AU2016280709B2 - Defined multi-conjugate oligonucleotides - Google Patents

Defined multi-conjugate oligonucleotides Download PDF

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Publication number
AU2016280709B2
AU2016280709B2 AU2016280709A AU2016280709A AU2016280709B2 AU 2016280709 B2 AU2016280709 B2 AU 2016280709B2 AU 2016280709 A AU2016280709 A AU 2016280709A AU 2016280709 A AU2016280709 A AU 2016280709A AU 2016280709 B2 AU2016280709 B2 AU 2016280709B2
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compound
oligonucleotides
thiol
sirna
single stranded
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AU2016280709A1 (en
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Jonathan Miles Brown
James Everett Dahlman
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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Priority to AU2022283737A priority Critical patent/AU2022283737B2/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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • 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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AU2016280709A 2015-06-15 2016-06-15 Defined multi-conjugate oligonucleotides Active AU2016280709B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2022283737A AU2022283737B2 (en) 2015-06-15 2022-12-08 Defined multi-conjugate oligonucleotides
AU2024278244A AU2024278244A1 (en) 2015-06-15 2024-12-10 Defined multi-conjugate oligonucleotides

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201562175718P 2015-06-15 2015-06-15
US201562175714P 2015-06-15 2015-06-15
US201562175709P 2015-06-15 2015-06-15
US62/175,718 2015-06-15
US62/175,714 2015-06-15
US62/175,709 2015-06-15
US201562203243P 2015-08-10 2015-08-10
US62/203,243 2015-08-10
US201562216318P 2015-09-09 2015-09-09
US201562216317P 2015-09-09 2015-09-09
US201562216314P 2015-09-09 2015-09-09
US62/216,317 2015-09-09
US62/216,314 2015-09-09
US62/216,318 2015-09-09
PCT/US2016/037685 WO2016205410A2 (en) 2015-06-15 2016-06-15 Defined multi-conjugate oligonucleotides

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AU2016280709B2 true AU2016280709B2 (en) 2022-09-15

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US (3) US11352629B2 (enExample)
EP (2) EP4541374A2 (enExample)
JP (1) JP7033452B2 (enExample)
KR (2) KR20250016526A (enExample)
CN (1) CN108026527B (enExample)
AU (3) AU2016280709B2 (enExample)
CA (1) CA2988603A1 (enExample)
HK (1) HK1252970A1 (enExample)
IL (2) IL316159A (enExample)
MX (2) MX2017016088A (enExample)
WO (1) WO2016205410A2 (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
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CA3144467A1 (en) * 2019-07-30 2021-02-04 Jonathan Miles Brown Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity
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US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
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CN112111524B (zh) * 2020-01-10 2024-02-27 深圳瑞吉生物科技有限公司 mRNA-GalNAc靶向分子的制备方法及其体内递送系统和应用
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JP2024506042A (ja) * 2021-02-08 2024-02-08 ラクティゲン セラピューティクス 多価オリゴヌクレオチド薬剤およびその使用方法
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CN115010794A (zh) * 2022-06-30 2022-09-06 武汉瑞佶生物科技有限公司 蛋白质介导的mRNA靶向分子及其制备方法和应用
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