DK2279254T3 - Hidtil ukendte lipidformuleringer til nukleinsyreindgivelse - Google Patents

Hidtil ukendte lipidformuleringer til nukleinsyreindgivelse Download PDF

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DK2279254T3
DK2279254T3 DK09731866.1T DK09731866T DK2279254T3 DK 2279254 T3 DK2279254 T3 DK 2279254T3 DK 09731866 T DK09731866 T DK 09731866T DK 2279254 T3 DK2279254 T3 DK 2279254T3
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mol
lipid
nucleic acid
particle
sirna
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Ian Maclachlan
Edward Yaworski
Kieu Lam
Lloyd B Jeffs
Lorne R Palmer
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Protiva Biotherapeutics Inc
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
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    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J9/00—Normal steroids containing carbon, hydrogen, halogen or oxygen substituted in position 17 beta by a chain of more than two carbon atoms, e.g. cholane, cholestane, coprostane
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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
    • 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
    • C12N15/113—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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    • 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
    • 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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    • 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
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Claims (15)

1. Nukleinsyre-lipidpartikel, der omfatter: (a) en nukleinsyre; (b) et kationisk lipid, der omfatter fra 50 mol-% til 65 mol-% af det totale lipid til stede i partiklen; (c) et ikke-kationisk lipid, der omfatter op til 49,5 mol-% af det totale lipid til stede i partiklen og, der omfatter en blanding af et phospholipid og cholesterol eller et derivat deraf, hvor cholesterolet eller derivatet deraf omfatter fra 30 mol-% til 40 mol-% af det totale lipid til stede i partiklen; og (d) et konjugeret lipid, der hæmmer aggregering af partikler, der omfatter fra 0,5 mol-% til 2 mol-% af det totale lipid til stede i partiklen.
2. Nukleinsyre-lipidpartikel ifølge krav 1, hvor nukleinsyren omfatter et mindre interferens-RNA (siRNA).
3. Nukleinsyre-lipidpartikel ifølge krav 2, hvor: (a) siRNA’et omfatter fra ca. 15 til ca. 60 nukleotider; eller (b) siRNA’et omfatter mindst ét modificeret nukleotid; eller (c) siRNA’et omfatter mindst ét 2’-0-methyl- (2’OMe) nukleotid; eller (d) siRNA’et er ca. 19 til ca. 25 basepar langt; eller (e) siRNA’et omfatter 3’-overhæng.
4. Nukleinsyre-lipidpartikel ifølge krav 1, hvor: (a) det kationiske lipid omfatter l,2-dilinoleyloxy-N,N-dimethylaminopropan (DLinDMA), l,2-dilinolenyloxy-N,N-dimethylaminopropan (DLenDMA), eller en blanding deraf; eller (b) det kationiske lipid omfatter 2,2-dilinoleyl-4-(2-dimethylaminoethyl)-[l,3]-dioxolan (DLin-K-C2-DMA); eller (c) det kationiske lipid omfatter fra 50 mol-% til 60 mol-% af det totale lipid til stede i partiklen; eller (d) det kationiske lipid omfatter fra 52 mol-% til 62 mol-% af det totale lipid til stede i partiklen.
5. Nukleinsyre-lipidpartikel ifølge krav 1, hvor: (a) det konjugerede lipid, der hæmmer aggregering af partikler, omfatter et polyethylenglycol (PEG)-lipidkonjugat; eller (b) det konjugerede lipid, der hæmmer aggregering af partikler, omfatter fra 1 mol-% til 2 mol-% af det totale lipid til stede i partiklen.
6. Nukleinsyre-lipidpartikel ifølge krav 1, hvor: (a) nukleinsyren i nukleinsyre-lipidpartiklen i det væsentlige ikke er nedbrudt efter inkubation af partiklen i serum ved 37 °C i 30 minutter; eller (b) nukleinsyren er fuldt indkapslet i nukleinsyre-lipidpartiklen; eller (c) nukleinsyre-lipidpartiklen har et masseforhold mellem lipid og nukleinsyre på fra ca. 5 til ca. 15; eller (d) nukleinsyre-lipidpartiklen har en mediandiameter på fra ca. 40 nm til ca. 150 nm.
7. Nukleinsyre-lipidpartikel ifølge krav 1, hvor: (a) phospholipidet omfatter dipalmitoylphosphatidylcholin (DPPC), distearoylphosphatidylcholin (DSPC), eller en blanding deraf; eller (b) phospholipidet omfatter fra 4 mol-% til 10 mol-% af det totale lipid til stede i partiklen.
8. Nukleinsyre-lipidpartikel ifølge krav 1, hvor: (a) cholesterolet eller derivatet deraf omfatter fra 30 mol-% til 35 mol-% af det totale lipid til stede i partiklen; eller (b) cholesterolet eller derivatet deraf omfatter fra 32 mol-% til 36 mol-% af det totale lipid til stede i partiklen og phospholipidet omfatter fra 3 mol-% til 15 mol-% af det totale lipid til stede i partiklen.
9. Nukleinsyre-lipidpartikel ifølge krav 5(a), hvor PEG-lipidkonjugatet omfatter et PEG-diacylglycerol- (PEGDAG) konjugat, et PEG-dialkyloxypropyl- (PEG-DAA) konjugat, eller en blanding deraf; eventuelt hvor PEGDAA-konjugatet omfatter et PEG-dimyristyloxypropyl- (PEG-DMA) konjugat, et PEG-distearyloxypropyl- (PEG-DSA) konjugat, eller en blanding deraf; fortrinsvis hvor PEG i PEG-DAA-konjugatet har en middelmolekylvægt på ca. 2.000 dalton.
10. Nukleinsyre-lipidpartikel ifølge krav 5(a), hvor nukleinsyren er et siRNA og nukleinsyre-lipidpartiklen omfatter ca. 57,1 mol-% kationisk lipid, ca. 7,1 mol-% phospholipid, ca. 34,3 mol-% cholesterol eller et derivat deraf, og ca. 1,4 mol-% PEG-lipidkonjugat.
11. Farmaceutisk sammensætning, der omfatter en nukleinsyre-lipidpartikel ifølge et hvilket som helst af de foregående krav og en farmaceutisk acceptabel bærer.
12. Fremgangsmåde til indføring af en nukleinsyre i en celle, hvilken fremgangsmåde omfatter: etablering af kontakt mellem cellen in vitro med en nukleinsyre-lipidpartikel ifølge et hvilket som helst af kravene 1 til 10, eventuelt hvor cellen er en pattedyrecelle.
13. Nukleinsyre-lipidpartikel ifølge et hvilket som helst af kravene 1 til 10 til anvendelse i en fremgangsmåde til indgivelse in vivo af en nukleinsyre, hvilken fremgangsmåde omfatter administration af nukleinsyre-lipidpartiklen til et pattedyr.
14. Nukleinsyre-lipidpartikel til anvendelse ifølge krav 13, hvor administrationen er udvalgt fra gruppen bestående af oral, intranasal, intravenøs, intraperitoneal, intramuskulær, intra-artikulær, intralæsional, intratrakeal, subkutan og intradermal.
15. Nukleinsyre-lipidpartikel ifølge et hvilket som helst af kravene 1 til 10 til anvendelse i en fremgangsmåde til behandling af en sygdom eller forstyrrelse hos et pattedyr med behov derfor, hvilken fremgangsmåde omfatter administration af en terapeutisk effektiv mængde af nukleinsyre-lipidpartiklen til pattedyret, hvor sygdommen eller forstyrrelsen eventuelt er udvalgt fra gruppen bestående af en virusinfektion, en leversygdom eller -forstyrrelse og cancer.
DK09731866.1T 2008-04-15 2009-04-15 Hidtil ukendte lipidformuleringer til nukleinsyreindgivelse DK2279254T3 (da)

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US4522808P 2008-04-15 2008-04-15
PCT/CA2009/000496 WO2009127060A1 (en) 2008-04-15 2009-04-15 Novel lipid formulations for nucleic acid delivery

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US (11) US8058069B2 (da)
EP (1) EP2279254B1 (da)
JP (1) JP5475753B2 (da)
CN (1) CN102119217B (da)
AU (1) AU2009238175C1 (da)
CA (1) CA2721333C (da)
DK (1) DK2279254T3 (da)
ES (1) ES2638448T3 (da)
HU (1) HUE034483T2 (da)
IL (1) IL208744A (da)
NZ (1) NZ588583A (da)
PL (1) PL2279254T3 (da)
PT (1) PT2279254T (da)
WO (1) WO2009127060A1 (da)

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