TW202540427A - 脂蛋白元c3(apoc3)irna組成物及其用途 - Google Patents

脂蛋白元c3(apoc3)irna組成物及其用途

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Publication number
TW202540427A
TW202540427A TW114122039A TW114122039A TW202540427A TW 202540427 A TW202540427 A TW 202540427A TW 114122039 A TW114122039 A TW 114122039A TW 114122039 A TW114122039 A TW 114122039A TW 202540427 A TW202540427 A TW 202540427A
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Taiwan
Prior art keywords
nucleotides
nucleotide
dsrna agent
positive
strand
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TW114122039A
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English (en)
Chinese (zh)
Inventor
弗雷德里克 特倫布萊
盧卡斯D 邦都蘭特
詹姆斯D 西尼奇
艾登 卡斯特瑞諾
馬克K 史奇蓋爾
恰拉蘭波斯 凱坦尼斯
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美商阿尼拉製藥公司
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Publication of TW202540427A publication Critical patent/TW202540427A/zh

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    • CCHEMISTRY; METALLURGY
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
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  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Obesity (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Steroid Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
TW114122039A 2020-02-18 2021-02-17 脂蛋白元c3(apoc3)irna組成物及其用途 TW202540427A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202062977875P 2020-02-18 2020-02-18
US62/977,875 2020-02-18
US202163144516P 2021-02-02 2021-02-02
US63/144,516 2021-02-02

Publications (1)

Publication Number Publication Date
TW202540427A true TW202540427A (zh) 2025-10-16

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TW114122039A TW202540427A (zh) 2020-02-18 2021-02-17 脂蛋白元c3(apoc3)irna組成物及其用途
TW110105302A TWI890737B (zh) 2020-02-18 2021-02-17 脂蛋白元c3(apoc3)irna組成物及其用途

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Country Status (13)

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US (2) US11162103B2 (https=)
EP (1) EP4107265A1 (https=)
JP (2) JP7735288B2 (https=)
KR (1) KR20220143106A (https=)
CN (1) CN115397989A (https=)
AU (1) AU2021224778A1 (https=)
BR (1) BR112022016324A2 (https=)
CA (1) CA3171654A1 (https=)
IL (1) IL295496A (https=)
MX (1) MX2022010052A (https=)
TW (2) TW202540427A (https=)
WO (1) WO2021167841A1 (https=)
ZA (1) ZA202308022B (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7735288B2 (ja) 2020-02-18 2025-09-08 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 アポリポタンパク質C3(APOC3)iRNA組成物およびその使用法
US20230242918A1 (en) * 2020-05-22 2023-08-03 President And Fellows Of Harvard College Interferon- inducing oligonucleotide duplexes and methods of use
JP2024543195A (ja) 2021-12-01 2024-11-19 ディセルナ ファーマシューティカルズ インコーポレイテッド Apoc3発現を調節するための組成物及び方法
CN116410978A (zh) * 2021-12-31 2023-07-11 佑嘉(杭州)生物医药科技有限公司 一种小干扰核酸及其制剂与应用
CN119421952A (zh) * 2022-06-27 2025-02-11 大睿生物医药科技(上海)有限公司 抑制载脂蛋白C3表达的siRNA
CN116814621B (zh) * 2022-08-05 2025-09-09 厦门甘宝利生物医药有限公司 一种抑制apoc3基因表达的rna抑制剂及其应用
CN116400082A (zh) * 2023-02-02 2023-07-07 广东省妇幼保健院 检测ApoC3蛋白含量的试剂在制备预测卵母细胞发育试剂中的应用
CN121079106A (zh) * 2023-05-31 2025-12-05 上海拓界生物医药科技有限公司 一种靶向LPA的dsRNA及其应用
CN116854754B (zh) * 2023-09-01 2023-12-12 北京悦康科创医药科技股份有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物
TW202526017A (zh) * 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少apociii表現的化合物及方法
WO2025184301A1 (en) * 2024-02-29 2025-09-04 Arrowhead Pharmaceuticals, Inc. Rnai agents for dual inhibition of expression of apolipoprotein c-iii (apoc3) and proprotein convertase subtilisin kexin 9 (pcsk9), compositions thereof, and methods of use

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