AU2009241591A1 - Optimized methods for delivery of DSRNA targeting the PCSK9 gene - Google Patents

Optimized methods for delivery of DSRNA targeting the PCSK9 gene Download PDF

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Publication number
AU2009241591A1
AU2009241591A1 AU2009241591A AU2009241591A AU2009241591A1 AU 2009241591 A1 AU2009241591 A1 AU 2009241591A1 AU 2009241591 A AU2009241591 A AU 2009241591A AU 2009241591 A AU2009241591 A AU 2009241591A AU 2009241591 A1 AU2009241591 A1 AU 2009241591A1
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AU
Australia
Prior art keywords
dsrna
pcsk9
nucleotide
cholesterol
days
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Abandoned
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AU2009241591A
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Inventor
Akin Akinc
Antonin De Fougerolles
Kevin Fitzgerald
Victor E. Kotelianski
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of AU2009241591A1 publication Critical patent/AU2009241591A1/en
Abandoned legal-status Critical Current

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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  • Diabetes (AREA)
  • Obesity (AREA)
  • Virology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2009241591A 2008-01-31 2009-01-30 Optimized methods for delivery of DSRNA targeting the PCSK9 gene Abandoned AU2009241591A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US2496808P 2008-01-31 2008-01-31
US61/024,968 2008-01-31
US3908308P 2008-03-24 2008-03-24
US61/039,083 2008-03-24
US7654808P 2008-06-27 2008-06-27
US61/076,548 2008-06-27
US18876508P 2008-08-11 2008-08-11
US61/188,765 2008-08-11
PCT/US2009/032743 WO2009134487A2 (en) 2008-01-31 2009-01-30 Optimized methods for delivery of dsrna targeting the pcsk9 gene

Publications (1)

Publication Number Publication Date
AU2009241591A1 true AU2009241591A1 (en) 2009-11-05

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AU2009241591A Abandoned AU2009241591A1 (en) 2008-01-31 2009-01-30 Optimized methods for delivery of DSRNA targeting the PCSK9 gene

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US (2) US20100010066A1 (enExample)
EP (1) EP2245039A4 (enExample)
JP (1) JP2011511004A (enExample)
AU (1) AU2009241591A1 (enExample)
BR (1) BRPI0907008A2 (enExample)
CA (1) CA2713379A1 (enExample)
MX (1) MX2010008394A (enExample)
WO (1) WO2009134487A2 (enExample)

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