CA3053709A1 - Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders - Google Patents

Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders Download PDF

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Publication number
CA3053709A1
CA3053709A1 CA3053709A CA3053709A CA3053709A1 CA 3053709 A1 CA3053709 A1 CA 3053709A1 CA 3053709 A CA3053709 A CA 3053709A CA 3053709 A CA3053709 A CA 3053709A CA 3053709 A1 CA3053709 A1 CA 3053709A1
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sequence
dna
pcsk9
gene
cell
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French (fr)
Inventor
Ante Sven LUNDBERG
Samarth Kulkarni
Lawrence Klein
Yvonne Sarah Aratyn
Roman Lvovitch Bogorad
Hari Kumar PADMANABHAN
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CRISPR Therapeutics AG
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CRISPR Therapeutics AG
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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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/37Digestive system
    • A61K35/407Liver; Hepatocytes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
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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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6402Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from non-mammals
    • C12N9/6405Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from non-mammals not being snakes
    • CCHEMISTRY; METALLURGY
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    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21061Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21111Aqualysin 1 (3.4.21.111)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Dispersion Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Materials For Medical Uses (AREA)
  • Medicinal Preparation (AREA)
CA3053709A 2017-02-22 2018-02-15 Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders Pending CA3053709A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762461852P 2017-02-22 2017-02-22
US62/461,852 2017-02-22
US201762526646P 2017-06-29 2017-06-29
US62/526,646 2017-06-29
PCT/IB2018/000208 WO2018154380A1 (en) 2017-02-22 2018-02-15 Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders

Publications (1)

Publication Number Publication Date
CA3053709A1 true CA3053709A1 (en) 2018-08-30

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CA3053709A Pending CA3053709A1 (en) 2017-02-22 2018-02-15 Compositions and methods for treatment of proprotein convertase subtilisin/kexin type 9 (pcsk9)-related disorders

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US (1) US20200248168A1 (enExample)
EP (1) EP3585894A1 (enExample)
JP (1) JP7277052B2 (enExample)
CN (1) CN110582570A (enExample)
AU (2) AU2018224380A1 (enExample)
CA (1) CA3053709A1 (enExample)
WO (1) WO2018154380A1 (enExample)

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US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
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US20200248168A1 (en) 2020-08-06
WO2018154380A1 (en) 2018-08-30
EP3585894A1 (en) 2020-01-01
CN110582570A (zh) 2019-12-17
JP2020508659A (ja) 2020-03-26
JP7277052B2 (ja) 2023-05-18
AU2018224380A1 (en) 2019-08-29
AU2024205415A1 (en) 2024-10-24
WO2018154380A8 (en) 2019-09-06

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