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
Inventor
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
Priority to AU2024278244A priority patent/AU2024278244A1/en
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    • C—CHEMISTRY; METALLURGY
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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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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    • C12N2310/14—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
    • C12N2310/317—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
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    • 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
    • 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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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • 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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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
US201562175709P 2015-06-15 2015-06-15
US201562175714P 2015-06-15 2015-06-15
US201562175718P 2015-06-15 2015-06-15
US62/175,718 2015-06-15
US62/175,709 2015-06-15
US62/175,714 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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US (3) US11352629B2 (https=)
EP (2) EP4541374A3 (https=)
JP (1) JP7033452B2 (https=)
KR (2) KR20250016526A (https=)
CN (1) CN108026527B (https=)
AU (3) AU2016280709B2 (https=)
CA (1) CA2988603A1 (https=)
HK (1) HK1252970A1 (https=)
IL (2) IL316159A (https=)
MX (2) MX2017016088A (https=)
WO (1) WO2016205410A2 (https=)

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JP2022543191A (ja) * 2019-07-30 2022-10-11 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の皮下送達
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
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CN111041025B (zh) 2019-12-17 2021-06-18 深圳市瑞吉生物科技有限公司 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法
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
KR20230003579A (ko) * 2020-04-30 2023-01-06 엠펙 엘에이, 엘.엘.씨. 분할된 가닥을 갖는 다량체 올리고뉴클레오타이드
CA3178559A1 (en) * 2020-05-19 2021-11-25 Jonathan Miles Brown Orthogonally linked multimeric oligonucleotides
WO2021242883A1 (en) 2020-05-26 2021-12-02 University Of Massachusetts Synthetic oligonucleotides having regions of block and cluster modifications
CN111744019B (zh) 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
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
CN117677699A (zh) 2021-06-23 2024-03-08 马萨诸塞大学 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物
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
JP2025531341A (ja) * 2022-09-19 2025-09-19 カイロンノヴァ （シャーメン） バイオファーマ カンパニー，リミテッド 炭水化物－オリゴヌクレオチド複合体、薬物組成物および治療への応用
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