AU2018215684B2 - Multimeric oligonucleotides having decreased kidney clearance - Google Patents

Multimeric oligonucleotides having decreased kidney clearance Download PDF

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AU2018215684B2
AU2018215684B2 AU2018215684A AU2018215684A AU2018215684B2 AU 2018215684 B2 AU2018215684 B2 AU 2018215684B2 AU 2018215684 A AU2018215684 A AU 2018215684A AU 2018215684 A AU2018215684 A AU 2018215684A AU 2018215684 B2 AU2018215684 B2 AU 2018215684B2
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oligonucleotide
multimeric oligonucleotide
multimeric
oligonucleotides
provides
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AU2018215684A1 (en
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Jonathan Miles Brown
Philipp Hadwiger
Kristin K.H. Neuman
Hans-Peter Vornlocher
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MPEG LA LLC
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MPEG LA LLC
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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Engineering & Computer Science (AREA)
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  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
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  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
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AU2018215684A 2017-02-06 2018-02-06 Multimeric oligonucleotides having decreased kidney clearance Active AU2018215684B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762455231P 2017-02-06 2017-02-06
US62/455,231 2017-02-06
US201762522363P 2017-06-20 2017-06-20
US62/522,363 2017-06-20
US201762561853P 2017-09-22 2017-09-22
US62/561,853 2017-09-22
PCT/US2018/017062 WO2018145086A1 (en) 2017-02-06 2018-02-06 Multimeric oligonucleotides having decreased kidney clearance

Publications (2)

Publication Number Publication Date
AU2018215684A1 AU2018215684A1 (en) 2019-08-01
AU2018215684B2 true AU2018215684B2 (en) 2024-03-07

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US (2) US11078484B2 (enExample)
EP (1) EP3576752A4 (enExample)
JP (1) JP7183167B2 (enExample)
KR (2) KR102623311B1 (enExample)
CN (1) CN110248665A (enExample)
AU (1) AU2018215684B2 (enExample)
CA (1) CA3051480A1 (enExample)
IL (2) IL267806B2 (enExample)
MX (1) MX2019009347A (enExample)
SG (1) SG11201906728TA (enExample)
WO (1) WO2018145086A1 (enExample)

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JP2022523226A (ja) * 2019-03-04 2022-04-21 エムペグ エルエイ リミテッド ライアビリティ カンパニー 増強された生理活性を有する多量体オリゴヌクレオチド
AU2020252020A1 (en) * 2019-03-29 2021-11-11 University Of Massachusetts Oligonucleotide-based modulation of C90RF72
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CN110248665A (zh) 2019-09-17
KR102776167B1 (ko) 2025-03-05
IL267806A (en) 2019-09-26
AU2018215684A1 (en) 2019-08-01
US12378551B2 (en) 2025-08-05
IL267806B2 (en) 2024-03-01
IL267806B1 (en) 2023-11-01
WO2018145086A1 (en) 2018-08-09
JP2020518552A (ja) 2020-06-25
KR102623311B1 (ko) 2024-01-10
KR20190115445A (ko) 2019-10-11
US20210380979A1 (en) 2021-12-09
CA3051480A1 (en) 2018-08-09
EP3576752A4 (en) 2020-12-16
US11078484B2 (en) 2021-08-03
MX2019009347A (es) 2019-10-07
KR20240010750A (ko) 2024-01-24
EP3576752A1 (en) 2019-12-11
US20180223284A1 (en) 2018-08-09
JP7183167B2 (ja) 2022-12-05
IL307726A (en) 2023-12-01
SG11201906728TA (en) 2019-08-27

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