BR112014027834A2 - composições de nanopartícula lipídica para entrega de oligonucleotídeos antissenso - Google Patents

composições de nanopartícula lipídica para entrega de oligonucleotídeos antissenso

Info

Publication number
BR112014027834A2
BR112014027834A2 BR112014027834A BR112014027834A BR112014027834A2 BR 112014027834 A2 BR112014027834 A2 BR 112014027834A2 BR 112014027834 A BR112014027834 A BR 112014027834A BR 112014027834 A BR112014027834 A BR 112014027834A BR 112014027834 A2 BR112014027834 A2 BR 112014027834A2
Authority
BR
Brazil
Prior art keywords
lipid nanoparticle
akt
nucleic acid
directed
aso
Prior art date
Application number
BR112014027834A
Other languages
English (en)
Portuguese (pt)
Inventor
Ho Ahn Chang
Joong Kim Deog
J Lee Robert
Bok Lee Young
Original Assignee
Univ Ohio State
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 Univ Ohio State filed Critical Univ Ohio State
Publication of BR112014027834A2 publication Critical patent/BR112014027834A2/pt

Links

Classifications

    • 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
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/08—Peptides having 5 to 11 amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/10—Peptides having 12 to 20 amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A61K47/544—Phospholipids
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6907—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a microemulsion, nanoemulsion or micelle
    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • 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
    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • 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
    • 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
    • C12N2310/10—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
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21064—Peptidase K (3.4.21.64)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Dispersion Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nanotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
BR112014027834A 2012-05-23 2013-05-23 composições de nanopartícula lipídica para entrega de oligonucleotídeos antissenso BR112014027834A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261650729P 2012-05-23 2012-05-23
US201361784892P 2013-03-14 2013-03-14
PCT/US2013/042454 WO2013177415A1 (en) 2012-05-23 2013-05-23 Lipid nanoparticle compositions for antisense oligonucleotides delivery

Publications (1)

Publication Number Publication Date
BR112014027834A2 true BR112014027834A2 (pt) 2017-08-08

Family

ID=48539454

Family Applications (2)

Application Number Title Priority Date Filing Date
BR112014029247A BR112014029247A2 (pt) 2012-05-23 2013-05-23 nanopartícula de lipídio, composição farmacêutica, método para fazer uma nanopartícula de lipído, produto, método de tratamento de um distúrbio, e sistema de entrega
BR112014027834A BR112014027834A2 (pt) 2012-05-23 2013-05-23 composições de nanopartícula lipídica para entrega de oligonucleotídeos antissenso

Family Applications Before (1)

Application Number Title Priority Date Filing Date
BR112014029247A BR112014029247A2 (pt) 2012-05-23 2013-05-23 nanopartícula de lipídio, composição farmacêutica, método para fazer uma nanopartícula de lipído, produto, método de tratamento de um distúrbio, e sistema de entrega

Country Status (10)

Country Link
US (4) US20160015824A1 (enExample)
EP (3) EP2852380A4 (enExample)
JP (4) JP2015525209A (enExample)
KR (3) KR102169891B1 (enExample)
CN (3) CN104582691A (enExample)
AU (4) AU2013266232B2 (enExample)
BR (2) BR112014029247A2 (enExample)
CA (3) CA2874495A1 (enExample)
MX (3) MX384803B (enExample)
WO (3) WO2013177419A2 (enExample)

Families Citing this family (105)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2820873T3 (es) 2008-05-16 2021-04-22 Taiga Biotechnologies Inc Anticuerpos y su procesos de preparación
ES2681478T3 (es) 2008-08-28 2018-09-13 Taiga Biotechnologies, Inc. Moduladores de MYC, métodos de uso de los mismos y métodos para identificar agentes que modulan MYC
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
RS63430B1 (sr) 2010-10-01 2022-08-31 Modernatx Inc Ribonukleinske kiseline koje sadrže n1-metil-pseudouracile i njihove primene
EP2691101A2 (en) 2011-03-31 2014-02-05 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
DK3682905T3 (da) 2011-10-03 2022-02-28 Modernatx Inc Modificerede nukleosider, nukleotider og nukleinsyrer og anvendelser deraf
DE12858350T1 (de) 2011-12-16 2021-10-07 Modernatx, Inc. Modifizierte mrna zusammensetzungen
HK1206601A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
BR112014029247A2 (pt) 2012-05-23 2017-06-27 Univ Ohio State nanopartícula de lipídio, composição farmacêutica, método para fazer uma nanopartícula de lipído, produto, método de tratamento de um distúrbio, e sistema de entrega
CA3133302A1 (en) 2012-07-20 2014-01-23 Taiga Biotechnologies, Inc. Enhanced reconstitution and autoreconstitution of the hematopoietic compartment comprising a myc polypeptide
HRP20220607T1 (hr) 2012-11-26 2022-06-24 Modernatx, Inc. Terminalno modificirana rna
US10272115B2 (en) 2013-03-11 2019-04-30 Taiga Biotechnologies, Inc. Production and use of red blood cells
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9745583B2 (en) 2013-04-30 2017-08-29 Delta-Fly Pharma, Inc. Liposome for topical administration and application thereof
US9802967B2 (en) * 2013-06-17 2017-10-31 The University Of North Carolina At Chapel Hill Polymer coated particles and methods thereof
WO2015013673A1 (en) 2013-07-25 2015-01-29 Aurasense Therapeutics, Llc Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
WO2015051214A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
EP3556353A3 (en) 2014-02-25 2020-03-18 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
US9833416B2 (en) * 2014-04-04 2017-12-05 Ohio State Innovation Foundation Oligonucleotide lipid nanoparticle compositions, methods of making and methods of using the same
WO2015175965A1 (en) 2014-05-15 2015-11-19 The Research Foundation For Suny Compositions targeting the interaction domain between p27kip1 and brk and methods of use thereof
ES2725948T3 (es) 2014-06-04 2019-09-30 Exicure Inc Suministro multivalente de inmunomoduladores mediante ácidos nucleicos esféricos liposomales para aplicaciones profilácticas o terapéuticas
JP6797108B2 (ja) 2014-08-19 2020-12-09 ノースウェスタン ユニバーシティ タンパク質／オリゴヌクレオチドコアシェルナノ粒子治療薬
US9855345B2 (en) 2014-10-30 2018-01-02 Delta-Fly Pharma, Inc. Method for producing lipoplex for topical administration and antitumor agent using such lipoplex
CA2968531A1 (en) 2014-11-21 2016-05-26 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
WO2016115320A1 (en) 2015-01-14 2016-07-21 Exicure, Inc. Nucleic acid nanostructructures with core motifs
US20160287152A1 (en) * 2015-03-30 2016-10-06 Verily Life Sciences Llc Functionalized Nanoparticles, Methods and In Vivo Diagnostic System
EP3283059B1 (en) 2015-04-13 2024-01-03 CureVac Manufacturing GmbH Method for producing rna compositions
WO2016197024A2 (en) * 2015-06-05 2016-12-08 MiRagen Therapeutics, Inc. Mir-155 inhibitors for treating cutaneous t cell lymphoma (ctcl)
WO2017049245A2 (en) 2015-09-17 2017-03-23 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
CN105288647B (zh) * 2015-10-10 2018-05-25 中国药科大学 功能化白蛋白及其纳米制剂的制备方法
JP7080172B2 (ja) 2015-12-10 2022-06-03 モデルナティエックス インコーポレイテッド 治療薬の送達のための組成物及び方法
ES2913626T5 (en) 2015-12-22 2025-05-12 Modernatx Inc Compounds and compositions for intracellular delivery of agents
WO2018039629A2 (en) 2016-08-25 2018-03-01 Northwestern University Micellar spherical nucleic acids from thermoresponsive, traceless templates
CN107951862B (zh) * 2016-10-17 2021-03-12 南京绿叶制药有限公司 一种抑制bcl-2的反义寡聚核酸的脂质纳米粒及其制备方法
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3538067A1 (en) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
AU2017367730A1 (en) * 2016-12-02 2019-06-06 Taiga Biotechnologies, Inc. Nanoparticle formulations
US20200069594A1 (en) * 2016-12-09 2020-03-05 Board Of Regents, The University Of Texas System Hybrid exosomal-polymeric (hexpo) nano-platform for delivery of rnai therapeutics
EP3554561B1 (en) 2016-12-14 2023-06-28 Janssen Biotech, Inc. Cd137 binding fibronectin type iii domains
EP3554535A4 (en) 2016-12-14 2020-10-21 Janssen Biotech, Inc. PD-L1 BINDING FIBRONECTIN TYPE III DOMAINS
RU2759952C2 (ru) 2016-12-14 2021-11-19 Янссен Байотек, Инк. Cd8a-связывающие домены типа iii фибронектина
US20180179577A1 (en) * 2016-12-22 2018-06-28 Jiaming HU Lipid-polymer Hybrid Nanoparticle Biochip and Application Thereof
EP3558408B1 (en) * 2016-12-22 2024-04-17 Biotronik AG Intratumoral drug delivery materials and methods for treating breast cancer
WO2018124423A1 (ko) * 2016-12-30 2018-07-05 주식회사 삼양바이오팜 플라스미드 디엔에이 전달용 고분자 나노입자 조성물 및 그의 제조방법
JP7220154B2 (ja) 2017-03-15 2023-02-09 モデルナティエックス インコーポレイテッド アミノ脂質の結晶形態
EP3596041B1 (en) 2017-03-15 2022-11-02 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
WO2018170336A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Lipid nanoparticle formulation
US11905525B2 (en) 2017-04-05 2024-02-20 Modernatx, Inc. Reduction of elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
WO2018209270A1 (en) 2017-05-11 2018-11-15 Northwestern University Adoptive cell therapy using spherical nucleic acids (snas)
CN107184552B (zh) * 2017-06-07 2021-03-30 东华大学 一种半乳糖化聚乙烯亚胺修饰的载药醇质体的制备方法
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
JP7231147B2 (ja) * 2017-06-29 2023-03-01 国立大学法人東海国立大学機構 Ｒｎａ導入試薬及びその利用
CN118853394A (zh) * 2017-07-18 2024-10-29 阿维塔斯有限公司 使用连续流动的流体系统进行跨细胞膜的有效装载的传递
US10149898B2 (en) 2017-08-03 2018-12-11 Taiga Biotechnologies, Inc. Methods and compositions for the treatment of melanoma
EP4328314A3 (en) * 2017-08-22 2024-03-20 Tessera Therapeutics, Inc. Lipid nanoparticle methods and compositions for producing engineered erythroid cells
JP7275111B2 (ja) 2017-08-31 2023-05-17 モデルナティエックス インコーポレイテッド 脂質ナノ粒子の生成方法
CN107982537A (zh) * 2017-11-17 2018-05-04 厦门大学 针对microRNA-155的治疗性药物及其应用
KR102245539B1 (ko) * 2018-02-12 2021-04-29 주식회사 지앤피바이오사이언스 코어-쉘 구조의 마이크로 입자를 유효성분으로 포함하는 성장인자 유전자 발현 증가용 조성물
EP3789043A4 (en) * 2018-03-29 2022-08-03 Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences EXTRACTION OF "MEDICINAL SOUP" FROM PLANT SOURCE AND MANUAL PREPARATION OF "MEDICINAL PLANT" AND ASSOCIATED PRODUCTS
JP7592493B2 (ja) * 2018-04-18 2024-12-02 オイシン バイオテクノロジーズ，インク． 治療用タンパク質の標的細胞に特異的な産生のための、および、標的細胞に関連する疾患、疾病、または障害の処置のための、融合性脂質ナノ粒子、および、融合性脂質ナノ粒子の製造方法と使用方法
KR20210013580A (ko) * 2018-05-15 2021-02-04 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 유해물 방제 조성물 및 그의 용도
US10987428B2 (en) * 2018-06-01 2021-04-27 City Of Hope Phosphorothioate-conjugated miRNAs and methods of using the same
EP3808344A4 (en) * 2018-06-01 2022-03-30 Sogang University Research Foundation NANOPARTICLE COMPOSITE WITH IMPROVED ENDOCYTICAL EFFICIENCY THROUGH SURFACE MODIFICATION USING A LIPID AND MANUFACTURING PROCESSES THEREOF
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
CN108743962B (zh) * 2018-06-19 2021-07-02 东华大学 一种基于荧光碳点修饰的树状大分子的双载药靶向纳米平台的制备方法
AU2019335055B2 (en) * 2018-09-04 2025-05-29 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
CN112996519B (zh) * 2018-09-04 2025-02-28 德克萨斯大学系统董事会 用于核酸的器官特异性递送的组合物和方法
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
SG11202107282VA (en) * 2019-01-04 2021-07-29 Oncorus Inc Encapsulated rna polynucleotides and methods of use
AU2020272664A1 (en) 2019-04-08 2021-11-04 Taiga Biotechnologies, Inc. Compositions and methods for the cry opreservation of immune cells
AU2020274117A1 (en) 2019-05-14 2021-12-02 Taiga Biotechnologies, Inc. Compositions and methods for treating T cell exhaustion
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
US11628222B2 (en) 2019-10-14 2023-04-18 Aro Biotherapeutics Company CD71 binding fibronectin type III domains
US11781138B2 (en) 2019-10-14 2023-10-10 Aro Biotherapeutics Company FN3 domain-siRNA conjugates and uses thereof
US12491255B2 (en) 2019-10-14 2025-12-09 Aro Biotherapeutics Company EPCAM binding fibronectin type III domains
CN115778904B (zh) * 2019-10-29 2025-03-28 珠海丽凡达生物技术有限公司 一种用于体外转染和体内递送mRNA的制剂
KR102198736B1 (ko) 2020-01-15 2021-01-05 이화여자대학교 산학협력단 생체 내 약물 전달을 위한 지질 나노입자 및 이의 용도
CA3170142A1 (en) * 2020-02-19 2021-08-26 Nammi Therapeutics, Inc. Formulated and/or co-formulated liposome compositions containing tfgb antagonist prodrugs useful in the treatment of cancer and methods thereof
BR112023000327A2 (pt) 2020-07-16 2023-01-31 Acuitas Therapeutics Inc Lipídeos catiônicos para o uso em nanopartículas lipídicas
JP2023537609A (ja) * 2020-08-14 2023-09-04 アークトゥラス・セラピューティクス・インコーポレイテッド 脂質ナノ粒子を凍結乾燥する方法
WO2022192674A1 (en) * 2021-03-11 2022-09-15 The Trustees Of The University Of Pennsylvania Targeted therapeutic lipid nanoparticles and methods of use
CA3215367A1 (en) 2021-04-14 2022-10-20 Swapnil Kulkarni Fn3 domain-sirna conjugates and uses thereof
CA3214552A1 (en) 2021-04-14 2022-10-20 Russell C. Addis Cd71 binding fibronectin type iii domains
EP4419112A4 (en) * 2021-10-22 2025-08-27 Ohio State Innovation Foundation IMMUNOTHERAPY FOR CANCER TREATMENT
AU2022381929B2 (en) * 2021-11-03 2025-07-03 Biond Biologics Ltd. Intracellular delivery compositions
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
CN114306369B (zh) * 2021-12-23 2023-12-26 北京悦康科创医药科技股份有限公司 一种硫代寡核苷酸注射液及其制备方法
WO2023192503A1 (en) * 2022-03-30 2023-10-05 The Johns Hopkins University Compositions of lipid nanoparticles for plasmid dna delivery to the liver and methods for preparing the same
WO2024044370A1 (en) * 2022-08-25 2024-02-29 Ohio State Innovation Foundation Compositions and methods for the delivery of active agents including nucleic acids
WO2024050310A1 (en) * 2022-08-29 2024-03-07 Sorrento Therapeutics, Inc. Lipid-coated nanoparticles
WO2024136219A1 (ko) * 2022-12-19 2024-06-27 주식회사 포스테라헬스사이언스 나노 입자를 포함하는 흡입 제형 및 이의 제조 방법
CN121013733A (zh) * 2023-03-31 2025-11-25 白橡树集团公司 Archexin的脂质体组合物
WO2025034965A1 (en) * 2023-08-09 2025-02-13 Board Of Regents, The University Of Texas System Targeted therapeutic delivery of lipid nanoparticles
TWI880568B (zh) * 2024-01-04 2025-04-11 訊聯生物科技股份有限公司 包含生物巨分子及奈米載體之組合物、包含其的醫藥組成物及其用途
WO2025171027A1 (en) * 2024-02-05 2025-08-14 University Of Rhode Island Board Of Trustees Phlip ®-lnp for targeted intracellular delivery of nucleic acid therapeutics
US20250297260A1 (en) 2024-03-22 2025-09-25 Takeda Pharmaceutical Company Limited Compositions and methods for inhibiting cytochrome p450 family 7 subfamily a member 1 (cyp7a1) expression

Family Cites Families (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US590877A (en) * 1897-09-28 Danger-signal for railway-crossings
US2003203A (en) * 1933-05-15 1935-05-28 Kim Young Wo Game
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
DK0641192T3 (da) 1992-05-18 1998-03-02 Minnesota Mining & Mfg Anordning til transmucosal lægemiddelafgivelse
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US20020022264A1 (en) * 1995-05-26 2002-02-21 Sullivan Sean M. Delivery vehicles comprising stable lipid/nucleic acid complexes
US5908777A (en) * 1995-06-23 1999-06-01 University Of Pittsburgh Lipidic vector for nucleic acid delivery
US6248720B1 (en) * 1996-07-03 2001-06-19 Brown University Research Foundation Method for gene therapy using nucleic acid loaded polymeric microparticles
US6056973A (en) * 1996-10-11 2000-05-02 Sequus Pharmaceuticals, Inc. Therapeutic liposome composition and method of preparation
TWI225412B (en) * 1997-06-23 2004-12-21 Sequus Pharm Inc Liposome-entrapped polynucleotide composition and method
US6395253B2 (en) * 1998-04-23 2002-05-28 The Regents Of The University Of Michigan Microspheres containing condensed polyanionic bioactive agents and methods for their production
AU5717400A (en) * 1999-06-14 2001-01-02 Cancer Research Ventures Limited Cancer therapy
CZ20021029A3 (cs) * 1999-08-27 2002-08-14 Inex Pharmaceuticals Corp. Kompozice a její pouľití pro stimulaci sekrece cytokinů a indukci imunitní odpovědi
US7060291B1 (en) 1999-11-24 2006-06-13 Transave, Inc. Modular targeted liposomal delivery system
US6821955B2 (en) * 2000-04-07 2004-11-23 Baylor College Of Medicine Macroaggregated protein conjugates as oral genetic immunization delivery agents
US6613308B2 (en) 2000-09-19 2003-09-02 Advanced Inhalation Research, Inc. Pulmonary delivery in treating disorders of the central nervous system
US20030203865A1 (en) * 2001-04-30 2003-10-30 Pierrot Harvie Lipid-comprising drug delivery complexes and methods for their production
US7025987B2 (en) 2001-05-30 2006-04-11 The Scripps Research Institute Delivery system for nucleic acids
JP2003088371A (ja) * 2001-09-04 2003-03-25 Mitsubishi Pharma Corp 遺伝子導入用組成物
KR100484371B1 (ko) 2001-10-25 2005-04-20 가부시키가이샤 아텍스 히터선부착 시트체의 제조방법
EP1441763A2 (en) * 2001-11-07 2004-08-04 Inex Pharmaceuticals Corp. Mucosal adjuvants comprising an oligonucleotide and a cationic lipid
US7678386B2 (en) 2002-07-15 2010-03-16 Board Of Regents The University Of Texas Liposomes coated with selected antibodies that bind to aminophospholipids
CN100558740C (zh) * 2002-08-16 2009-11-11 雷克珊公司 反义寡核苷酸用于抑制Akt-1表达的用途
WO2004064731A2 (en) * 2003-01-14 2004-08-05 University Of Washington Lipid-drug formulations and methods for targeted delivery of lipid-drug complexes to lymlplhoid tissues
AU2004207576B8 (en) * 2003-01-28 2008-11-20 Rexahn Pharmaceuticals, Inc. Antisense oligonucleotides that inhibit expression of HIF-1
BRPI0407070A (pt) 2003-01-31 2006-01-24 Rexahn Corp Oligonucleotìdeos antisentido que inibem a expressão de hif-1
NZ581166A (en) * 2003-09-15 2011-06-30 Protiva Biotherapeutics Inc Polyethyleneglycol-modified lipid compounds and uses thereof
KR100638041B1 (ko) 2003-12-24 2006-10-23 주식회사 삼양사 수용성 약물의 경구투여용 나노입자 조성물 및 그의제조방법
JP2006111591A (ja) * 2004-10-15 2006-04-27 Anges Mg Inc 核酸医薬を標的特異的に細胞内送達するための製剤
JP2006298780A (ja) * 2005-04-15 2006-11-02 Tohoku Univ Ｈｇｆ遺伝子と粗大凝集アルブミン−ポリエチレンイミン（ｍａａ−ｐｅｉ）との複合体
US20070087045A1 (en) * 2005-10-14 2007-04-19 Industrial Technology Research Institute Lipid carrier and method of preparing the same
TW200800235A (en) * 2005-10-18 2008-01-01 Otsuka Pharma Co Ltd Carrier composition for nucleic acid transport
CN101346393B (zh) * 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
US8067380B2 (en) 2005-12-19 2011-11-29 Industrial Technology Research Institute Glutathione-based delivery system
GB2450475A (en) 2007-06-12 2008-12-31 Univ Nottingham Trent Antimicrobial polymer nanocomposite
US20110177155A1 (en) * 2007-08-21 2011-07-21 Immune Disease Institute, Inc. Methods of delivery of agents to leukocytes and endothelial cells
PL208054B1 (pl) * 2007-09-06 2011-03-31 Akademia Medyczna Im Piastow Śląskich We Wrocławiu Kompozycja lipidowa do wytwarzania lipidowego nośnika dla leków genetycznych i jej zastosowanie
EP2217221B1 (en) * 2007-10-17 2018-06-27 Korea Advanced Institute of Science and Technology Ldl-like cationic nanoparticles for deliverying nucleic acid gene, method for preparing thereof and method for deliverying nucleic acid gene using the same
BRPI0820302A2 (pt) * 2007-11-09 2015-05-19 Univ Northeastern Nanopartículas semelhantes a micelas de automontagem para liberação sistêmica de gene
EP2222851B1 (en) * 2007-11-20 2017-06-28 Ionis Pharmaceuticals, Inc. Modulation of cd40 expression
WO2009120247A2 (en) 2007-12-27 2009-10-01 The Ohio State University Research Foundation Lipid nanoparticle compositions and methods of making and using the same
EP2265174B1 (en) 2008-04-04 2018-06-06 The Regents of The University of California Functionalized magnetic nanoparticles and methods of use thereof
US8222220B2 (en) 2008-05-13 2012-07-17 George Mason Intellectual Properties, Inc. Nanogenomics for medicine: siRNA engineering
AU2009249403A1 (en) * 2008-05-19 2009-11-26 The University Of North Carolina At Chapel Hill Methods and compositions comprising novel cationic lipids
US20090312402A1 (en) * 2008-05-20 2009-12-17 Contag Christopher H Encapsulated nanoparticles for drug delivery
EP2321414B1 (en) * 2008-07-25 2018-01-10 Alnylam Pharmaceuticals, Inc. Enhancement of sirna silencing activity using universal bases or mismatches in the sense strand
WO2010014595A2 (en) 2008-07-31 2010-02-04 The Ohio State University Research Foundation Methods and compositions for delivering therapeutic agents in the treatment of b-cell related disorders
EP2366386A4 (en) * 2008-10-28 2013-11-27 Univ Santiago Compostela NANOTEHTIC SYSTEMS MANUFACTURED FROM ANIONIC POLYMERS
WO2010054384A1 (en) * 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Lipids and compositions for the delivery of therapeutics
EP2601934A1 (en) 2009-01-22 2013-06-12 Ludwig-Maximilians-Universität München Vesicular phospholipid gels comprising proteinaceous substances
ES2351756B1 (es) 2009-07-28 2011-10-05 Universidad Del Pais Vasco Nanopartículas lipídicas para terapia génica.
AU2010321555B2 (en) * 2009-11-23 2015-10-15 The Ohio State University Materials and methods useful for affecting tumor cell growth, migration and invasion
JP2013528665A (ja) * 2010-03-26 2013-07-11 メルサナ セラピューティックス， インコーポレイテッド ポリヌクレオチドの送達のための修飾ポリマー、その製造方法、およびその使用方法
CN103025384B (zh) * 2010-03-26 2016-04-20 俄亥俄州立大学 涉及miR-155对于错配修复和基因组稳定性的调节的材料和方法
EP2561072A4 (en) 2010-04-20 2016-04-06 Univ Florida NANOZYME, METHOD FOR THE PRODUCTION OF NANOCYMENTS AND METHOD FOR THE USE OF NANOCYMENTS
US20130260460A1 (en) * 2010-04-22 2013-10-03 Isis Pharmaceuticals Inc Conformationally restricted dinucleotide monomers and oligonucleotides
US20110319473A1 (en) * 2010-06-29 2011-12-29 Surmodics, Inc. Compositions and methods for enhancement of nucleic acid delivery
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
CN102552105B (zh) * 2011-10-17 2014-04-02 复旦大学 一种级联脑部靶向药物递送系统及其制备方法和用途
BR112014029247A2 (pt) 2012-05-23 2017-06-27 Univ Ohio State nanopartícula de lipídio, composição farmacêutica, método para fazer uma nanopartícula de lipído, produto, método de tratamento de um distúrbio, e sistema de entrega

Also Published As

Publication number Publication date
AU2013266232A1 (en) 2014-11-20
KR20150032945A (ko) 2015-03-31
KR20150020180A (ko) 2015-02-25
WO2013177419A3 (en) 2014-02-20
AU2017245294B2 (en) 2019-09-12
US20180021447A1 (en) 2018-01-25
WO2013177421A2 (en) 2013-11-28
MX384803B (es) 2025-03-14
WO2013177415A1 (en) 2013-11-28
BR112014029247A2 (pt) 2017-06-27
MX353567B (es) 2018-01-18
EP2852380A2 (en) 2015-04-01
CN104582691A (zh) 2015-04-29
JP2015525209A (ja) 2015-09-03
CN104428005B (zh) 2019-05-10
JP2015519346A (ja) 2015-07-09
EP2852381A2 (en) 2015-04-01
AU2013266236A1 (en) 2014-12-18
KR102169891B1 (ko) 2020-10-26
AU2013266238B2 (en) 2017-07-27
US20130315937A1 (en) 2013-11-28
WO2013177421A3 (en) 2014-02-27
JP2018002727A (ja) 2018-01-11
KR20150032944A (ko) 2015-03-31
JP6220389B2 (ja) 2017-10-25
EP2852415A1 (en) 2015-04-01
US10307490B2 (en) 2019-06-04
EP2852381B1 (en) 2020-10-07
CA2874490A1 (en) 2013-11-28
CN105163721B (zh) 2018-05-22
CA2874495A1 (en) 2013-11-28
EP2852381A4 (en) 2016-01-13
CA2871477A1 (en) 2013-11-28
CN105163721A (zh) 2015-12-16
MX2014014196A (es) 2015-08-14
WO2013177419A2 (en) 2013-11-28
AU2013266232B2 (en) 2017-08-03
CN104428005A (zh) 2015-03-18
JP6228191B2 (ja) 2017-11-08
MX2014014251A (es) 2015-06-17
CA2874490C (en) 2021-11-16
US20160015824A1 (en) 2016-01-21
EP2852380A4 (en) 2016-01-20
AU2013266238A1 (en) 2014-12-18
US9750819B2 (en) 2017-09-05
AU2017245294A1 (en) 2017-11-02
US20150118288A1 (en) 2015-04-30
JP2015523969A (ja) 2015-08-20

Similar Documents

Publication Publication Date Title
BR112014027834A2 (pt) composições de nanopartícula lipídica para entrega de oligonucleotídeos antissenso
ZA202103233B (en) Lipid nanoparticle formulations
BR212016030926U2 (pt) conjugados de alvo e partículas e formulações dos mesmos
Irache et al. Nanomedicine: novel approaches in human and veterinary therapeutics
BR112016027705A2 (pt) lipídios biodegradáveis para distribuição de ácidos nucleicos
BR112012024590A2 (pt) métodos de reforço da liberação de fármaco e da eficácia de agentes terapêuticos
HK1217085A1 (zh) 包封在顆粒中的靶向綴合物及其製劑
MX2019005287A (es) Lipidos cationicos para suministro de acido nucleico y su preparacion.
BR112014011841A2 (pt) material de entrega de fármacos hidrofóbicos, método para a produção do mesmo e métodos para a entrega de uma composição de entrega de fármacos
EP4393549A3 (en) Compositions for the delivery of trna as nanoparticles and methods of use therewith
CO7101246A2 (es) Compuesto de carbamato y preparación y uso de los mismos
CA2863964C (en) Compartmentalized method of nucleic acid delivery and compositions and uses thereof
BR112014010291A2 (pt) composições de lipossoma combinacionais para terapia de câncer
BR112015021470A2 (pt) conjugado de polímero para administração de um agente bioativo
PH12012502399A1 (en) Combination therapy methods for treating proliferative diseases
BR112014027681A2 (pt) preparações de agentes terapêuticos hidrofóbicos, métodos de fabricação e uso dos mesmos
BR112013020273A2 (pt) oligonucleotídeos antissenso
BR112013018920A2 (pt) composições de nanopartículas, formulações destas e seus usos
BRPI0809164B8 (pt) Composição para administração tópica e composição farmacêutica compreendendo a referida composição
BR112014008932A2 (pt) bicamadas lipídicas suportadas em nanopartículas porosas (protocélulas) para administração direcionada, incluindo a administração transdérmica de uma carga e métodos associados
BR112015032432A2 (pt) Conjugado de oligonucleotídeo antisense, oligômero, composição farmacêutica, uso de um oligômero ou conjugado de oligonucleotídeo antisense ou composição farmacêutica, método de tratamento da hipercolesterolemia ou de distúrbios relacionados e método in vivo ou in vitro
BR112017027985A2 (pt) peptídeos terapêuticos e métodos de uso dos mesmos
BR112015017246A2 (pt) formulação farmacêutica que compreende um corticosteroide insolúvel e um corticosteroide solúvel
BR112013017750A8 (pt) nanocápsulas com revestimento polimérico
BR112017028552A2 (pt) partículas e conjugados direcionados e formulações dos mesmos

Legal Events

Date Code Title Description
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B15K Others concerning applications: alteration of classification

Ipc: A61K 47/00 (2006.01), A61P 35/00 (2006.01)

B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B11B Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements