AR134635A1 - siRNA MOLECULE TO INHIBIT PCSK9 EXPRESSION AND ITS USE - Google Patents

siRNA MOLECULE TO INHIBIT PCSK9 EXPRESSION AND ITS USE

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Publication number
AR134635A1
AR134635A1 ARP240103412A ARP240103412A AR134635A1 AR 134635 A1 AR134635 A1 AR 134635A1 AR P240103412 A ARP240103412 A AR P240103412A AR P240103412 A ARP240103412 A AR P240103412A AR 134635 A1 AR134635 A1 AR 134635A1
Authority
AR
Argentina
Prior art keywords
pcsk9
products
sirna molecule
expression
sirna
Prior art date
Application number
ARP240103412A
Other languages
Spanish (es)
Inventor
Rui Zhang
Dongdong Wang
Xinxing Liu
Rui Zhou
Qingqiao Tan
Original Assignee
Hangzhou Dinovate Biotech Co Ltd
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Hangzhou Dinovate Biotech Co Ltd filed Critical Hangzhou Dinovate Biotech Co Ltd
Publication of AR134635A1 publication Critical patent/AR134635A1/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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • 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
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/321—2'-O-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
    • C12N2310/32—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Oncology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención se refiere al campo técnico de la terapia génica, y, específicamente, a una molécula de siRNA (small interference RNA, por sus siglas en inglés, ARN pequeño de interferencia) para inhibir la expresión de PCSK9 y al uso de la misma. Particularmente, se divulgan una molécula de siRNA para inhibir la expresión de PCSK9 (que comprende una hebra sentido y una hebra antisentido que son complementarias para formar una región de doble hebra) y una composición farmacéutica que comprende la misma. La molécula de siRNA tiene una alta eficiencia de silenciamiento y un efecto de silenciamiento de larga duración para el gen PCSK9 y es útil en la fabricación de productos para inhibir la expresión de la proteína PCSK9, productos para reducir la concentración de lipoproteínas de baja densidad o colesterol de lipoproteínas de baja densidad en suero; y productos para aliviar los síntomas de enfermedades mediadas por el gen PCSK9.The present invention relates to the technical field of gene therapy, and specifically to a small interfering RNA (siRNA) molecule for inhibiting PCSK9 expression and its use. In particular, a siRNA molecule for inhibiting PCSK9 expression (comprising a sense strand and an antisense strand that are complementary to form a double-stranded region) and a pharmaceutical composition comprising the same are disclosed. The siRNA molecule has high silencing efficiency and a long-lasting silencing effect on the PCSK9 gene and is useful in the manufacture of products for inhibiting PCSK9 protein expression, products for reducing serum low-density lipoprotein cholesterol levels, and products for alleviating the symptoms of PCSK9-mediated diseases.

ARP240103412A 2023-12-11 2024-12-11 siRNA MOLECULE TO INHIBIT PCSK9 EXPRESSION AND ITS USE AR134635A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN202311686000.7A CN117384907B (en) 2023-12-11 2023-12-11 siRNA molecules that inhibit PCSK9 expression and their applications

Publications (1)

Publication Number Publication Date
AR134635A1 true AR134635A1 (en) 2026-02-04

Family

ID=89441337

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP240103412A AR134635A1 (en) 2023-12-11 2024-12-11 siRNA MOLECULE TO INHIBIT PCSK9 EXPRESSION AND ITS USE

Country Status (4)

Country Link
CN (1) CN117384907B (en)
AR (1) AR134635A1 (en)
TW (1) TW202540421A (en)
WO (1) WO2025124364A1 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117384907B (en) * 2023-12-11 2024-03-29 上海鼎新基因科技有限公司 siRNA molecules that inhibit PCSK9 expression and their applications

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101484588B (en) * 2006-05-11 2013-11-06 阿尔尼拉姆医药品有限公司 Compositions and methods for inhibiting PCSK9 gene expression
US9187746B2 (en) * 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
IL292159B1 (en) * 2012-12-05 2026-04-01 Alnylam Pharmaceuticals Inc PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CN108348541A (en) * 2015-08-25 2018-07-31 阿尔尼拉姆医药品有限公司 Methods and compositions for treating disorders associated with the proprotein convertase subtilisin KEXIN (PCSK9) gene
CN107400669A (en) * 2016-08-18 2017-11-28 广州市锐博生物科技有限公司 For suppressing the oligonucleotide molecule and its composition set of HIF1A target genes mRNA expression
JOP20190215A1 (en) * 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc Modulators of pcsk9 expression
CN109957565B (en) * 2017-12-26 2023-04-07 广州市锐博生物科技有限公司 Modified siRNA molecule and application thereof
JP7357002B2 (en) * 2018-04-18 2023-10-05 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 Oligonucleotides targeting PCSK9 for treating hypercholesterolemia and related conditions
CN115066498A (en) * 2020-03-16 2022-09-16 阿尔戈诺特Rna有限公司 Antagonists of PCSK9
WO2022079221A1 (en) * 2020-10-16 2022-04-21 Sanofi Rna compositions and methods for inhibiting lipoprotein(a)
WO2022121959A1 (en) * 2020-12-09 2022-06-16 纳肽得有限公司 Sirna molecule and application thereof in treatment of coronary artery diseases
CN114716518A (en) * 2021-01-06 2022-07-08 圣诺制药公司 Molecular structure capable of inhibiting expression of PCSK9 and pharmaceutical composition
CN113234725B (en) * 2021-05-28 2022-02-18 厦门甘宝利生物医药有限公司 An RNA inhibitor for inhibiting the expression of PCSK9 gene and its application
WO2023284763A1 (en) * 2021-07-16 2023-01-19 苏州瑞博生物技术股份有限公司 Double-stranded oligonucleotide, composition and conjugate containing double-stranded oligonucleotide, and preparation methods and uses
US20250346902A1 (en) * 2022-01-13 2025-11-13 Cspc Zhongqi Pharmaceutical Technology (Shijiazhuang) Co., Ltd. Rnai agent inhibiting pcsk9 gene expression and application thereof
CN117186172A (en) * 2023-07-19 2023-12-08 上海鼎新基因科技有限公司 Conjugate group, conjugate, preparation method and application thereof
CN117384907B (en) * 2023-12-11 2024-03-29 上海鼎新基因科技有限公司 siRNA molecules that inhibit PCSK9 expression and their applications

Also Published As

Publication number Publication date
CN117384907A (en) 2024-01-12
TW202540421A (en) 2025-10-16
WO2025124364A1 (en) 2025-06-19
CN117384907B (en) 2024-03-29

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