CA3167849A1 - Antagonist of pcsk9 - Google Patents

Antagonist of pcsk9 Download PDF

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Publication number
CA3167849A1
CA3167849A1 CA3167849A CA3167849A CA3167849A1 CA 3167849 A1 CA3167849 A1 CA 3167849A1 CA 3167849 A CA3167849 A CA 3167849A CA 3167849 A CA3167849 A CA 3167849A CA 3167849 A1 CA3167849 A1 CA 3167849A1
Authority
CA
Canada
Prior art keywords
nucleic acid
acid molecule
seq
double stranded
molecule
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
CA3167849A
Other languages
English (en)
French (fr)
Inventor
Daniel Mitchell
Michael Khan
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Argonaute RNA Ltd
Original Assignee
Individual
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
Priority claimed from GBGB2003756.0A external-priority patent/GB202003756D0/en
Priority claimed from GBGB2010276.0A external-priority patent/GB202010276D0/en
Priority claimed from GBGB2013998.6A external-priority patent/GB202013998D0/en
Priority claimed from GBGB2020553.0A external-priority patent/GB202020553D0/en
Application filed by Individual filed Critical Individual
Publication of CA3167849A1 publication Critical patent/CA3167849A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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)
    • C12Y304/21111—Aqualysin 1 (3.4.21.111)
    • 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)
    • C12Y304/21112—Site-1 protease (3.4.21.112), i.e. subtilisin kexin isozyme-1
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3167849A 2020-03-16 2021-03-15 Antagonist of pcsk9 Pending CA3167849A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
GBGB2003756.0A GB202003756D0 (en) 2020-03-16 2020-03-16 Antagonist of PCSK9
GB2003756.0 2020-03-16
GBGB2010276.0A GB202010276D0 (en) 2020-07-03 2020-07-03 PCSK9 antagonist
GB2010276.0 2020-07-03
GB2013998.6 2020-09-07
GBGB2013998.6A GB202013998D0 (en) 2020-09-07 2020-09-07 Antagonist of pcsk9
GB2020553.0 2020-12-23
GBGB2020553.0A GB202020553D0 (en) 2020-12-23 2020-12-23 Antagonist of pcsk9
PCT/EP2021/056540 WO2021185765A1 (en) 2020-03-16 2021-03-15 Antagonist of pcsk9

Publications (1)

Publication Number Publication Date
CA3167849A1 true CA3167849A1 (en) 2021-09-23

Family

ID=75111564

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3167849A Pending CA3167849A1 (en) 2020-03-16 2021-03-15 Antagonist of pcsk9

Country Status (7)

Country Link
US (1) US20230183694A1 (https=)
EP (1) EP4081642A1 (https=)
JP (1) JP2023519140A (https=)
CN (1) CN115066498A (https=)
CA (1) CA3167849A1 (https=)
GB (1) GB2594788B (https=)
WO (1) WO2021185765A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
GB202020534D0 (en) * 2020-12-23 2021-02-03 Argonaute Rna Ltd Conjugate
US20250304966A1 (en) * 2020-12-23 2025-10-02 Argonaute RNA Limited Treatment of cardiovascular disease
WO2023041508A2 (en) * 2021-09-14 2023-03-23 Argonaute RNA Limited Treatment of cardiovascular disease
GB2618915B (en) * 2022-05-18 2024-08-14 Argonaute Rna Ltd Treatment of cardiovascular disease
CN117106781B (zh) * 2023-10-16 2024-03-22 深圳市茵冠生物科技有限公司 修饰的核酸及其产品和应用
CN117210468B (zh) * 2023-11-06 2024-02-20 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用
CN117384907B (zh) * 2023-12-11 2024-03-29 上海鼎新基因科技有限公司 抑制PCSK9表达的siRNA分子及其应用
WO2025252223A1 (zh) * 2024-06-07 2025-12-11 迈威(上海)生物科技股份有限公司 一种抑制PCSK9基因表达的RNAi制剂及其应用

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004261002A (ja) * 2003-01-08 2004-09-24 Tsutomu Suzuki ｓｉＲＮＡの製造方法
US8067572B2 (en) 2005-05-25 2011-11-29 The University Of York Hybrid interfering RNA
ES2874149T3 (es) * 2006-05-11 2021-11-04 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen PCSK9
CN200950065Y (zh) 2006-05-24 2007-09-19 王锦峰 一种半自动夹板装置
AU2007275365A1 (en) 2006-07-17 2008-01-24 Sirna Therapeutics Inc. RNA interference mediated inhibition of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
MX2010008394A (es) 2008-01-31 2010-11-12 Alnylam Pharmaceuticals Inc Metodos optimizados para administracion de arndc focalizando el gen pcsk9.
WO2009114475A2 (en) * 2008-03-09 2009-09-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
US9493774B2 (en) * 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
WO2011028938A1 (en) * 2009-09-02 2011-03-10 Alnylam Pharmaceuticals, Inc. Methods for lowering serum cholestrol in a subject using inhibition of pcsk9
EP3358014B1 (en) * 2010-04-19 2020-12-23 TAGCyx Biotechnologies Inc. Method for stabilizing functional nucleic acids
WO2012058693A2 (en) 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes
ES2770667T3 (es) * 2013-06-27 2020-07-02 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados que se dirigen a PCSK9
CN118697894A (zh) 2015-08-25 2024-09-27 阿尔尼拉姆医药品有限公司 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物
LT3529360T (lt) 2016-10-18 2024-07-25 Novartis Ag Metodai, skirti širdies ir kraujagyslių ligų prevencijai mažinant proproteinų konvertazės subtilizino keksino 9 (pcsk9) baltymo kiekį
CN109957565B (zh) * 2017-12-26 2023-04-07 广州市锐博生物科技有限公司 一种修饰的siRNA分子及其应用
US20230139322A1 (en) * 2017-12-26 2023-05-04 Guangzhou Ribobio Co., Ltd. SiRNA molecule inhibiting the expression of the PCSK9 gene and use thereof
CN117210468B (zh) * 2023-11-06 2024-02-20 北京悦康科创医药科技股份有限公司 靶向调控PCSK9基因表达的siRNA及其应用

Also Published As

Publication number Publication date
CN115066498A (zh) 2022-09-16
GB2594788A (en) 2021-11-10
US20230183694A1 (en) 2023-06-15
JP2023519140A (ja) 2023-05-10
GB202103594D0 (en) 2021-04-28
EP4081642A1 (en) 2022-11-02
GB2594788B (en) 2023-06-07
WO2021185765A1 (en) 2021-09-23

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