HUE052709T2 - Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására - Google Patents

Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására

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Publication number
HUE052709T2
HUE052709T2 HUE15786489A HUE15786489A HUE052709T2 HU E052709 T2 HUE052709 T2 HU E052709T2 HU E15786489 A HUE15786489 A HU E15786489A HU E15786489 A HUE15786489 A HU E15786489A HU E052709 T2 HUE052709 T2 HU E052709T2
Authority
HU
Hungary
Prior art keywords
conjugates
modify
antisense oligonucleotides
modified antisense
pkk expression
Prior art date
Application number
HUE15786489A
Other languages
English (en)
Hungarian (hu)
Inventor
Thazha P Prakash
Punit P Seth
Eric E Swayze
Susan M Freier
Huynh-Hoa Bui
Original Assignee
Ionis 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
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of HUE052709T2 publication Critical patent/HUE052709T2/hu

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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
    • C12N15/1137—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 against enzymes
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K47/555—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 pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
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    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10—Antioedematous agents; Diuretics
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    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N15/09—Recombinant DNA-technology
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    • 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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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/30—Chemical structure
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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HUE15786489A 2014-05-01 2015-05-01 Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására HUE052709T2 (hu)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461987478P 2014-05-01 2014-05-01
US201462058629P 2014-10-01 2014-10-01
US201462088459P 2014-12-05 2014-12-05

Publications (1)

Publication Number Publication Date
HUE052709T2 true HUE052709T2 (hu) 2021-05-28

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HUE15786489A HUE052709T2 (hu) 2014-05-01 2015-05-01 Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására

Country Status (15)

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US (4) US10294477B2 (enExample)
EP (3) EP4534092A3 (enExample)
JP (4) JP6665111B2 (enExample)
KR (1) KR102366078B1 (enExample)
CN (1) CN106232125B (enExample)
AU (2) AU2015252917B2 (enExample)
BR (1) BR112016022855B1 (enExample)
CA (1) CA2943705A1 (enExample)
DK (1) DK3137091T3 (enExample)
ES (1) ES2849600T3 (enExample)
HU (1) HUE052709T2 (enExample)
IL (2) IL247862B (enExample)
MX (2) MX377141B (enExample)
RU (1) RU2703411C2 (enExample)
WO (1) WO2015168532A2 (enExample)

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WO2019105418A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
KR102834361B1 (ko) 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
JP7365052B2 (ja) 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
CA3087106A1 (en) 2017-12-29 2019-07-04 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
JP2021533800A (ja) 2018-08-21 2021-12-09 スーチョウ リボ ライフ サイエンス カンパニー、リミテッドSuzhou Ribo Life Science Co., Ltd. 核酸、当該核酸を含む薬物組成物及び複合体ならびにその使用
CN111655297A (zh) 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
CN113891939B (zh) * 2019-05-24 2024-04-02 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN115362256A (zh) * 2020-02-07 2022-11-18 因特利亚治疗公司 用于激肽释放酶（klkb1）基因编辑的组合物和方法
EP4118208A4 (en) * 2020-03-13 2025-03-12 Ionis Pharmaceuticals, Inc. Compositions and methods for treating and preventing prekallikrein-associated diseases
CN116669746B (zh) * 2020-12-29 2025-09-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
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CN116917477B (zh) * 2022-01-30 2024-04-09 大睿生物医药科技(上海)有限公司 含有n-乙酰半乳糖胺的靶向配体
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