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DK2162538T3 - Oligomerer til terapi - Google Patents

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Publication number
DK2162538T3
DK2162538T3 DK08780679.0T DK08780679T DK2162538T3 DK 2162538 T3 DK2162538 T3 DK 2162538T3 DK 08780679 T DK08780679 T DK 08780679T DK 2162538 T3 DK2162538 T3 DK 2162538T3
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Denmark
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rna
monomers
monomer
oligonucleotide
rna duplex
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DK08780679.0T
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Jesper Wengel
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Arcturus Therapeutics Inc
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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
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Claims (24)

1
1. RNA-dupleks, der omfatter mindst ét oligonukleotid, der omfatter en nukleotidsekvens, der er komplementær til et mål-mRNA, hvor oligo- 5 nukleotidet omfatter en acyklisk 2'-3'-seco-nukleotidmonomer, hvor RNA-duplekset øger ekspressionen af et mål-mRNA.
2. RNA-dupleks ifølge krav 1, hvor den acykliske monomer er en 2'-3'-seco-nukleotidmonomer, der har 2’-hydroxygruppen substitueret af en 10 amino-, acyleret amino-, alkyleret amino-, dialkyleret amino-, car- bamoyleret amino-, piperazin-, acyleret piperazin-, alkyleret piperazin-, carbamoyleret piperazin-, mercapto-, acyleret mercapto-, alkyleret mer-capto-, disulfid-, acyleret hydroxy-, alkyleret hydroxy- eller carbamoyleret hydroxygruppe. 15
3. RNA-dupleks ifølge krav 1 eller krav 2, hvor den acykliske nukleotidmo-nomer er udvalgt fra gruppen bestående af monomerer D, F, G, Η, I og J:
Monomer D Monomer F Monomer G 2Q v“
Monomer J hvor R er udvalgt fra gruppen udvalgt fra H, alkyl, cholesterolderivater, fluorphorer, polyaminer, fedtsyrer, aminosyrer, saccharider eller po-lypeptider.
4. RNA-dupleks ifølge et hvilket som helst af kravene 1-3, hvor den acykliske monomer er placeret i seed-regionen af antisensestrengen.
5. RNA-dupleks ifølge et hvilket som helst af kravene 1-4, der omfatter mindst ét oligonukleotid, der omfatter fra 15 til 40 nukleotidmonomerer og en acyklisk nukleotidmonomer.
6. RNA-dupleks ifølge et hvilket som helst af kravene 1-5, der omfatter mindst ét oligonukleotid, der omfatter fra en til fem acykliske nukleotidmonomerer.
7. RNA-dupleks ifølge et hvilket som helst af kravene 1-6, der omfatter mindst ét oligonukleotid, der omfatter mindre end fire konsekutive DNA-monomerer og en acyklisk nukleotidmonomer.
8. RNA-dupleks ifølge et hvilket som helst af kravene 1-7, der omfatter mindst ét oligonukleotid, der omfatter mere end 50 % RNA-monomerer og en acyklisk nukleotidmonomer.
9. RNA-dupleks ifølge et hvilket som helst af kravene 1-8, der omfatter mindst ét oligonukleotid, der omfatter en acyklisk nukleotidmonomer og en nukleotidanalog udvalgt fra 2'-0-alkyl-RNA-monomerer, 2'-amino-DNA-monomerer, 2'-fluor-DNA-monomerer, LNA-monomerer, PNA-monomerer, HNA-monomerer, ANA-monomerer, FANA-monomerer, CeNA-monomerer, ENA-monomerer, DNA-monomerer og INA-monomerer.
10. RNA-dupleks ifølge et hvilket som helst af kravene 1-9, der omfatter mindst ét oligonukleotid, der omfatter en acyklisk nukleotidmonomer og mindst to LNA-nukleotidanaloger.
11. RNA-dupleks ifølge et hvilket som helst af kravene 1-10, der omfatter mindst ét oligonukleotid, der omfatter en acyklisk nukleotidmonomer og en phosphorthioatbinding eller en boranphosphatbinding.
12. RNA-dupleks ifølge et hvilket som helst af kravene 1-11, hvor RNA-dupleks er i stand til at mediere RISC-afhængig translationel repression eller degradering af mål-mRNA’er, der er komplementære til oligonu-kleotidet.
13. RNA-dupleks ifølge et hvilket som helst af kravene 1-12, der omfatter et antal af basepar fra 15 til 40; eller der omfatter et antal basepar fra 14 til 26 basepar.
14. RNA-dupleks ifølge et hvilket som helst af kravene 1-13, der omfatter mindst ét overhæng, fortrinsvis mindst ét 3'-overhæng.
15. RNA-dupleks ifølge krav 14, hvor længden af overhænget er fra 1 til 14 nukleotider.
16. RNA-dupleks ifølge et hvilket som helst af kravene 1-15, der endvidere omfatter mindst ét overhæng af antisensestrengen, der omfatter mindst én acyklisk monomer.
17. RNA-dupleks ifølge et hvilket som helst af kravene 1-16, der omfatter mindst én stump ende.
18. RNA-dupleks ifølge et hvilket som helst af kravene 1-17, hvor RNA-duplekset omfatter et oligonukleotid, der omfatter en nukleotidsekvens, der er komplementær til et mål-mRNA, hvor RNA-duplekset øger ekspressionen af mål-mRNA’et.
19. Oligonukleotid, der omfatter en acyklisk monomer, hvor oligonukleoti-det øger ekspressionen af et mål-mRNA, og hvor den acykliske monomer er en 2'-3'-seco-nukleotidmonomer, der har 2’-hydroxygruppen substitueret af en amino-, acyleret amino-, alkyleret amino-, dialkyleret amino-, carbamoyleret amin-, piperazin-, acyleret piperazin-, alkyleret piperazin-, carbamoyleret piperazin-, mercapto-, acyleret mercapto-, alkyleret mercapto-, disulfid-, acyleret hydroxy-, alkyleret hydroxy- eller carbamoyleret hydroxygruppe.
20. Oligonukleotid, der omfatter en acyklisk monomer udvalgt fra gruppen bestående af monomerer F, G, Η, I og J:
Monomer F Monomer G
Monomer J hvor oligonukleotidet øger ekspressionen af et mål-mRNA, og R er alkyl, et cholesterolderivat, en fluorphor, et polyamin, en fedtsyre, en aminosyre, et saccharid eller et polypeptid.
21. Farmaceutisk sammensætning, der omfatter et oligonukleotid ifølge krav 19 eller krav 20, eller et RNA-dupleks ifølge et hvilket som helst af kravene 1-18, og et farmaceutisk acceptabelt fortyndingsmiddel, bærestof eller hjælpestof.
22. RNA-dupleks ifølge et hvilket som helst af kravene 1-18 til anvendelse til sænkning af ekspressionsniveauet af et mål-mRNA i en celle.
23. RNA-dupleks ifølge et hvilket som helst af kravene 1-18 til anvendelse til behandling af sygdom.
24. RNA-dupleks ifølge et hvilket som helst af kravene 1-18 til anvendelse i RNA styret genregulering eller genanalyse; fortrinsvis til anvendelse i RNA-interferens.
DK08780679.0T 2007-05-22 2008-05-21 Oligomerer til terapi DK2162538T3 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
DKPA200700751 2007-05-22
DKPA200701718 2007-11-30
DKPA200701785 2007-12-14
DKPA200800534 2008-04-11
PCT/US2008/064417 WO2008147824A2 (en) 2007-05-22 2008-05-21 Hydroxymethyl substituted rna oligonucleotides and rna complexes

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DK2162538T3 true DK2162538T3 (da) 2016-06-06

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US (6) US8314227B2 (da)
EP (2) EP3045535B1 (da)
JP (2) JP5726520B2 (da)
KR (2) KR101629017B1 (da)
CN (2) CN101679978B (da)
AU (1) AU2008256871B2 (da)
BR (1) BRPI0811170B8 (da)
CA (1) CA2687850C (da)
DK (1) DK2162538T3 (da)
ES (1) ES2573936T3 (da)
HK (1) HK1138322A1 (da)
IL (1) IL202040A (da)
MX (1) MX2009012568A (da)
MY (1) MY153691A (da)
NZ (1) NZ580712A (da)
PL (1) PL2162538T3 (da)
WO (1) WO2008147824A2 (da)
ZA (1) ZA200907529B (da)

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