DK2548560T3 - Sammensætninger og fremgangsmåder til modulering af SMN2-splejsning - Google Patents

Sammensætninger og fremgangsmåder til modulering af SMN2-splejsning Download PDF

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Publication number
DK2548560T3
DK2548560T3 DK12188625.3T DK12188625T DK2548560T3 DK 2548560 T3 DK2548560 T3 DK 2548560T3 DK 12188625 T DK12188625 T DK 12188625T DK 2548560 T3 DK2548560 T3 DK 2548560T3
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Prior art keywords
isis
antisense
oligonucleotide
smn2
dna
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DK12188625.3T
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English (en)
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Brenda F Baker
Adrian R Krainer
Yimin Hua
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Cold Spring Harbor Lab
Isis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C07H21/02—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 ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Claims (12)

1. Antisense-oligonukleotid targeteret til intron 7 af et nukleinsyremolekyle der koder for SMN2, hvor oligonukleotidet er op til 20 nukleotider i længde og omfatter en 2'-0-methoxyethyl-sukkermodifikation ved hver position, hvor nukleotidsekvensen af oligonukleotidet omfatter SEQ ID NO: 83, SEQ ID NO: 85, SEQ ID NO: 87 eller SEQ ID NO: 89.
2. Oligonukleotidet ifølge krav 1, hvor oligonukleotidet omfatter mindst én 5-methylcytosin, og eventuelt alle cytosinrester er 5-methylcytosiner.
3. Oligonukleotidet ifølge krav 1 eller krav 2, hvor oligonukleotidet er et farmaceutisk acceptabelt salt, og eventuelt et natriumsalt.
4. Oligonukleotidet ifølge et hvilket som helst af de foregående krav, hvor nukleotidsekvensen af dette nukleotid: (a) indeholder ikke mere end 5% baseparrede fejlparringer i forhold til SEQ ID NO: 1; eller (b) indeholder ingen baseparrede fejlparringer i forhold til SEQ ID NO:l.
5. Oligonukleotidet ifølge et hvilket som helst af kravene 1-4, der er 15 nukleotider i længde.
6. Oligonukleotidet ifølge et hvilket som helst af kravene 1-4, der er 18 nukleotider i længde.
7. Oligonukleotidet ifølge et hvilket som helst af kravene 1-4, der er 20 nukleotider i længde.
8. Oligonukleotidet ifølge et hvilket som helst af de foregående krav, omfattende mindst én modificeret internukleosidbinding, og eventuelt omfattende mindst én phosphorthioat-binding.
9. Fremgangsmåde til at fremme inklusion af exon 7 i SMN2-transkripter i en celle, væv eller organ, omfattende at bringe denne celle, væv eller organ i in vitro kontakt med antisense-oligonukleotidet ifølge et hvilket som helst af kravene 1-8.
10. Antisense-oligonukleotid ifølge et hvilket som helst af kravene 1-8 til anvendelse i terapi, og eventuelt til anvendelse i behandlingen af spinal muskulær atrofi.
11. Anvendelse af et antisense-oligonukleotid ifølge et hvilket som helst af kravene 1-8 til fremstillingen af et medikament til behandlingen af spinal muskulær atrofi.
12. Farmaceutisk sammensætning omfattende antisense-oligonukleotidet ifølge et hvilket som helst af kravene 1-8 og et farmaceutisk acceptabelt fortyndingsmiddel eller bærer.
DK12188625.3T 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulering af SMN2-splejsning DK2548560T3 (da)

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US69354205P 2005-06-23 2005-06-23
EP06773838A EP1910395B1 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of SMN2 splicing

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DK06773838.5T DK1910395T3 (da) 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning
DK17185196.7T DK3308788T3 (da) 2005-06-23 2006-06-23 Sammensætninger og fremgangsmåder til modulering af smn2-splejsning

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US (6) US8361977B2 (da)
EP (5) EP3308788B1 (da)
CY (2) CY2017040I2 (da)
DK (3) DK2548560T3 (da)
ES (3) ES2545223T3 (da)
HK (2) HK1181307A1 (da)
HU (2) HUE027486T2 (da)
LT (2) LT3308788T (da)
LU (1) LUC00046I2 (da)
PL (3) PL2548560T3 (da)
PT (3) PT1910395E (da)
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