CA2658183A1 - Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina) - Google Patents

Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina) Download PDF

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Publication number
CA2658183A1
CA2658183A1 CA002658183A CA2658183A CA2658183A1 CA 2658183 A1 CA2658183 A1 CA 2658183A1 CA 002658183 A CA002658183 A CA 002658183A CA 2658183 A CA2658183 A CA 2658183A CA 2658183 A1 CA2658183 A1 CA 2658183A1
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Prior art keywords
nucleotides
nucleic acid
sina
strand
double stranded
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Abandoned
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CA002658183A
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English (en)
French (fr)
Inventor
James Mcswiggen
Vasant Jadhav
Chandra Vargeese
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Sirna Therapeutics Inc
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Individual
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Priority claimed from US11/487,788 external-priority patent/US20070173473A1/en
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Publication of CA2658183A1 publication Critical patent/CA2658183A1/en
Abandoned legal-status Critical Current

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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
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/04Anorexiants; Antiobesity agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/06Antiarrhythmics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/12Antihypertensives
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Heart & Thoracic Surgery (AREA)
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  • Obesity (AREA)
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  • Plant Pathology (AREA)
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  • Child & Adolescent Psychology (AREA)
  • Vascular Medicine (AREA)
  • Emergency Medicine (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA002658183A 2006-07-17 2007-07-17 Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina) Abandoned CA2658183A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US11/487,788 2006-07-17
US11/487,788 US20070173473A1 (en) 2001-05-18 2006-07-17 RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
US86136906P 2006-11-28 2006-11-28
US60/861,369 2006-11-28
PCT/US2007/073723 WO2008011431A2 (en) 2006-07-17 2007-07-17 Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)

Publications (1)

Publication Number Publication Date
CA2658183A1 true CA2658183A1 (en) 2008-01-24

Family

ID=38957565

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002658183A Abandoned CA2658183A1 (en) 2006-07-17 2007-07-17 Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)

Country Status (6)

Country Link
EP (1) EP2052079A2 (ja)
JP (1) JP2010503382A (ja)
CN (1) CN102124107A (ja)
AU (1) AU2007275365A1 (ja)
CA (1) CA2658183A1 (ja)
WO (1) WO2008011431A2 (ja)

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CN115368916A (zh) * 2016-04-05 2022-11-22 斯特拉斯堡大学 用于捕获分子靶标的液滴内表面工程

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US7404969B2 (en) * 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
EP1922300A2 (en) 2005-02-14 2008-05-21 Sirna Therapeutics Inc. Cationic lipids and formulated molecular compositions containing them
EP2835429B1 (en) 2006-05-11 2017-12-13 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
EP2076597A2 (en) * 2006-10-09 2009-07-08 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9
US8084437B2 (en) 2006-11-27 2011-12-27 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
CA2713379A1 (en) * 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of dsrna targeting the pcsk9 gene
WO2009114475A2 (en) * 2008-03-09 2009-09-17 Intradigm Corporation Compositions comprising human pcsk9 and apolipoprotein b sirna and methods of use
US20110224280A1 (en) * 2008-04-16 2011-09-15 Niels Fisker Nielsen Pharmaceutical Composition Comprising Anti PCSK9 Oligomers
CA2635187A1 (en) 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
US8790664B2 (en) 2008-09-05 2014-07-29 Institut National De La Sante Et De La Recherche Medicale (Inserm) Multimodular assembly useful for intracellular delivery
MX2011013421A (es) * 2009-06-15 2012-03-16 Alnylam Pharmaceuticals Inc Arnds formulado con lipido de direccionamiento del gen pcsk9.
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
WO2011009697A1 (en) 2009-07-21 2011-01-27 Santaris Pharma A/S Antisense oligomers targeting pcsk9
WO2011038031A1 (en) 2009-09-22 2011-03-31 Alnylam Pharmaceuticals, Inc. Dual targeting sirna agents
EP2516010A2 (en) * 2009-12-23 2012-10-31 Novartis AG Lipids, lipid compositions, and methods of using them
CN103703141B (zh) * 2011-04-08 2016-08-17 伯乐实验室公司 具有降低的非特异性活性的pcr反应混合物
CA2856289A1 (en) * 2011-11-18 2013-05-23 Alnylam Pharmaceuticals, Inc. Modified rnai agents
JP6002382B2 (ja) * 2011-12-07 2016-10-05 株式会社バイオシンクタンク 遺伝子発現阻害剤及び阻害方法
EP2667197B1 (en) * 2012-05-25 2015-09-16 Zora Biosciences OY Sensitive efficacy and specificity biomarkers for proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibition
EP3336187A1 (en) * 2012-12-05 2018-06-20 Alnylam Pharmaceuticals, Inc. Pcsk9 irna compositions and methods of use thereof
CN105358692B (zh) 2013-06-27 2020-08-21 罗氏创新中心哥本哈根有限公司 靶向pcsk9的反义寡聚体和缀合物
US9034332B1 (en) 2014-07-15 2015-05-19 Kymab Limited Precision medicine by targeting rare human PCSK9 variants for cholesterol treatment
US9045548B1 (en) 2014-07-15 2015-06-02 Kymab Limited Precision Medicine by targeting rare human PCSK9 variants for cholesterol treatment
US9023359B1 (en) 2014-07-15 2015-05-05 Kymab Limited Targeting rare human PCSK9 variants for cholesterol treatment
US8883157B1 (en) 2013-12-17 2014-11-11 Kymab Limited Targeting rare human PCSK9 variants for cholesterol treatment
US9051378B1 (en) 2014-07-15 2015-06-09 Kymab Limited Targeting rare human PCSK9 variants for cholesterol treatment
US9914769B2 (en) 2014-07-15 2018-03-13 Kymab Limited Precision medicine for cholesterol treatment
FR3014695A1 (ja) * 2013-12-17 2015-06-19 Kymab Ltd
US9139648B1 (en) 2014-07-15 2015-09-22 Kymab Limited Precision medicine by targeting human NAV1.9 variants for treatment of pain
GB2537614A (en) * 2015-04-20 2016-10-26 Heart Biotech Ltd Formulations for inhibition of PCSK9 for the treatment of hypercholesterolemia
IL310959A (en) 2015-08-25 2024-04-01 Alnylam Pharmaceuticals Inc Methods and preparations for treating a disorder related to the PCSK9 gene
WO2017131124A1 (ja) * 2016-01-26 2017-08-03 日産化学工業株式会社 一本鎖オリゴヌクレオチド
EP3521430A4 (en) 2016-09-29 2020-05-20 National University Corporation Tokyo Medical and Dental University DOUBLE-STRANDED NUCLEIC ACID COMPLEX WITH OVERHEAD
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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
CN109957567B (zh) * 2017-12-26 2022-09-23 阿格纳生物制药有限公司 一种抑制PCSK9基因表达的siRNA分子及其应用
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CN109666727B (zh) * 2018-12-29 2020-11-06 中国药科大学 一种高活性抑制pcsk9表达的微小rna的用途
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CN115176011A (zh) * 2019-08-27 2022-10-11 赛诺菲 用于抑制pcsk9的组合物和方法
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US20070173473A1 (en) * 2001-05-18 2007-07-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115368916A (zh) * 2016-04-05 2022-11-22 斯特拉斯堡大学 用于捕获分子靶标的液滴内表面工程

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Publication number Publication date
EP2052079A2 (en) 2009-04-29
WO2008011431A8 (en) 2009-11-05
WO2008011431A2 (en) 2008-01-24
CN102124107A (zh) 2011-07-13
AU2007275365A1 (en) 2008-01-24
WO2008011431A3 (en) 2008-07-17
JP2010503382A (ja) 2010-02-04

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