DK2410054T3 - Antisense forbindelser - Google Patents

Antisense forbindelser Download PDF

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Publication number
DK2410054T3
DK2410054T3 DK11186203.3T DK11186203T DK2410054T3 DK 2410054 T3 DK2410054 T3 DK 2410054T3 DK 11186203 T DK11186203 T DK 11186203T DK 2410054 T3 DK2410054 T3 DK 2410054T3
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nucleoside
antisense
wing
nucleosides
antisense oligonucleotide
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DK11186203.3T
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DK2410054T4 (da
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Eric E Swayze
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Ionis Pharmaceuticals Inc
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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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-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
    • 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
    • C12N2310/30—Chemical structure
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    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
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  • Molecular Biology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Claims (18)

1. Gapmer antisense oligonukleotid med et deoxy-mellemrum og mindst en modificeret internukleosidbinding, et 5' sideområde placeret ved 5' enden af deoxy-mellemrummet, og et 3' sideområde placeret ved 3' enden af deoxy-mellemrummet, hvor 3' siden har mindst et 4' til 2' bicyklisk nukleosid og 5' siden har mindst et ikke-bicyklisk 2'-modificeret nukleosid.
2. Gapmer antisense oligonukleotid ifølge krav 1, hvor det ikke-bicykliske 2'-modificerede nukleosid er substitueret ved 2' positionen med et substitueret eller ikke-substitueret -O-alkyl eller substitueret eller ikke-substitueret -0-(2-acetylamid), hvor det ikke-bicykliske 2'-modificerede nukleosid omfatter et 2'-OCH3, 2'-0(CH2)20CH3, eller 2'-0CH2C(0)-NRiR2, hvor Ri og R2 uafhængigt er hydrogen eller substitueret eller ikke-substitueret alkyl eller, alternativt, tages sammen for at lave en heterocyklisk del.
3. Gapmer antisense oligonukleotid ifølge krav 2, hvor det ikke-bicykliske 2'-modificerede nukleosid er et 2'-0-methylnukleosid, eller hvor det ikke-bicykliske 2'-modificerede nukleosid er et 2'-0-methoxyethylnukleosid.
4. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 3, hvor det 4' til 2' bicykliske nukleosid er et LNA-nukleosid (et methylenoxy (4'-(CH2-O-2') bicyklisk nukleosid) eller ethylenoxy (4'-CH2CH2-0-2') bicyklisk nukleosid.
5. Gapmer antisense oligonukleotid ifølge et hvilket som helst af krav 4, hvor det 4' til 2' bicykliske nukleosid er α-L-LNA eller β-D-LNA.
6. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 5, hvor sideområderne er på mellem ca. 1 til ca. 7 nukleosider i længden.
7. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 5, hvor sideområderne er på mellem ca. 1 til ca. 3 nukleosider i længden.
8. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 7, hvor deoxy-mellemrumsområdet er 6 til 18 nukleosider i længden.
9. Gapmer antisense oligonukleotid ifølge krav 8, hvor deoxy-mellemrumsområdet er 7 til 10 nukleosider i længden.
10. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 9, hvor gapmer antisense oligonukleotidet ikke har nogen BNA'er i 5' sideområdet.
11. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 9, med et 5' sideområde kun med 2'-0(CH2)0CH3 modificerede nukleotider og kun LNA-nukleosider eller ethylenoxy-BNA'er i 3' siden.
12. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 9, hvor 5' siden omfatter mindst et 4' til 2' bicyklisk nukleosid.
13. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 12, hvor 3' siden omfatter mindst et ikke-bicyklisk 2' modificeret nukleosid.
14. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 13, hvor det 4' til 2' bicykliske nukleosid er et methylenoxy (4'-CH2-0-2') bicyklisk nukleosid eller ethylenoxy (4'-CH2CH2-0-2') bicyklisk nukleosid.
15. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 14, hvor det ikke-bicykliske 2'-modificeret nukleosid er substitueret ved 2' positionen med et substitueret eller ikke-substitueret -O-alkyl eller substitueret eller ikke-substitueret -0-(2-acetylamid).
16. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 15, hvor den modificerede internukleosidbinding er et phosphorthioat, hvor den oligomere forbindelse har en flerhed af phosphorthioat-internukleosidbindinger.
17. Gapmer antisense oligonukleotid ifølge et hvilket som helst af de foregående krav, som er 10 til 25, 10 til 30, 10 til 14, 12 til 25, 15 til 25 eller 18 til 24 nukleotider i længden.
18. Gapmer antisense oligonukleotid ifølge et hvilket som helst af kravene 1 til 17 til anvendelse i en fremgangsmåde til at reducere ekspressionen af et mål-RNA hos et dyr, omfattende indgivelse til dyret af gapmer antisense oligonukleotidet, hvor sekvensen af antisense oligonukleotidet er komplementær med mål-RNA'et.
DK11186203.3T 2006-10-18 2007-10-18 Antisenseforbindelser DK2410054T4 (da)

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Application Number Priority Date Filing Date Title
US85289406P 2006-10-18 2006-10-18
EP07844422.1A EP2092065B2 (en) 2006-10-18 2007-10-18 Antisense compounds

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DK2410054T3 true DK2410054T3 (da) 2017-04-10
DK2410054T4 DK2410054T4 (da) 2020-02-10

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DK07844422.1T DK2092065T4 (da) 2006-10-18 2007-10-18 Antisense-forbindelser
DK11186113.4T DK2410053T4 (da) 2006-10-18 2007-10-18 Antisense-forbindelser

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US (4) US9550988B2 (da)
EP (5) EP2410053B2 (da)
JP (2) JP5665317B2 (da)
AT (1) ATE540118T1 (da)
AU (3) AU2007310989B2 (da)
CA (1) CA2667055C (da)
DK (3) DK2410054T4 (da)
ES (3) ES2526295T5 (da)
WO (1) WO2008049085A1 (da)

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