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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Pending
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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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    • A61P3/00—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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
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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
    • 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
    • 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
    • A61P9/12—Antihypertensives
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
    • 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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  • Organic Chemistry (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Hematology (AREA)
  • Obesity (AREA)
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  • Endocrinology (AREA)
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  • Urology & Nephrology (AREA)
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  • Gastroenterology & Hepatology (AREA)
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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 (1) EP3221451A1 (enExample)
JP (5) JP2017535552A (enExample)
CN (2) CN107250362B (enExample)
AU (3) AU2015350120B2 (enExample)
CA (1) CA2968114A1 (enExample)
HK (1) HK1244843A1 (enExample)
WO (1) WO2016081444A1 (enExample)

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