CN118076738A - 靶向LPA的siRNA及缀合物 - Google Patents

靶向LPA的siRNA及缀合物 Download PDF

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Publication number
CN118076738A
CN118076738A CN202280067981.4A CN202280067981A CN118076738A CN 118076738 A CN118076738 A CN 118076738A CN 202280067981 A CN202280067981 A CN 202280067981A CN 118076738 A CN118076738 A CN 118076738A
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seq
sirna
group
antisense strand
sense strand
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Pending
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CN202280067981.4A
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Chinese (zh)
Inventor
李云飞
邓永岩
侯哲
张建羽
王艳辉
茅松
黄金宇
刘楠
蔡国庆
吕珍珍
黄燕芬
周雅琴
罗敏
张芳
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Shanghai Tuojie Biomedical Technology Co ltd
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Shanghai Tuojie Biomedical Technology Co ltd
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Publication of CN118076738A publication Critical patent/CN118076738A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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
    • C12N2310/10—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
    • 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
    • C12N2310/315—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
    • C12N2310/32—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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202280067981.4A 2021-12-16 2022-12-16 靶向LPA的siRNA及缀合物 Pending CN118076738A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2021115456996 2021-12-16
CN202111545699 2021-12-16
PCT/CN2022/139500 WO2023109940A1 (zh) 2021-12-16 2022-12-16 靶向lpa的sirna及缀合物

Publications (1)

Publication Number Publication Date
CN118076738A true CN118076738A (zh) 2024-05-24

Family

ID=86774879

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280067981.4A Pending CN118076738A (zh) 2021-12-16 2022-12-16 靶向LPA的siRNA及缀合物

Country Status (8)

Country Link
US (1) US20250332190A1 (https=)
EP (1) EP4450624A4 (https=)
JP (1) JP2024545222A (https=)
KR (1) KR20240116539A (https=)
CN (1) CN118076738A (https=)
CA (1) CA3240604A1 (https=)
TW (1) TW202340465A (https=)
WO (1) WO2023109940A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119506275A (zh) * 2023-08-22 2025-02-25 苏州炫景生物科技有限公司 抑制lpa基因表达的双链寡核苷酸及其应用
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
WO2025064821A2 (en) 2023-09-21 2025-03-27 Ionis Pharmaceuticals, Inc. Compounds and methods for inhibiting lpa
WO2025082059A1 (zh) * 2023-10-16 2025-04-24 润佳(上海)医药技术有限公司 经修饰的dsRNA、双链RNAi剂及其用途
WO2025120571A1 (en) * 2023-12-05 2025-06-12 Janssen Biotech, Inc. Compositions and methods to discover targeted oligonucleotide conjugates
WO2025252159A1 (zh) * 2024-06-06 2025-12-11 大睿生物医药科技(上海)有限公司 调控LPA表达的dsRNA分子

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013299717B2 (en) 2012-08-06 2018-06-28 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
DK2991656T3 (da) * 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
JOP20160211B1 (ar) * 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
WO2018013525A1 (en) * 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
WO2019204668A1 (en) * 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
CN111378655B (zh) * 2018-12-28 2024-03-19 苏州瑞博生物技术股份有限公司 抑制CTGF基因表达的siRNA、含有该siRNA的药物组合物及其用途
CN112876534B (zh) * 2019-11-29 2024-02-09 苏州瑞博生物技术股份有限公司 肝靶向化合物及缀合物
US20230201361A1 (en) * 2020-01-11 2023-06-29 Oncolmmunin, Inc. Targeted and localized in vivo delivery of oligonucleotides
WO2021254360A1 (zh) 2020-06-16 2021-12-23 上海拓界生物医药科技有限公司 一种碳水化合物分子簇及其制备方法和医药用途
KR102812602B1 (ko) * 2020-08-05 2025-05-28 다이서나 파마수이티컬, 인크. Lpa 발현을 저해하기 위한 조성물 및 방법
WO2022121959A1 (zh) * 2020-12-09 2022-06-16 纳肽得有限公司 siRNA分子及其在治疗冠状动脉疾病中的应用
JP2024534508A (ja) * 2021-09-18 2024-09-20 成都心正合医▲葯▼科技有限公司 Ｌｐａ阻害剤及びその使用
CN114703184B (zh) * 2022-03-11 2024-06-18 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
CN115851723B (zh) * 2022-10-24 2023-10-03 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用

Also Published As

Publication number Publication date
EP4450624A4 (en) 2025-03-26
TW202340465A (zh) 2023-10-16
KR20240116539A (ko) 2024-07-29
EP4450624A1 (en) 2024-10-23
US20250332190A1 (en) 2025-10-30
JP2024545222A (ja) 2024-12-05
CA3240604A1 (en) 2023-06-22
WO2023109940A1 (zh) 2023-06-22

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