HRP20171969T1 - Modulacija ekspresije faktora 11 - Google Patents

Modulacija ekspresije faktora 11 Download PDF

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Publication number
HRP20171969T1
HRP20171969T1 HRP20171969TT HRP20171969T HRP20171969T1 HR P20171969 T1 HRP20171969 T1 HR P20171969T1 HR P20171969T T HRP20171969T T HR P20171969TT HR P20171969 T HRP20171969 T HR P20171969T HR P20171969 T1 HRP20171969 T1 HR P20171969T1
Authority
HR
Croatia
Prior art keywords
segment
wing
compound according
modified oligonucleotide
modified
Prior art date
Application number
HRP20171969TT
Other languages
English (en)
Croatian (hr)
Inventor
Susan M. Freier
Brett P. Monia
Hong Zhang
Chenguang Zhao
Jeffrey R. Crosby
Andrew M. Siwkowski
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 HRP20171969T1 publication Critical patent/HRP20171969T1/hr

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365Lactones
    • A61K31/366Lactones having six-membered rings, e.g. delta-lactones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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
    • A61K31/4365Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/4523Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545Non 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
    • AHUMAN NECESSITIES
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/726Glycosaminoglycans, i.e. mucopolysaccharides
    • A61K31/727Heparin; Heparan
    • AHUMAN NECESSITIES
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/737Sulfated polysaccharides, e.g. chondroitin sulfate, dermatan sulfate
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    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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    • A61P9/10Drugs 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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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/14Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
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    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21027Coagulation factor XIa (3.4.21.27)
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Wood Science & Technology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Cardiology (AREA)
  • Microbiology (AREA)
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  • Plant Pathology (AREA)
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  • Heart & Thoracic Surgery (AREA)
  • Dermatology (AREA)
  • Virology (AREA)
  • Vascular Medicine (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Urology & Nephrology (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
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HRP20171969TT 2008-10-15 2009-10-15 Modulacija ekspresije faktora 11 HRP20171969T1 (hr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US10577208P 2008-10-15 2008-10-15
US17446109P 2009-04-30 2009-04-30
PCT/US2009/060922 WO2010045509A2 (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression
EP09821293.9A EP2379084B1 (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression

Publications (1)

Publication Number Publication Date
HRP20171969T1 true HRP20171969T1 (hr) 2018-04-06

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Application Number Title Priority Date Filing Date
HRP20171969TT HRP20171969T1 (hr) 2008-10-15 2009-10-15 Modulacija ekspresije faktora 11

Country Status (23)

Country Link
US (7) US8334372B2 (OSRAM)
EP (2) EP2379084B1 (OSRAM)
JP (4) JP5809058B2 (OSRAM)
KR (2) KR101773551B1 (OSRAM)
CN (4) CN102245186B (OSRAM)
AU (2) AU2009305636A1 (OSRAM)
BR (1) BRPI0920263B1 (OSRAM)
CA (2) CA2966011C (OSRAM)
CY (1) CY1119909T1 (OSRAM)
DK (1) DK2379084T3 (OSRAM)
ES (1) ES2657679T3 (OSRAM)
HR (1) HRP20171969T1 (OSRAM)
HU (1) HUE035888T2 (OSRAM)
IL (2) IL212267A (OSRAM)
LT (1) LT2379084T (OSRAM)
MX (1) MX2011004097A (OSRAM)
NO (1) NO2379084T3 (OSRAM)
NZ (2) NZ592203A (OSRAM)
PL (1) PL2379084T3 (OSRAM)
PT (1) PT2379084T (OSRAM)
RU (2) RU2535964C2 (OSRAM)
SI (1) SI2379084T1 (OSRAM)
WO (1) WO2010045509A2 (OSRAM)

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KR102834361B1 (ko) 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
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WO2019204308A1 (en) * 2018-04-20 2019-10-24 The University Of Vermont And State Agriculture College Compositions and methods for treating cardiovascular disease in selected patients
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KR20210110839A (ko) 2018-12-28 2021-09-09 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 핵산을 함유하는 조성물 및 접합체, 이의 제조 방법 및 용도
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