CA3229020A1 - Treatment of cardiovascular disease - Google Patents

Treatment of cardiovascular disease Download PDF

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Publication number
CA3229020A1
CA3229020A1 CA3229020A CA3229020A CA3229020A1 CA 3229020 A1 CA3229020 A1 CA 3229020A1 CA 3229020 A CA3229020 A CA 3229020A CA 3229020 A CA3229020 A CA 3229020A CA 3229020 A1 CA3229020 A1 CA 3229020A1
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Canada
Prior art keywords
nucleic acid
acid molecule
seq
nucleotide sequence
sense
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
CA3229020A
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English (en)
French (fr)
Inventor
Stella Khan
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
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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 GBGB2113104.0A external-priority patent/GB202113104D0/en
Priority claimed from GBGB2207239.1A external-priority patent/GB202207239D0/en
Application filed by Individual filed Critical Individual
Publication of CA3229020A1 publication Critical patent/CA3229020A1/en
Pending legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
    • 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
    • C12N2310/3519Fusion with another nucleic acid
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CA3229020A 2021-09-14 2022-09-13 Treatment of cardiovascular disease Pending CA3229020A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GBGB2113104.0A GB202113104D0 (en) 2021-09-14 2021-09-14 Antagonist of pcsk9
GB2113104.0 2021-09-14
GBGB2207239.1A GB202207239D0 (en) 2022-05-18 2022-05-18 Treatment of cardiovascular disease 2
GB2207239.1 2022-05-18
PCT/EP2022/075355 WO2023041508A2 (en) 2021-09-14 2022-09-13 Treatment of cardiovascular disease

Publications (1)

Publication Number Publication Date
CA3229020A1 true CA3229020A1 (en) 2023-03-23

Family

ID=83691543

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3229020A Pending CA3229020A1 (en) 2021-09-14 2022-09-13 Treatment of cardiovascular disease

Country Status (5)

Country Link
US (1) US20250283076A1 (https=)
EP (1) EP4402263A2 (https=)
JP (1) JP2024531728A (https=)
CA (1) CA3229020A1 (https=)
WO (1) WO2023041508A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL312811A (en) 2021-11-16 2024-07-01 Shanghai Argo Biopharmaceutical Co Ltd Composition and method for inhibiting angiotensinogen (agt) protein expression
TW202345865A (zh) * 2022-01-24 2023-12-01 大陸商上海舶望製藥有限公司 抑制LPA(Apo(a))蛋白表達的組合物和方法
EP4680740A1 (en) * 2023-03-14 2026-01-21 Argonaute Rna Limited Conjugate comprising a double stranded rna molecule linked to a single stranded dna molecule
WO2024228030A2 (en) * 2023-05-04 2024-11-07 Argonaute RNA Limited Dual silencing
WO2024245151A1 (zh) * 2023-05-26 2024-12-05 维亚臻生物技术(苏州)有限公司 一种用于代谢疾病的双链核苷酸化合物及其应用
AU2024321098A1 (en) * 2023-08-09 2026-03-19 Beijing Foyou Pharma Co., Ltd Sirna for inhibiting dgat2 gene expression, sirna conjugate or prodrug and pharmaceutical composition thereof, and use thereof
WO2025040139A1 (zh) * 2023-08-23 2025-02-27 云合智药(苏州)生物科技有限公司 用于抑制二酰甘油-O-酰基转移酶2表达的RNAi剂及其应用
WO2025103391A1 (zh) * 2023-11-15 2025-05-22 北京加科瑞康医药科技有限公司 用于抑制lpa的基因表达的sirna,含其的组合物及其用途

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001064008A2 (en) 2000-03-03 2001-09-07 Smithkline Beecham Biologicals S.A. Vaccine for the treatment of artherosclerosis
GB0121171D0 (en) 2001-08-31 2001-10-24 Glaxosmithkline Biolog Sa Vaccine
GB0305794D0 (en) 2003-03-13 2003-04-16 Glaxosmithkline Biolog Sa Vaccine
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
WO2005045035A2 (en) 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF NOGO AND NOGO RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
US8067572B2 (en) * 2005-05-25 2011-11-29 The University Of York Hybrid interfering RNA
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
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)
ES2804764T3 (es) * 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
WO2012058693A2 (en) * 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes
US10125369B2 (en) * 2012-12-05 2018-11-13 Alnylam Pharmaceuticals, Inc. PCSK9 iRNA compositions and methods of use thereof
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
WO2014205449A2 (en) 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
JOP20160211B1 (ar) 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
BR112019010057A2 (pt) 2016-11-16 2019-09-03 Purdue Research Foundation composição, método para suprimir a ingestão de comida, sequência de shrna, método de inibição da síntese de ácidos graxos
MX2020004897A (es) 2017-11-13 2020-10-05 Silence Therapeutics Gmbh Acidos nucleicos para inhibir la expresion de lpa en una celula.
BR112021009213A2 (pt) * 2018-11-13 2021-08-10 Silence Therapeutics Gmbh ácidos nucleicos para inibição da expressão de lpa em uma célula
US20230027604A1 (en) * 2019-07-02 2023-01-26 Argonaute RNA Limited Apolipoprotein b antagonist
US20230078200A1 (en) * 2019-12-09 2023-03-16 Amgen Inc. RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
US20230183694A1 (en) * 2020-03-16 2023-06-15 Argonaute RNA Limited Antagonist of pcsk9

Also Published As

Publication number Publication date
JP2024531728A (ja) 2024-08-29
WO2023041508A3 (en) 2023-06-08
EP4402263A2 (en) 2024-07-24
US20250283076A1 (en) 2025-09-11
WO2023041508A2 (en) 2023-03-23

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