[go: up one dir, main page]

DK2768958T3 - Kationiske aminlipider og anvendelser deraf - Google Patents

Kationiske aminlipider og anvendelser deraf Download PDF

Info

Publication number
DK2768958T3
DK2768958T3 DK12841768.0T DK12841768T DK2768958T3 DK 2768958 T3 DK2768958 T3 DK 2768958T3 DK 12841768 T DK12841768 T DK 12841768T DK 2768958 T3 DK2768958 T3 DK 2768958T3
Authority
DK
Denmark
Prior art keywords
aminlipids
cationic
applications
cationic aminlipids
Prior art date
Application number
DK12841768.0T
Other languages
English (en)
Inventor
Bob Dale Brown
Sujit Kumar Basu
David Schwartz
Allister Fraser
Original Assignee
Dicerna Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Application granted granted Critical
Publication of DK2768958T3 publication Critical patent/DK2768958T3/da

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/06—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C211/00—Compounds containing amino groups bound to a carbon skeleton
    • C07C211/01—Compounds containing amino groups bound to a carbon skeleton having amino groups bound to acyclic carbon atoms
    • C07C211/20—Compounds containing amino groups bound to a carbon skeleton having amino groups bound to acyclic carbon atoms of an acyclic unsaturated carbon skeleton
    • C07C211/21—Monoamines
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/06—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D211/56—Nitrogen atoms
    • C07D211/58—Nitrogen atoms attached in position 4
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D213/00—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members
    • C07D213/02—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members
    • C07D213/04—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D213/60—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D213/72—Nitrogen atoms
    • C07D213/74—Amino or imino radicals substituted by hydrocarbon or substituted hydrocarbon radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D233/00—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings
    • C07D233/54—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members
    • C07D233/56—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms
    • C07D233/61—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms with hydrocarbon radicals, substituted by nitrogen atoms not forming part of a nitro radical, attached to ring nitrogen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D233/00—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings
    • C07D233/54—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members
    • C07D233/64—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with substituted hydrocarbon radicals attached to ring carbon atoms, e.g. histidine
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
    • C07D239/32—One oxygen, sulfur or nitrogen atom
    • C07D239/42—One nitrogen atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D241/00—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings
    • C07D241/02—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings
    • C07D241/04—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D243/00—Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms
    • C07D243/06—Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms having the nitrogen atoms in positions 1 and 4
    • C07D243/08—Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms having the nitrogen atoms in positions 1 and 4 not condensed with other rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/02—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/02—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements
    • C07D295/027—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements containing only one hetero ring
    • C07D295/03—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements containing only one hetero ring with the ring nitrogen atoms directly attached to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/12—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
    • C07D295/125—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/13—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/14—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C07D295/145—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/15—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings
    • C07D401/04—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/04—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • 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
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Pyridine Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Hydrogenated Pyridines (AREA)
DK12841768.0T 2011-10-18 2012-10-18 Kationiske aminlipider og anvendelser deraf DK2768958T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161548598P 2011-10-18 2011-10-18
PCT/US2012/060875 WO2013059496A1 (en) 2011-10-18 2012-10-18 Amine cationic lipids and uses thereof

Publications (1)

Publication Number Publication Date
DK2768958T3 true DK2768958T3 (da) 2019-09-16

Family

ID=48141352

Family Applications (2)

Application Number Title Priority Date Filing Date
DK12841768.0T DK2768958T3 (da) 2011-10-18 2012-10-18 Kationiske aminlipider og anvendelser deraf
DK19191168.4T DK3597644T3 (da) 2011-10-18 2012-10-18 Kationiske aminlipider og anvendelser deraf

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK19191168.4T DK3597644T3 (da) 2011-10-18 2012-10-18 Kationiske aminlipider og anvendelser deraf

Country Status (13)

Country Link
US (4) US9549983B2 (da)
EP (3) EP3960726A1 (da)
JP (2) JP6294229B2 (da)
KR (1) KR102011048B1 (da)
CN (2) CN107266391B (da)
AU (1) AU2012325997C1 (da)
CA (1) CA2852917C (da)
DK (2) DK2768958T3 (da)
ES (2) ES2745373T3 (da)
HU (1) HUE057604T2 (da)
PL (1) PL3597644T3 (da)
PT (1) PT3597644T (da)
WO (1) WO2013059496A1 (da)

Families Citing this family (141)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2594368T3 (es) 2005-12-08 2016-12-19 Insmed Incorporated Composiciones a base de lípidos de antiinfecciosos para tratar infecciones pulmonares
US9119783B2 (en) 2007-05-07 2015-09-01 Insmed Incorporated Method of treating pulmonary disorders with liposomal amikacin formulations
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP3590949B1 (en) 2010-10-01 2022-05-18 ModernaTX, Inc. Ribonucleic acids containing n1-methyl-pseudouracils and uses thereof
WO2012083005A2 (en) 2010-12-15 2012-06-21 Miragen Therapeutics Microrna inhibitors comprising locked nucleotides
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
RS62993B1 (sr) 2011-10-03 2022-03-31 Modernatx Inc Modifikovani nukleozidi, nukleotidi, i nukleinske kiseline, i njihove upotrebe
CA2850223A1 (en) 2011-10-06 2013-04-11 Eva Van Rooij Control of whole body energy homeostasis by microrna regulation
DK2768958T3 (da) * 2011-10-18 2019-09-16 Dicerna Pharmaceuticals Inc Kationiske aminlipider og anvendelser deraf
CA3165769A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
AU2012352180A1 (en) 2011-12-16 2014-07-31 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
JP2015513912A (ja) 2012-04-02 2015-05-18 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． タンパク質の産生のための修飾ポリヌクレオチド
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP3354734B1 (en) 2012-06-21 2020-06-10 Miragen Therapeutics, Inc. Oligonucleotide-based inhibitors comprising locked nucleic acid motif
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
ES2743039T3 (es) 2012-11-29 2020-02-18 Insmed Inc Formulaciones de vancomicina estabilizada
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9567641B2 (en) 2013-07-03 2017-02-14 Samsung Electronics Co., Ltd. Combination therapy for the treatment of cancer using an anti-C-met antibody
MX2016000021A (es) 2013-07-05 2017-04-06 Bioneer Corp Sirna virus especifico del dengue, estructura arn oligo helicoidal, y composicion para la supresion de proliferacion del virus del dengue que comprende la estructura arn.
CN110592083A (zh) * 2013-07-05 2019-12-20 柏业公司 呼吸疾病相关基因特异性siRNA、含有siRNA的双螺旋寡RNA结构及其用途
MX378462B (es) 2013-07-05 2025-03-10 Bioneer Corp Estructura de nanoparticula del tipo oligonuclèotido mejorada que tiene alta eficiencia y mètodo para preparar la misma.
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
CN105980401A (zh) 2013-10-03 2016-09-28 现代治疗公司 编码低密度脂蛋白受体的多核苷酸
CN105916839B (zh) * 2014-01-09 2018-09-18 卫材R&D管理有限公司 阳离子性脂质
WO2015152693A2 (ko) 2014-04-04 2015-10-08 (주)바이오니아 신규 이중 나선 올리고 rna 및 이를 포함하는 섬유증 또는 호흡기 질환의 예방 또는 치료용 약학조성물
SG11201608798YA (en) 2014-04-23 2016-11-29 Modernatx Inc Nucleic acid vaccines
PT3142643T (pt) 2014-05-15 2019-10-28 Insmed Inc Métodos para o tratamento de infeções mico bacterianas pulmonares não tuberculares
CN105085437B (zh) * 2014-05-23 2018-07-03 上海交通大学 3-(1-叔丁氧羰酰哌嗪-4-yl)丙酸的两亲性衍生物及其用途
PT3766916T (pt) 2014-06-25 2022-11-28 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
EP3169693B1 (en) 2014-07-16 2022-03-09 ModernaTX, Inc. Chimeric polynucleotides
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
US9885042B2 (en) 2015-01-20 2018-02-06 MiRagen Therapeutics, Inc. miR-92 inhibitors and uses thereof
JP2018526321A (ja) 2015-04-27 2018-09-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 適応免疫応答を誘導するためのヌクレオシド修飾ｒｎａ
CN104873976B (zh) * 2015-05-08 2016-04-20 厦门成坤生物技术有限公司 一类阳离子类脂化合物的应用
CN107922364B (zh) * 2015-06-29 2021-12-31 爱康泰生治疗公司 用于递送核酸的脂质和脂质纳米颗粒制剂
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
WO2017015463A2 (en) 2015-07-21 2017-01-26 Modernatx, Inc. Infectious disease vaccines
WO2017031232A1 (en) 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
JP2018531916A (ja) * 2015-09-09 2018-11-01 ウォーレン シー． ラウ， 新規のｆｙｎキナーゼ阻害剤の方法、組成物、及び使用
EP3350157B1 (en) 2015-09-17 2022-01-05 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
CN105205747A (zh) * 2015-10-08 2015-12-30 张姬娟 送餐信息的处理方法
EA201891000A1 (ru) 2015-10-22 2018-12-28 МОДЕРНАТиЭкс, ИНК. Вакцина против респираторно-синцитиального вируса
HRP20220872T1 (hr) 2015-10-22 2022-12-23 Modernatx, Inc. Cjepiva protiv respiratornih virusa
CA3003103A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Broad spectrum influenza virus vaccine
JP6925688B2 (ja) 2015-10-22 2021-08-25 モデルナティーエックス， インコーポレイテッド 水痘帯状疱疹ウイルス（ｖｚｖ）のための核酸ワクチン
MA46316A (fr) 2015-10-22 2021-03-24 Modernatx Inc Vaccin contre le cytomégalovirus humain
EP3364950A4 (en) 2015-10-22 2019-10-23 ModernaTX, Inc. VACCINES AGAINST TROPICAL DISEASES
MA46024A (fr) 2015-10-22 2019-07-03 Modernatx Inc Vaccin contre le virus de l'herpès simplex
US20180289792A1 (en) 2015-10-22 2018-10-11 ModernaTX. Inc. Sexually transmitted disease vaccines
WO2017075038A1 (en) 2015-10-26 2017-05-04 Rana Therapeutics, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
PT3368507T (pt) 2015-10-28 2023-02-07 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
CA3003151A1 (en) * 2015-11-18 2017-05-26 Insmed Incorporated Compositions and methods for treating bacterial infections
AU2016366978B2 (en) 2015-12-10 2022-07-28 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
RU2609107C1 (ru) * 2015-12-16 2017-01-30 Федеральное Государственное Бюджетное Учреждение Науки Институт Молекулярной Биологии Им. В.А. Энгельгардта Российской Академии Наук (Имб Ран) Подавление экспрессии гена C-KIT в клетках нейробластом и лентивирусные конструкции, направляющие синтез shPHK, специфичных в отношении данного гена
JP7114465B2 (ja) 2015-12-22 2022-08-08 モデルナティエックス インコーポレイテッド 薬剤の細胞内送達のための化合物および組成物
HRP20220525T1 (hr) 2015-12-23 2022-05-27 Modernatx, Inc. Postupci uporabe polinukleotida koji kodiraju ligand ox40
MA43587A (fr) 2016-01-10 2018-11-14 Modernatx Inc Arnm thérapeutiques codant pour des anticorps anti-ctla-4
WO2017174417A1 (en) * 2016-04-08 2017-10-12 Rhodia Operations Diamines surfactants suitable as emulsifier
CN109640962B (zh) 2016-05-18 2022-07-19 摩登纳特斯有限公司 编码松弛素的多核苷酸
RU2740921C2 (ru) 2016-06-24 2021-01-21 Эйсай Ар Энд Ди Менеджмент Ко., Лтд. Катионный липид
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US11186538B2 (en) 2016-11-08 2021-11-30 Rhodia Operations Twin tail amine compounds and their zwitterionic derivatives
EP3538067A1 (en) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
CN110167587A (zh) 2016-11-11 2019-08-23 摩登纳特斯有限公司 流感疫苗
EP3548005A4 (en) 2016-11-29 2020-06-17 Puretech Health LLC Exosomes for delivery of therapeutic agents
WO2018107088A2 (en) 2016-12-08 2018-06-14 Modernatx, Inc. Respiratory virus nucleic acid vaccines
EP3568155A4 (en) 2017-01-11 2020-09-23 The Trustees Of The University Of Pennsylvania RNA MODIFIED BY NUCLEOSIDE TO INDUCE AN IMMUNE RESPONSE AGAINST ZIKA VIRUS
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
EP3596042B1 (en) 2017-03-15 2022-01-12 Modernatx, Inc. Crystal forms of amino lipids
WO2018170256A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Herpes simplex virus vaccine
SMT202300097T1 (it) 2017-03-15 2023-05-12 Modernatx Inc Composto e composizioni per il rilascio intracellulare di agenti terapeutici
MA52262A (fr) 2017-03-15 2020-02-19 Modernatx Inc Vaccin à large spectre contre le virus de la grippe
JP7332478B2 (ja) 2017-03-15 2023-08-23 モデルナティエックス インコーポレイテッド 脂質ナノ粒子製剤
MA47787A (fr) 2017-03-15 2020-01-22 Modernatx Inc Vaccin contre le virus respiratoire syncytial
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
JP7297676B2 (ja) 2017-04-28 2023-06-26 アクイタス セラピューティクス インコーポレイテッド 新規なカルボニル脂質、および核酸を送達するための脂質ナノ粒子製剤
EP3638678B1 (en) 2017-06-14 2025-12-03 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
CA3071968A1 (en) 2017-08-04 2019-02-07 Kyowa Kirin Co., Ltd. Nucleic acid-containing lipid nanoparticle
WO2019036008A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036000A1 (en) * 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11744801B2 (en) 2017-08-31 2023-09-05 Modernatx, Inc. Methods of making lipid nanoparticles
EP3681514A4 (en) 2017-09-14 2021-07-14 ModernaTX, Inc. RNA VACZINE AGAINST ZIKA VIRUS
CN111491914B (zh) * 2017-12-20 2024-04-26 罗地亚经营管理公司 新的阳离子季铵化合物和包含其的组合物及其制造方法
JP7164547B2 (ja) 2017-12-27 2022-11-01 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
MA54676A (fr) 2018-01-29 2021-11-17 Modernatx Inc Vaccins à base d'arn contre le vrs
CA3089897A1 (en) * 2018-02-21 2019-08-29 Rhodia Operations Gelling fluids and related methods of use
CN110283223B (zh) * 2018-03-19 2022-01-04 广州市锐博生物科技有限公司 一种阳离子脂质分子及其在核酸递送中的应用
US11571386B2 (en) 2018-03-30 2023-02-07 Insmed Incorporated Methods for continuous manufacture of liposomal drug products
KR20210024451A (ko) 2018-05-02 2021-03-05 인스메드 인코포레이티드 리포솜 약물 제형을 제조하는 방법
CN112533909B (zh) * 2018-05-30 2024-11-15 川斯勒佰尔公司 维生素阳离子脂质
JP7178409B2 (ja) 2018-06-08 2022-11-25 富士フイルム株式会社 化合物またはその塩および脂質粒子
US12357580B2 (en) 2018-06-19 2025-07-15 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
JP2021535226A (ja) * 2018-09-04 2021-12-16 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム 核酸を臓器特異的送達するための組成物および方法
GB2617430B (en) 2018-09-04 2023-12-27 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
CA3113025A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
CN112996854B (zh) 2018-09-19 2024-08-30 摩登纳特斯有限公司 高纯度peg脂质和其用途
EP3852728B1 (en) 2018-09-20 2024-09-18 ModernaTX, Inc. Preparation of lipid nanoparticles and methods of administration thereof
CA3113449A1 (en) 2018-09-21 2020-03-26 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
PT3908568T (pt) 2019-01-11 2024-09-30 Acuitas Therapeutics Inc Lípidos para a administração de agentes ativos por nanopartículas lipídicas
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
US12070495B2 (en) 2019-03-15 2024-08-27 Modernatx, Inc. HIV RNA vaccines
PH12021500045A1 (en) * 2019-04-25 2023-01-23 Intellia Therapeutics Inc Ionizable amine lipids and lipid nanoparticles
KR102660546B1 (ko) 2019-06-07 2024-04-24 후지필름 가부시키가이샤 지질 조성물
BR112022004759A2 (pt) 2019-09-19 2022-06-21 Modernatx Inc Composições e compostos lipídicos com cauda ramificada para entrega intracelular de agentes terapêuticos
WO2021141944A1 (en) * 2020-01-07 2021-07-15 Translate Bio Ma, Inc. Formulations for delivery of oligonucleotides to lung cells
CN115484932B (zh) 2020-03-04 2024-12-10 维乎医疗有限公司 用于靶向rna递送的组合物和方法
WO2022016070A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
US20230295097A1 (en) * 2020-07-17 2023-09-21 Sanofi Pasteur Lipidic compounds comprising at least one terminal radical of formula -nh-cx-a or -nh-cx-nh-a, compositions containing them and uses thereof
JP2023546908A (ja) 2020-10-14 2023-11-08 ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド イオン化可能脂質ならびにその製造及び使用の方法
CN113185421B (zh) * 2020-11-27 2022-01-25 广州市锐博生物科技有限公司 脂质化合物及其组合物
CN116685356A (zh) * 2020-12-02 2023-09-01 默沙东有限责任公司 含有单酯阳离子脂质的脂质纳米颗粒组合物
CN114904002B (zh) * 2021-02-09 2024-04-26 广州立得生物医药科技有限公司 可电离的阳离子脂质类似物材料及其在作为药物递送载体中的应用
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
CN112979483B (zh) * 2021-05-14 2021-08-06 苏州艾博生物科技有限公司 一种阳离子脂质化合物、包含其的组合物及应用
CA3215963A1 (en) * 2021-05-28 2022-12-01 Yaoxin LIN Lipid compound and use thereof in delivery of nucleic acid
CN117897495A (zh) * 2021-06-17 2024-04-16 加利福尼亚大学董事会 用于递送抗真菌核酸的囊泡制剂
WO2023114943A2 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
JP2025508467A (ja) 2022-02-24 2025-03-26 アイオー バイオテック エーピーエス 癌治療のヌクレオチド送達
KR102465349B1 (ko) * 2022-02-25 2022-11-11 주식회사 테르나테라퓨틱스 신규 이온화 지질 및 이를 포함하는 지질 나노입자
EP4486728A1 (en) * 2022-03-02 2025-01-08 Barcode Nanotech Ltd. Ionizable lipids and compositions comprising same
CN116813493A (zh) * 2022-03-21 2023-09-29 苏州科锐迈德生物医药科技有限公司 一种脂质化合物及基于其的脂质载体、核酸脂质纳米粒组合物和药物制剂
US20250382640A1 (en) 2022-06-29 2025-12-18 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids
CN115232128B (zh) * 2022-08-15 2024-03-29 中国科学技术大学 一种咪唑并吡啶基脂质化合物及其制备方法与应用
WO2024177424A1 (ko) * 2023-02-24 2024-08-29 에스티팜 주식회사 신규 이온화 가능한 지질 및 이를 포함하는 지질나노입자
CN120858086A (zh) * 2023-03-13 2025-10-28 富士胶片株式会社 化合物或其盐、脂质组合物、药物组合物及递送载体
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
EP4520345A1 (en) 2023-09-06 2025-03-12 Myneo Nv Product
CN117482066B (zh) * 2023-11-02 2024-08-13 深圳市易瑞生物技术股份有限公司 脂质组合物和用于脂质组合物的化合物
WO2025147545A1 (en) 2024-01-03 2025-07-10 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids and related methods and uses
WO2025157978A1 (en) * 2024-01-26 2025-07-31 Merck Patent Gmbh Ionizable lipids for use in lipid nanoparticles
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3288774A (en) * 1963-12-03 1966-11-29 Universal Oil Prod Co Reaction product of substituted-aminoalkyl alkanolamine and polycarboxylic acid or the like
SE467861B (sv) 1987-12-18 1992-09-28 Kabi Pharmacia Ab Emulsion foer parenteral administrering
US5157309A (en) 1990-09-13 1992-10-20 Motorola Inc. Cold-cathode field emission device employing a current source means
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
FR2694559B1 (fr) * 1992-08-06 1994-10-28 Atta Nouveaux dérivés amphiphiles d'aminoacides ou de peptides, leur procédé de préparation et leur utilisation dans des préparations à usage biomédical.
EP1602725A3 (en) 1993-09-02 2006-03-29 Sirna Therpeutics, Inc. Abasic-nucleotide containing enzymatic nucleic acid
US5534499A (en) 1994-05-19 1996-07-09 The University Of British Columbia Lipophilic drug derivatives for use in liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
DE19521412A1 (de) * 1995-06-14 1996-12-19 Boehringer Mannheim Gmbh Neue kationische und polykationische Amphiphile, diese enthaltende Reagenzien und deren Verwendung
CA2227373A1 (en) * 1995-07-21 1997-02-06 Promega Biosciences, Inc. Novel amide-based cationic lipids
US5744335A (en) * 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
DE19631189A1 (de) * 1996-08-02 1998-02-05 Max Delbrueck Centrum Neuartige kationische Amphiphile für den liposomalen Gentransfer
TW589189B (en) 1997-08-04 2004-06-01 Scras Kit containing at least one double-stranded RNA combined with at least one anti-viral agent for therapeutic use in the treatment of a viral disease, notably of viral hepatitis
US6121457A (en) * 1997-11-14 2000-09-19 Megabios Corporation Compositions and methods using novel substituted imidazolium lipids
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
GB9827152D0 (en) 1998-07-03 1999-02-03 Devgen Nv Characterisation of gene function using double stranded rna inhibition
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
JP2003516124A (ja) 1999-10-15 2003-05-13 ユニバーシティー オブ マサチューセッツ 標的とした遺伝的干渉の手段としてのｒｎａ干渉経路遺伝子
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
CA2420098A1 (en) * 2000-08-19 2002-02-28 University Of Florida Functionalized polymers
DE60130583T3 (de) 2000-12-01 2018-03-22 Europäisches Laboratorium für Molekularbiologie Kleine rns moleküle, die rns-interferenz vermitteln
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
CN100543137C (zh) 2002-08-05 2009-09-23 阿图根股份公司 其它新形式的干扰rna分子
US20050277610A1 (en) 2004-03-15 2005-12-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US7303881B2 (en) 2004-04-30 2007-12-04 Pds Biotechnology Corporation Antigen delivery compositions and methods of use
WO2006118327A1 (ja) * 2005-04-27 2006-11-09 Oxygenix Co Ltd カチオン性アミノ酸型脂質
US7915230B2 (en) * 2005-05-17 2011-03-29 Molecular Transfer, Inc. Reagents for transfection of eukaryotic cells
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
WO2007095387A2 (en) 2006-02-17 2007-08-23 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
EP1844772A1 (en) * 2006-04-06 2007-10-17 Polyplus-Transfection SA Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications
NZ593080A (en) * 2006-05-05 2012-12-21 Molecular Transfer Inc Novel reagents for transfection of eukaryotic cells
US20090285881A1 (en) * 2008-04-16 2009-11-19 Abbott Laboratories Cationic lipids and uses thereof
US20100055168A1 (en) * 2008-04-16 2010-03-04 Abbott Laboratories Cationic lipids and uses thereof
WO2009129385A1 (en) * 2008-04-16 2009-10-22 Abbott Laboratories Cationic lipids and uses thereof
TW201019963A (en) * 2008-09-10 2010-06-01 Abbott Lab Polyethylene glycol lipid conjugates and uses thereof
US20100099738A1 (en) * 2008-09-10 2010-04-22 Abbott Laboratories Polyethylene glycol lipid conjugates and uses thereof
PT2341943T (pt) 2008-09-22 2019-02-06 Dicerna Pharmaceuticals Inc Composições e métodos para a inibição específica de expressão génica por dsrna que possui modificações
CA2743136C (en) * 2008-11-10 2019-02-26 Muthiah Manoharan Novel lipids and compositions for the delivery of therapeutics
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
EP3199165B1 (en) 2009-04-03 2022-06-08 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of kras by asymmetric double-stranded rna
WO2010115202A2 (en) 2009-04-03 2010-10-07 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of kras by blunt ended double-stranded rna
EP2756845B1 (en) 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
JP2012528596A (ja) 2009-06-03 2012-11-15 ダイセルナ ファーマシューティカルズ， インコーポレイテッド ペプチドダイサー基質剤およびその特異的な遺伝子発現阻害のための方法
WO2010141933A1 (en) 2009-06-05 2010-12-09 Dicerna Pharmaceuticals, Inc. Specific inhibition of gene expression by nucleic acid containing a dicer substrate
US20100310623A1 (en) 2009-06-05 2010-12-09 Laurencin Cato T Synergetic functionalized spiral-in-tubular bone scaffolds
EP2467357B1 (en) * 2009-08-20 2016-03-30 Sirna Therapeutics, Inc. Novel cationic lipids with various head groups for oligonucleotide delivery
WO2011072292A2 (en) 2009-12-11 2011-06-16 Dicerna Pharmaceuticals, Inc. Phase changing formulations of rna and rna derivatives
AU2010330814B2 (en) * 2009-12-18 2017-01-12 Acuitas Therapeutics Inc. Methods and compositions for delivery of nucleic acids
EP2525781A1 (en) * 2010-01-22 2012-11-28 Schering Corporation Novel cationic lipids for oligonucleotide delivery
WO2011109294A1 (en) 2010-03-01 2011-09-09 Dicerna Pharmaceuticals, Inc. Lipid delivery formulations
EP2569276B1 (en) * 2010-05-12 2021-02-24 Arbutus Biopharma Corporation Novel cationic lipids and methods of use thereof
WO2012000104A1 (en) * 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
DK2768958T3 (da) * 2011-10-18 2019-09-16 Dicerna Pharmaceuticals Inc Kationiske aminlipider og anvendelser deraf

Also Published As

Publication number Publication date
AU2012325997A2 (en) 2015-10-15
ES2897565T8 (es) 2022-03-10
CN103987847B (zh) 2017-06-16
EP2768958B1 (en) 2019-08-14
CA2852917A1 (en) 2013-04-25
CA2852917C (en) 2020-07-07
EP3960726A9 (en) 2023-04-26
US20140371293A1 (en) 2014-12-18
EP3597644B1 (en) 2021-09-29
US11059807B2 (en) 2021-07-13
AU2012325997B2 (en) 2018-01-04
KR102011048B1 (ko) 2019-08-14
US20190084965A1 (en) 2019-03-21
US10144725B2 (en) 2018-12-04
JP6294229B2 (ja) 2018-03-14
EP2768958A4 (en) 2015-05-27
ES2745373T3 (es) 2020-03-02
US20170137409A1 (en) 2017-05-18
KR20140098082A (ko) 2014-08-07
AU2012325997A1 (en) 2014-06-05
AU2012325997B9 (en) 2018-06-28
HUE057604T2 (hu) 2022-06-28
PT3597644T (pt) 2021-11-03
CN103987847A (zh) 2014-08-13
EP3597644A1 (en) 2020-01-22
ES2897565T3 (es) 2022-03-01
JP6685338B2 (ja) 2020-04-22
HK1245271A1 (zh) 2018-08-24
EP2768958A1 (en) 2014-08-27
US9549983B2 (en) 2017-01-24
WO2013059496A1 (en) 2013-04-25
AU2012325997C1 (en) 2018-07-05
CN107266391A (zh) 2017-10-20
JP2015501309A (ja) 2015-01-15
US20210292303A1 (en) 2021-09-23
DK3597644T3 (da) 2021-11-08
EP3960726A1 (en) 2022-03-02
PL3597644T3 (pl) 2022-01-17
CN107266391B (zh) 2020-04-17
JP2018115164A (ja) 2018-07-26

Similar Documents

Publication Publication Date Title
DK2768958T3 (da) Kationiske aminlipider og anvendelser deraf
IL269045A (en) Aptamers and uses thereof
DK2920167T3 (da) Hydrazinyl-indol-forbindelser og konjugater
DK2914291T3 (da) Anti-komplement-c1s-antistoffer og anvendelser deraf
DK2850202T3 (da) Fremgangsmåder og grupper
BR112014009050A2 (pt) pirrolbenzodiazepinas e conjugados destas
DK3346008T3 (da) Rekombinante mikroorganismer og anvendelser deraf
DK3415157T3 (da) N-acyldipeptidderivater og anvendelser deraf
DK2906684T3 (da) T-celle-modificerende forbindelser og anvendelser deraf
DK2794654T3 (da) Anti-phf-tau-antistoffer og deres anvendelser
DK3608325T3 (da) Polycykliske-carbamoylpyridonforbindelser og deres farmaceutiske anvendelse
HUE040323T2 (hu) PRMT5-inhibitorok és alkalmazásaik
DK2920197T3 (da) Prolinlåste sammenhæftede peptider og anvendelser deraf
EP2661839A4 (en) SAFE PORTABLE ELEMENT
DK3339323T3 (da) Anti-n3pglu-amyloid-beta-peptid-antistoffer og anvendelser deraf
DK3453723T3 (da) Anti-ASIC1-antistoffer og anvendelser deraf
DK2704744T3 (da) Komplementfaktor-b-analoger og anvendelser deraf
DK2917181T3 (da) Heteroarylderivater og anvendelser deraf
DK2807160T3 (da) Antifibrotiske forbindelser og anvendelser deraf
DK3392252T3 (da) Pyrimido-pyridazinonforbindelser og deres anvendelse
EP2842163A4 (en) NONLINEAR MEMRISTORS
IL237914A0 (en) Benzamides
DK3326642T3 (da) Langtidsvirkende koagulationsfaktorer og brugen deraf
DK2943460T3 (da) 6-chlor-3-(phenyl-d5)-inden-1-on og anvendelse deraf
DK2877736T3 (da) Skrue, skruedel og fremgangsmåde dertil