (ja) 2019-08-05 2025-01-28 Apoc3関連疾患および障害の処置のための方法

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)

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JP2025012194A Division JP2025061882A (ja) 2019-08-05 2025-01-28 Apoc3関連疾患および障害の処置のための方法

Publications (3)

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JP2022543136A JP2022543136A (ja) 2022-10-07
JP2022543136A5 JP2022543136A5 (OSRAM) 2023-08-14
JP7829478B2 true JP7829478B2 (ja) 2026-03-13

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JP2022506919A Active JP7829478B2 (ja) 2019-08-05 2020-08-04 Apoc3関連疾患および障害の処置のための方法
JP2025012194A Pending JP2025061882A (ja) 2019-08-05 2025-01-28 Apoc3関連疾患および障害の処置のための方法

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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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014524738A (ja) 2011-06-21 2014-09-25 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 アンジオポエチン様3(ANGPTL3)iRNA組成物及びその使用方法
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
WO2018223073A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019105419A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 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

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2768091T3 (es) * 2009-02-10 2020-06-19 Amarin Pharmaceuticals Ie Ltd Uso del éster etílico del ácido eicosapentaenoico para tratar la hipertrigliceridemia
KR20230084331A (ko) * 2011-06-21 2023-06-12 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
AU2017376950B2 (en) * 2016-12-16 2024-02-22 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing TTR-associated diseases using transthyretin (TTR) iRNA compositions
TN2020000039A1 (en) * 2017-09-11 2021-10-04 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014524738A (ja) 2011-06-21 2014-09-25 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 アンジオポエチン様3(ANGPTL3)iRNA組成物及びその使用方法
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
WO2018223073A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2019105419A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 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

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WO2021026150A1 (en) 2021-02-11
AU2020324974B2 (en) 2026-04-30
TW202120104A (zh) 2021-06-01
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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