CA2921839A1 - Modulation of prekallikrein (pkk) expression - Google Patents

Modulation of prekallikrein (pkk) expression Download PDF

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Publication number
CA2921839A1
CA2921839A1 CA2921839A CA2921839A CA2921839A1 CA 2921839 A1 CA2921839 A1 CA 2921839A1 CA 2921839 A CA2921839 A CA 2921839A CA 2921839 A CA2921839 A CA 2921839A CA 2921839 A1 CA2921839 A1 CA 2921839A1
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CA
Canada
Prior art keywords
compound
eekdiokke
seq
modified oligonucleotide
certain embodiments
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Abandoned
Application number
CA2921839A
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English (en)
French (fr)
Inventor
Susan M. Freier
Huynh-Hoa Bui
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52587489&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CA2921839(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of CA2921839A1 publication Critical patent/CA2921839A1/en
Abandoned legal-status Critical Current

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    • 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
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A—HUMAN NECESSITIES
    • 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]
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10—Antioedematous agents; Diuretics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • 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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    • 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
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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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
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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
    • 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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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21034—Plasma kallikrein (3.4.21.34)
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
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    • 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
    • C12N2310/31—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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
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CA2921839A 2013-08-28 2014-08-28 Modulation of prekallikrein (pkk) expression Abandoned CA2921839A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361871175P 2013-08-28 2013-08-28
US61/871,175 2013-08-28
PCT/US2014/053266 WO2015031679A2 (en) 2013-08-28 2014-08-28 Modulation of prekallikrein (pkk) expression

Publications (1)

Publication Number Publication Date
CA2921839A1 true CA2921839A1 (en) 2015-03-05

Family

ID=52587489

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2921839A Abandoned CA2921839A1 (en) 2013-08-28 2014-08-28 Modulation of prekallikrein (pkk) expression

Country Status (14)

Country Link
US (5) US9670492B2 (enExample)
EP (2) EP3715457A3 (enExample)
JP (2) JP6652922B2 (enExample)
KR (1) KR102365486B1 (enExample)
CN (1) CN105517556B (enExample)
AU (2) AU2014312196C1 (enExample)
BR (1) BR112016004093A2 (enExample)
CA (1) CA2921839A1 (enExample)
ES (1) ES2790574T3 (enExample)
IL (1) IL244047A0 (enExample)
MX (1) MX2016002587A (enExample)
NZ (1) NZ716816A (enExample)
RU (1) RU2712559C9 (enExample)
WO (1) WO2015031679A2 (enExample)

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AU2014312196C1 (en) 2013-08-28 2019-12-19 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
MX377141B (es) 2014-05-01 2025-03-07 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de pkk.
JP7319916B2 (ja) 2016-09-16 2023-08-02 武田薬品工業株式会社 遺伝性血管性浮腫のｒｎａバイオマーカー
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2019105435A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
HRP20250277T1 (hr) 2017-12-01 2025-05-09 Suzhou Ribo Life Science Co., Ltd. Dvolančani oligonukleotid, sastav i konjugat koji sadrži dvolančani oligonukleotid, postupak njihove pripreme i upotreba
US12083142B2 (en) 2017-12-01 2024-09-10 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
WO2019105419A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN118291457A (zh) 2017-12-01 2024-07-05 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CA3087106A1 (en) 2017-12-29 2019-07-04 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
EP3992290A4 (en) * 2019-05-24 2023-11-15 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND CONJUGATE, METHOD FOR THE PRODUCTION THEREOF AND USE THEREOF
IL295280A (en) * 2020-02-07 2022-10-01 Intellia Therapeutics Inc Preparations and methods for editing the kallikrein gene (klkb1)
IL295631A (en) * 2020-03-13 2022-10-01 Ionis Pharmaceuticals Inc Compositions and methods for the treatment and prevention of conditions associated with prekallikrein
WO2021248027A1 (en) * 2020-06-05 2021-12-09 Ionis Pharmaceuticals, Inc. Compositions and methods of inhibiting angiotensin-converting enzyme 2 (ace2) and transmembrane serine protease 2 (tmprss2)
TW202227101A (zh) 2020-11-18 2022-07-16 美商Ionis製藥公司 用於調節血管收縮素原表現之化合物及方法
US20250320500A1 (en) * 2020-12-29 2025-10-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and sirna conjugate containing the nucleic acid, preparation method therefor, and use thereof
US11833221B2 (en) * 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
KR20240082351A (ko) 2021-10-01 2024-06-10 아다르엑스 파마슈티컬스, 인크. 프리칼리크레인 조절 조성물 및 이의 사용 방법
TW202346586A (zh) * 2022-01-28 2023-12-01 大陸商上海舶望製藥有限公司 用於抑制前激肽釋放酶（pkk）蛋白表達的組合物和方法

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