CA2968114A1 - Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof - Google Patents

Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof Download PDF

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Publication number
CA2968114A1
CA2968114A1 CA2968114A CA2968114A CA2968114A1 CA 2968114 A1 CA2968114 A1 CA 2968114A1 CA 2968114 A CA2968114 A CA 2968114A CA 2968114 A CA2968114 A CA 2968114A CA 2968114 A1 CA2968114 A1 CA 2968114A1
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Prior art keywords
nucleotides
nucleotide
apoc3
strand
irna
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CA2968114A
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English (en)
French (fr)
Inventor
Kevin Fitzgerald
William Querbes
James Butler
Stephanie Williams
Abigail LIEBOW
Gregory Hinkle
Martin Maier
Stuart Milstein
Satyanarayana Kuchimanchi
Muthiah Manoharan
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Alnylam Pharmaceuticals Inc
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Alnylam Pharmaceuticals Inc
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Publication of CA2968114A1 publication Critical patent/CA2968114A1/en
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61P3/00—Drugs for disorders of the metabolism
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
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    • 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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    • 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]
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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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CA2968114A 2014-11-17 2015-11-17 Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof Pending CA2968114A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462080941P 2014-11-17 2014-11-17
US62/080,941 2014-11-17
US201562136159P 2015-03-20 2015-03-20
US62/136,159 2015-03-20
PCT/US2015/061065 WO2016081444A1 (en) 2014-11-17 2015-11-17 Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
CA2968114A1 true CA2968114A1 (en) 2016-05-26

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CA2968114A Pending CA2968114A1 (en) 2014-11-17 2015-11-17 Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof

Country Status (8)

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US (7) US10407679B2 (enExample)
EP (2) EP3221451A1 (enExample)
JP (5) JP2017535552A (enExample)
CN (2) CN113846101A (enExample)
AU (3) AU2015350120B2 (enExample)
CA (1) CA2968114A1 (enExample)
HK (1) HK1244843A1 (enExample)
WO (1) WO2016081444A1 (enExample)

Cited By (1)

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