WO2014205451A3 - Compositions and methods for modulation of target nucleic acids - Google Patents

Compositions and methods for modulation of target nucleic acids Download PDF

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Publication number
WO2014205451A3
WO2014205451A3 PCT/US2014/043731 US2014043731W WO2014205451A3 WO 2014205451 A3 WO2014205451 A3 WO 2014205451A3 US 2014043731 W US2014043731 W US 2014043731W WO 2014205451 A3 WO2014205451 A3 WO 2014205451A3
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WO
WIPO (PCT)
Prior art keywords
modulation
methods
target nucleic
compositions
nucleic acids
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2014/043731
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French (fr)
Other versions
WO2014205451A2 (en
Inventor
Thazha P. Prakash
Walter F. Lima
Garth A. Kinberger
Heather MURRAY
Eric E. Swayze
Stanley T. Crooke
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Ionis Pharmaceuticals Inc
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Isis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to EP20173519.8A priority Critical patent/EP3730619A1/en
Priority to JP2016521893A priority patent/JP6694382B2/en
Priority to EP19179457.7A priority patent/EP3564374A1/en
Priority to CA2916252A priority patent/CA2916252A1/en
Priority to AU2014284152A priority patent/AU2014284152B2/en
Priority to EP14813192.3A priority patent/EP3011028B1/en
Priority to US14/898,873 priority patent/US20160122761A1/en
Application filed by Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Publication of WO2014205451A2 publication Critical patent/WO2014205451A2/en
Publication of WO2014205451A3 publication Critical patent/WO2014205451A3/en
Anticipated expiration legal-status Critical
Priority to US16/181,206 priority patent/US20190127737A1/en
Priority to AU2019284048A priority patent/AU2019284048A1/en
Priority to US16/813,241 priority patent/US20200270609A1/en
Ceased legal-status Critical Current

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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
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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Abstract

The present disclosure pertains generally to chemically-modified oligonucleotides for use in research, diagnostics, and/or therapeutics. In certain embodiments, the present disclosure describes compounds and methods for the modulation of a target nucleic acid. In certain embodiments, the present disclosure describes compounds and methods for the modulation of Apoliprotein C-III expression.
PCT/US2014/043731 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids Ceased WO2014205451A2 (en)

Priority Applications (10)

Application Number Priority Date Filing Date Title
US14/898,873 US20160122761A1 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids
EP19179457.7A EP3564374A1 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids
CA2916252A CA2916252A1 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids
AU2014284152A AU2014284152B2 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids
EP14813192.3A EP3011028B1 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids
EP20173519.8A EP3730619A1 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids
JP2016521893A JP6694382B2 (en) 2013-06-21 2014-06-23 Compositions and methods for modulating target nucleic acids
US16/181,206 US20190127737A1 (en) 2013-06-21 2018-11-05 Single-stranded rnai oligonucleotides targeting apoc-iii
AU2019284048A AU2019284048A1 (en) 2013-06-21 2019-12-23 Compositions and methods for modulation of target nucleic acids
US16/813,241 US20200270609A1 (en) 2013-06-21 2020-03-09 Single-Stranded RNAI Oligonucleotides Targeting APOC-III

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361838190P 2013-06-21 2013-06-21
US61/838,190 2013-06-21
US201361871683P 2013-08-29 2013-08-29
US61/871,683 2013-08-29

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US14/898,873 A-371-Of-International US20160122761A1 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids
US16/181,206 Continuation US20190127737A1 (en) 2013-06-21 2018-11-05 Single-stranded rnai oligonucleotides targeting apoc-iii

Publications (2)

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WO2014205451A2 WO2014205451A2 (en) 2014-12-24
WO2014205451A3 true WO2014205451A3 (en) 2015-02-19

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PCT/US2014/043731 Ceased WO2014205451A2 (en) 2013-06-21 2014-06-23 Compositions and methods for modulation of target nucleic acids

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US (3) US20160122761A1 (en)
EP (3) EP3730619A1 (en)
JP (2) JP6694382B2 (en)
AU (2) AU2014284152B2 (en)
CA (1) CA2916252A1 (en)
WO (1) WO2014205451A2 (en)

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