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DK2972360T3 - Synergistisk forbedring af leveringen af nukleinsyrer via blandede formuleringer - Google Patents

Synergistisk forbedring af leveringen af nukleinsyrer via blandede formuleringer Download PDF

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DK2972360T3
DK2972360T3 DK14721647.7T DK14721647T DK2972360T3 DK 2972360 T3 DK2972360 T3 DK 2972360T3 DK 14721647 T DK14721647 T DK 14721647T DK 2972360 T3 DK2972360 T3 DK 2972360T3
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lipid
mrna
lipid nanoparticle
polynucleotides
cells
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DK14721647.7T
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Frank Derosa
Lianne Smith
Michael Heartlein
Braydon Charles Guild
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Translate Bio 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61K48/0033—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 the non-active part being non-polymeric
    • 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/146—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic macromolecular compounds
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/543—Immunoassay; Biospecific binding assay; Materials therefor with an insoluble carrier for immobilising immunochemicals

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  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Claims (17)

1. Farmaceutisk sammensætning til levering af et messenger-RNA (mRNA) polynukleotid til én eller flere målceller, hvilken sammensætning omfatter et mRNA og en blanding af mindst en første lipidnanopartikel og en anden lipidnanopartikel, hvor den første lipidnanopartikel omfatter mRNA'et; og hvor den første lipidnanopartikel omfatter mindst ét lipid forskellig fra den anden lipidnanopartikel.
2. Farmaceutisk sammensætning ifølge krav 1, hvor den anden lipidnanopartikel omfatter et mRNA der er (i) identisk med mRNA'et i den første lipidnanopartikel eller (ii) ikke identisk med mRNA'et i den første nanopartikel.
3. Farmaceutisk sammensætning ifølge krav 1, hvor det mindst ene forskellige lipid er et kationisk lipid, eventuelt hvor kationlipidet er valgt fra gruppen bestående af C12-200, DOTAP (l,2-dioleyl-3-trimethylammoniumpropan), DODAP (l,2-dioleyl-3-dimethylammoniumpropan), DOTMA (l,2-di-0-octadecenyl-3-trimethylammoniumpropan), DLinDMA, DLin-KC2-DMA, HGT4003 (2-((2,3-Bis((9Z,12Z)-octadeca-9,12-dien-l-yloxy)propyl)disulfanyl)-N,N-dimethylethanamin) og ICE (imidazolcholesterolester).
4. Farmaceutisk sammensætning ifølge krav 1, hvor den første lipidnanopartikel og den anden lipidnanopartikel omfatter: (i) ét eller flere hjælperlipider, eventuelt hvor det ene eller flere hjælperlipider er valgt fra gruppen bestående af DSPC (1,2-distearoyl-sn-glycero-3-phosphocholin), DPPC (l,2-dipalmitoyl-sn-glycero-3-phosphocholin), DOPE (l,2-dioleyl-sn-glycero-3-phosphoethanolamin), DPPE (l,2-dipalmitoyl-sn-glycero-3-phosphoethanolamin), DMPE (1,2-dimyristoyl-sn-glycero-3-phosphoethanolamin), DOPG (,2-dioleoyl-sn-glycero-3-phospho-(l'-rac-glycerol)) og cholesterol, eller (ii) ét eller flere PEG-modificerede lipider, eventuelt hvor det ene eller flere PEG-modificerede lipider omfatter en poly(ethylen)glycolkæde på op til 5kDa i længde kovalent bundet til et lipid omfattende én eller flere alkylkæder af C6-C20 i længde.
5. Farmaceutisk sammensætning ifølge krav 1, hvor den første lipidnanopartikel eller den anden lipidnanopartikel omfatter (i) DLinDMA, CHOL, DOPE og DMG-PEG-2000, (ii) C12-200, CHOL, DOPE og DMG-PEG-2000, (iii) DLinKC2, CHOL, DOPE og DMG-PEG-2000, (iv) ét eller flere lipider valgt fra gruppen bestående af ICE, DSPC, CHOL, DODAP, DOTAP og C8-PEG-2000, (v) DSPC, CHOL, DODAP og C8-PEG-2000, (vi) ICE, DOPE og DMG-PEG-2000, eller (vii) HGT4003, DOPE, CHOL og DMG-PEG-2000.
6. Farmaceutisk sammensætning ifølge et hvilket som helst af de foregående krav, hvor mRNA'et koder for: (i) et enzym, eventuelt hvor enzymet er udskilt fra den ene eller flere målceller; (ii) et protein der er udskilt fra målcellerne; eller (iii) et polypeptid der er afvigende udtrykt af individet, eventuelt hvor polypeptidet er valgt fra gruppen bestående af agalsidase alfa, alpha-L-iduronidase, iduronat-2-sulfatase, N-acetylglucosamin-1-phosphattransferase, beta-glucosidase, galactose-6-sulfatsulfatase, beta-galactosidase, glucocerebrosidase, heparansulfamidase, hyaluronidase, galactocerebrosidase, eller ornithintranscarbamylase (OTC), carbamoyl-phosphatsynthetase 1 (CPS1), argininosuccinatsynthetase (ASSI), argininosuccinatlyase (ASL), og arginase 1 (ARGI); eventuelt hvor mRNA'et er valgt fra SEQ ID NO: 2 eller SEQ ID NO: 3.
7. Farmaceutisk sammensætning ifølge et hvilket som helst af de foregående krav, hvor mRNA'et omfatter en kemisk modifikation, eventuelt hvor (i) den kemiske modifikation gør mRNA'et mere stabil; (ii) den kemiske modifikation omfatter en endeblokeringsmodifikation afen 5'-ikke-translateret region af mRNA'et; (iii) den kemiske modifikation omfatter en endeblokeringsmodifikation afen 3'-ikke-translateret region af mRNA'et; (iv) den kemiske modifikation omfatter inklusionen afen delsekvens afen CMV umiddelbar-tidlig 1 (IEl)-gen til den 5'-ikke-translaterede region af mRNA'et; (v) den kemiske modifikation omfatter inklusionen af en poly A-hale til den 3'-ikke-translaterede region af mRNA'et; (vi) den kemiske modifikation omfatter inklusionen afen Capl-struktur til den 5'-ikke-translaterede region af mRNA'et; eller (vii) den kemiske modifikation omfatter inklusionen af en sekvens der koder for humant væksthormon (hGH) til den 3'-ikke-translaterede region af mRNA'et.
8. Farmaceutisk sammensætning ifølge et hvilket som helst af de foregående krav, hvor forholdet mellem den første lipidnanopartikel og den anden lipidnanopartikel i farmaceutisk sammensætning er ca. 1:1, ca. 2:1, ca. 3:1, eller ca. 4:1.
9. Farmaceutisk sammensætning ifølge et hvilket som helst af kravene 1-8 til anvendelse som medikament.
10. Farmaceutisk sammensætning til anvendelse ifølge krav 9, hvor mRNA'et leveret til målcellerne er valgt fra gruppen bestående af hapatocytter, epitelceller, hæmatopoietiske celler, epitelceller, endothelceller, lungeceller, knogleceller, stamceller, mesenchymalceller, neuralceller, hjerteceller, adipocytter, vaskulære glatmuskelceller, cardiomyocytter, skeletmuskelceller, betaceller, hypofyseceller, synoviallining-celler, æggestokceller, testikelceller, fibroblaster, B-celler, T-celler, reticulocytter, leukocytter, granulocytter og tumorceller, eventuelt hvor administrationen af sammensætningen resulterer i ekspression af mRNA'et i målcellerne.
11. Farmaceutisk sammensætning til anvendelse ifølge krav 9 eller 10, hvor ekspression af mRNA'et efterfulgt af administrationen af farmaceutisk sammensætning til et individ overstiger ekspressionen af mRNA'et administreret med den første lipidnanopartikel men uden den anden lipidnanopartikel, eventuelt hvor ekspressionen af mRNA'et efterfulgt af administrationen af farmaceutisk sammensætning til individet overstiger ekspressionen af mRNA'et administreret med den første lipidnanopartikel men uden den anden lipidnanopartikel ved mindst ca. to-fold, mindst ca. fem-fold eller mindst ca. ti-fold.
12. Farmaceutisk sammensætning til anvendelsen ifølge et hvilket som helst af kravene 9-11, hvor den farmaceutiske sammensætning administreres til et individ ad én eller flere følgende administrationsveje: intravenøst, oralt, rektalt, vaginalt, transmucosalt, sublingvalt, subduralt, nasalt, intramuskulært, subkutant, intramedullær injektion, intrathekalt, intraventriculært, intraperitonealt, intranasalt, intracerebroventrikulært (ICV), ophthalmisk og intraokulært.
13. Sammensætning omfattende et messenger RNA (mRNA) formuleret i mindst to forskellige lipidformuleringer, hvor de mindst to forskellige lipidformuleringer afviger i mindst ét kationisk lipid.
14. Sammensætningen ifølge krav 13, hvor de mindst to forskellige lipidformuleringer yderligere afviger i procentandelen af PEGyleret lipid.
15. Sammensætningen ifølge krav 13 eller 14, hvor de mindst to forskellige lipidformuleringer er til stede i et forhold i området mellem ca. 10:1 og 1:10, eventuelt ved et forhold i området mellem ca. 3:1 og 1:3, eventuelt ved et forhold på ca. 1:1.
16. Sammensætningen ifølge et af kravene 13-15, hvor mRNA'et koder for et enzym.
17. Sammensætningen ifølge et af kravene 13-16 til anvendelse som medikament.
DK14721647.7T 2013-03-15 2014-03-14 Synergistisk forbedring af leveringen af nukleinsyrer via blandede formuleringer DK2972360T3 (da)

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US201361789375P 2013-03-15 2013-03-15
PCT/US2014/028498 WO2014144196A1 (en) 2013-03-15 2014-03-14 Synergistic enhancement of the delivery of nucleic acids via blended formulations

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US (3) US10130649B2 (da)
EP (5) EP3388834B1 (da)
CY (1) CY1120507T1 (da)
DK (2) DK3388834T3 (da)
ES (4) ES3032013T3 (da)
HR (1) HRP20180749T1 (da)
HU (2) HUE039189T2 (da)
LT (1) LT2972360T (da)
NO (1) NO3014045T3 (da)
PL (1) PL4332576T3 (da)
PT (1) PT2972360T (da)
RS (1) RS57316B1 (da)
SI (1) SI2972360T1 (da)
SM (1) SMT201800264T1 (da)
WO (1) WO2014144196A1 (da)

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