AU2017297624B2 - Compounds and methods for modulation of transcript processing - Google Patents

Compounds and methods for modulation of transcript processing Download PDF

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Publication number
AU2017297624B2
AU2017297624B2 AU2017297624A AU2017297624A AU2017297624B2 AU 2017297624 B2 AU2017297624 B2 AU 2017297624B2 AU 2017297624 A AU2017297624 A AU 2017297624A AU 2017297624 A AU2017297624 A AU 2017297624A AU 2017297624 B2 AU2017297624 B2 AU 2017297624B2
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modified
oligomeric compound
certain embodiments
modified oligonucleotide
nucleosides
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AU2017297624A1 (en
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Thazha P. Prakash
Frank Rigo
Punit P. Seth
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • 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
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    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of 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
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    • C12N2310/00—Structure or type of the nucleic acid
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AU2017297624A 2016-07-15 2017-07-17 Compounds and methods for modulation of transcript processing Active AU2017297624B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662363193P 2016-07-15 2016-07-15
US62/363,193 2016-07-15
PCT/US2017/042465 WO2018014043A1 (en) 2016-07-15 2017-07-17 Compounds and methods for modulation of transcript processing

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AU2017297624A1 AU2017297624A1 (en) 2018-11-29
AU2017297624B2 true AU2017297624B2 (en) 2023-11-02

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US (2) US20190321387A1 (enExample)
EP (1) EP3485016A4 (enExample)
JP (2) JP7211933B2 (enExample)
AU (1) AU2017297624B2 (enExample)
CA (1) CA3027177A1 (enExample)
WO (1) WO2018014043A1 (enExample)

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US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
WO2018014043A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
AR121446A1 (es) 2020-02-28 2022-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2
WO2023178144A2 (en) 2022-03-16 2023-09-21 Empirico Inc. Galnac compositions for improving sirna bioavailability
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides

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US20160068845A1 (en) * 2010-07-19 2016-03-10 University Of Rochester Modulation of dystrophia myotonica-protein kinase (dmpk) expression

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US6147200A (en) * 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
DK2548560T3 (en) * 2005-06-23 2015-07-13 Cold Spring Harbor Lab Compositions and Methods for Modulating SMN2 Splicing
US20070213292A1 (en) * 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US9102938B2 (en) * 2010-04-01 2015-08-11 Alnylam Pharmaceuticals, Inc. 2′ and 5′ modified monomers and oligonucleotides
WO2011159836A2 (en) * 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
GB201410693D0 (en) * 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2018014043A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of transcript processing
AR121446A1 (es) * 2020-02-28 2022-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para modular smn2

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
US20160068845A1 (en) * 2010-07-19 2016-03-10 University Of Rochester Modulation of dystrophia myotonica-protein kinase (dmpk) expression

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JP2019527549A (ja) 2019-10-03
CA3027177A1 (en) 2018-01-18
JP7211933B2 (ja) 2023-01-24
AU2017297624A1 (en) 2018-11-29
US20210315918A1 (en) 2021-10-14
US20190321387A1 (en) 2019-10-24
WO2018014043A1 (en) 2018-01-18
EP3485016A1 (en) 2019-05-22
EP3485016A4 (en) 2020-03-18
JP2022188237A (ja) 2022-12-20

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