CA2740785C - Modulation of factor 11 expression - Google Patents

Modulation of factor 11 expression Download PDF

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Publication number
CA2740785C
CA2740785C CA2740785A CA2740785A CA2740785C CA 2740785 C CA2740785 C CA 2740785C CA 2740785 A CA2740785 A CA 2740785A CA 2740785 A CA2740785 A CA 2740785A CA 2740785 C CA2740785 C CA 2740785C
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Prior art keywords
compound
factor
modified oligonucleotide
isis
seq
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CA2740785A
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English (en)
French (fr)
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CA2740785A1 (en
Inventor
Susan M. Freier
Brett P. Monia
Hong Zhang
Chenguang Zhao
Jeffrey R. Crosby
Andrew M. Siwkowski
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Priority to CA2966011A priority Critical patent/CA2966011C/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
    • C12N15/113—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
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365—Lactones
    • A61K31/366—Lactones having six-membered rings, e.g. delta-lactones
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4365—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system having sulfur as a ring hetero atom, e.g. ticlopidine
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    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
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    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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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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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21027—Coagulation factor XIa (3.4.21.27)
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    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • 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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  • Dermatology (AREA)
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  • Vascular Medicine (AREA)
  • Diabetes (AREA)
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CA2740785A 2008-10-15 2009-10-15 Modulation of factor 11 expression Active CA2740785C (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA2966011A CA2966011C (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US10577208P 2008-10-15 2008-10-15
US61/105,772 2008-10-15
US17446109P 2009-04-30 2009-04-30
US61/174,461 2009-04-30
PCT/US2009/060922 WO2010045509A2 (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression

Related Child Applications (1)

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CA2966011A Division CA2966011C (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression

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CA2740785A1 CA2740785A1 (en) 2010-04-22
CA2740785C true CA2740785C (en) 2017-06-20

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CA2740785A Active CA2740785C (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression
CA2966011A Active CA2966011C (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression

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US (7) US8334372B2 (enExample)
EP (2) EP2379084B1 (enExample)
JP (4) JP5809058B2 (enExample)
KR (2) KR101979134B1 (enExample)
CN (4) CN102245186B (enExample)
AU (2) AU2009305636A1 (enExample)
BR (1) BRPI0920263B1 (enExample)
CA (2) CA2740785C (enExample)
CY (1) CY1119909T1 (enExample)
DK (1) DK2379084T3 (enExample)
ES (1) ES2657679T3 (enExample)
HR (1) HRP20171969T1 (enExample)
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LT (1) LT2379084T (enExample)
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PL (1) PL2379084T3 (enExample)
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Families Citing this family (43)

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CA2740785C (en) 2008-10-15 2017-06-20 Isis Pharmaceuticals, Inc. Modulation of factor 11 expression
RU2011146158A (ru) * 2009-04-15 2013-05-27 АйЭсАйЭс ФАРМАСЬЮТИКАЛЗ, ИНК. Модуляция воспалительных реакций фактором хi
WO2012064758A2 (en) * 2010-11-08 2012-05-18 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
SI3505528T1 (sl) 2011-04-21 2021-04-30 Glaxo Group Limited Modulacija izražanja virusa hepatitisa B (HBV)
TW201303013A (zh) * 2011-04-21 2013-01-16 Isis Pharmaceuticals Inc B型肝炎病毒(hbv)表現之調節
US9187749B2 (en) 2011-06-10 2015-11-17 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
AU2012327227A1 (en) * 2011-11-07 2013-05-23 Isis Pharmaceuticals, Inc. Administration of Factor XI antisense oligonucleotides
US10502737B2 (en) 2012-05-25 2019-12-10 The University Of Vermont And State Agriculture College Anti-thrombotic compositions and methods for assaying platelet reactivity and treatment selection
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
US10570169B2 (en) 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EA201790336A1 (ru) 2014-08-07 2017-06-30 Новартис Аг Антитела к ангиопоэтинподобному белку 4 и способы применения
TW201613977A (en) 2014-08-07 2016-04-16 Novartis Ag Angiopoetin-like 4 (ANGPTL4) antibodies and methods of use
JOP20160131B1 (ar) 2015-06-26 2022-03-14 Novartis Ag الأجسام المضادة للعامل xi وطرق الاستخدام
TW201802121A (zh) 2016-05-25 2018-01-16 諾華公司 抗因子XI/XIa抗體之逆轉結合劑及其用途
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
KR102616666B1 (ko) 2016-12-23 2023-12-27 노파르티스 아게 인자 xi 항체 및 사용 방법
CN110944675B9 (zh) 2017-12-01 2024-08-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN118291456A (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
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
JP7360716B2 (ja) 2017-12-01 2023-10-13 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および複合体ならびに調製方法と使用
WO2019105414A1 (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
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