CA3248387A1 - COMPOSITION AND METHOD FOR INHIBITING THE EXPRESSION OF THE LPA(APO(A)) PROTEIN - Google Patents

COMPOSITION AND METHOD FOR INHIBITING THE EXPRESSION OF THE LPA(APO(A)) PROTEIN

Info

Publication number
CA3248387A1
CA3248387A1 CA3248387A CA3248387A CA3248387A1 CA 3248387 A1 CA3248387 A1 CA 3248387A1 CA 3248387 A CA3248387 A CA 3248387A CA 3248387 A CA3248387 A CA 3248387A CA 3248387 A1 CA3248387 A1 CA 3248387A1
Authority
CA
Canada
Prior art keywords
lpa
subject
dsrna
dsrna agent
agent
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
CA3248387A
Other languages
English (en)
French (fr)
Inventor
Dongxu Shu
Pengcheng Patrick Shao
Shiwei Xia
Original Assignee
Shanghai Argo Biopharmaceutical 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 Shanghai Argo Biopharmaceutical Co Ltd filed Critical Shanghai Argo Biopharmaceutical Co Ltd
Publication of CA3248387A1 publication Critical patent/CA3248387A1/en
Pending legal-status Critical Current

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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • 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
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/312—Phosphonates
    • 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
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Dermatology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3248387A 2022-01-24 2023-01-23 COMPOSITION AND METHOD FOR INHIBITING THE EXPRESSION OF THE LPA(APO(A)) PROTEIN Pending CA3248387A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2022073415 2022-01-24
CNPCT/CN2022/073415 2022-01-24
PCT/CN2023/073456 WO2023138689A1 (zh) 2022-01-24 2023-01-23 抑制LPA(Apo(a))蛋白表达的组合物和方法

Publications (1)

Publication Number Publication Date
CA3248387A1 true CA3248387A1 (en) 2025-07-09

Family

ID=87347910

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3248387A Pending CA3248387A1 (en) 2022-01-24 2023-01-23 COMPOSITION AND METHOD FOR INHIBITING THE EXPRESSION OF THE LPA(APO(A)) PROTEIN

Country Status (16)

Country Link
US (2) US20250297250A1 (enExample)
EP (1) EP4471144A4 (enExample)
JP (2) JP7833044B2 (enExample)
KR (1) KR20240135657A (enExample)
CN (1) CN118679259A (enExample)
AU (1) AU2023210010A1 (enExample)
CA (1) CA3248387A1 (enExample)
CL (1) CL2024002168A1 (enExample)
CO (1) CO2024009492A2 (enExample)
DO (1) DOP2024000141A (enExample)
IL (1) IL314060A (enExample)
JO (1) JOP20240159A1 (enExample)
MX (1) MX2024008989A (enExample)
PE (1) PE20250779A1 (enExample)
TW (1) TW202345865A (enExample)
WO (1) WO2023138689A1 (enExample)

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KR20240103025A9 (ko) 2021-11-16 2025-12-10 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. 안지오텐시노겐(agt) 단백질의 발현을 저해하는 조성물 및 방법
AU2023404700A1 (en) * 2022-12-02 2025-07-10 Shanghai Argo Biopharmaceutical Co., Ltd. Bicyclic abasic nucleic acid analogs and oligomeric compounds prepared therefrom
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
UY40897A (es) * 2023-09-21 2025-03-31 Ionis Pharmaceuticals Inc Compuestos y métodos para inhibir 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
CN120344664A (zh) * 2023-10-27 2025-07-18 北京安龙生物医药有限公司 靶向脂蛋白a的寡核苷酸及其用途
CN120020252A (zh) * 2023-11-17 2025-05-20 纳肽得(青岛)生物医药有限公司 抑制LPA表达的siRNA及其用途
CN122514601A (zh) * 2023-11-29 2026-08-04 美国圣因生物股份有限公司 靶向Lp(a)的小干扰RNA及其用途
TW202546233A (zh) * 2024-02-05 2025-12-01 美商靖因藥業(上海)有限公司 用於抑制lp(a)或apoc3之表現的多核酸分子、醫藥組合物及其用途
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
WO2026026890A1 (zh) * 2024-07-31 2026-02-05 前沿生物药业(南京)股份有限公司 用于抑制LPA基因表达的siRNA及其修饰物与应用
WO2026092650A1 (zh) * 2024-11-01 2026-05-07 迈威(上海)生物科技股份有限公司 一种抑制LPA基因表达的RNAi制剂及其应用
WO2026131490A1 (en) * 2024-12-20 2026-06-25 Ribocure Pharmaceuticals Ab Oligonucleotide, oligonucleotide conjugate and composition, and use thereof
WO2026133253A1 (en) 2024-12-20 2026-06-25 Novartis Ag Combinations of antisense agents, pharmaceutical compositions and methods of use

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Also Published As

Publication number Publication date
KR20240135657A (ko) 2024-09-11
TW202345865A (zh) 2023-12-01
DOP2024000141A (es) 2024-10-15
PE20250779A1 (es) 2025-03-14
EP4471144A4 (en) 2026-01-21
US20250257356A1 (en) 2025-08-14
IL314060A (en) 2024-09-01
JOP20240159A1 (ar) 2024-07-21
JP7833044B2 (ja) 2026-03-18
CL2024002168A1 (es) 2025-01-10
MX2024008989A (es) 2024-07-29
CN118679259A (zh) 2024-09-20
CO2024009492A2 (es) 2024-10-31
EP4471144A1 (en) 2024-12-04
US20250297250A1 (en) 2025-09-25
WO2023138689A1 (zh) 2023-07-27
AU2023210010A1 (en) 2024-07-25
JP2026116903A (ja) 2026-07-14
JP2025504863A (ja) 2025-02-19

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