Provided herein is chitosan-derivative nanoparticle comprising chitosan functionalized with a cationic amino acid and a hydrophilic polyol; and methods of making and using same, e.g., for gene delivery in vivo.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
2.
COMBINATION GENE THERAPY FOR TREATMENT OF METASTATIC CANCER
The present disclosure relates to methods and compositions for the localized expression of IL-12, preferably in combination with an RIG-I agonist, for activating a memory T cell response to a cancer antigen. In embodiments, the method is effective for treating metastatic disease.
Provided herein are methods and compositions for the amelioration of inflammatory disorders comprising the intestinal expression of programmed death ligand 1.
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
A61P 1/04 - Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
A61P 1/06 - Anti-spasmodics, e.g. drugs for colics, esophagic dyskinesia
A61P 1/00 - Drugs for disorders of the alimentary tract or the digestive system
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
4.
DUALLY DERIVATIZED CHITOSAN NANOPARTICLES AND METHODS OF MAKING AND USING THE SAME FOR GENE TRANSFER IN VIVO
Provided herein is chitosan dually derivatized with arginine and gluconic acid; and methods of making and using the same, e.g., for gene delivery in vivo.
A61K 47/61 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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 the organic macromolecular compound being a polysaccharide or a derivative thereof
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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
C07H 13/04 - Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids by carboxylic acids having the esterifying carboxyl radicals attached to acyclic carbon atoms
Provided herein are methods and compositions for the treatment of lung disorders comprising the expression of therapeutic nucleic acid(s) in human airway epithelial cells, including the treatment of cystic fibrosis and disorders caused by expression of a mutated CFTR gene comprising the expression of functional CFTR in human airway epithelial cells.
Chitosan-nucleic acid polyplex compositions containing a reversibly bound polymer coat comprising linear block copolymers with a polyanionic anchor region and at least one polyethylene glycol tail region are described herein. In some cases, the compositions exhibit improved stability and/or mucosal diffusion as compared to uncoated particles. In some cases, the reversibly bound polymer coat does not interfere with, or enhances, transfection of target cells or tissues as compared to uncoated particles.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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
A61K 47/60 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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 the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
A61K 47/61 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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 the organic macromolecular compound being a polysaccharide or a derivative thereof
7.
CHITOSAN POLYPLEX-BASED LOCALIZED EXPRESSION OF IL-12 ALONE OR IN COMBINATION WITH TYPE-I IFN INDUCERS FOR TREATMENT OF MUCOSAL CANCERS
The present disclosure relates to methods and compositions comprising derivatized-chitosan polyplexes reversibly coated with a polyanion-containing block co-polymer for the localized expression of IL-12 in mucosal tissues, preferably in combination with an IFN-1 activator/inducer, for use in cancer immunotherapy.
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 47/60 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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 the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
8.
COMBINATION GENE THERAPY FOR TREATMENT OF METASTATIC CANCER
The present disclosure relates to methods and compositions for the localized expression of IL-12, preferably in combination with an RIG-I agonist, for activating a memory T cell response to a cancer antigen. In embodiments, the method is effective for treating metastatic disease.
A61K 47/36 - PolysaccharidesDerivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
Provided herein is chitosan-derivative nanoparticle comprising chitosan functionalized with a cationic amino acid and a hydrophilic polyol; and methods of making and using same, e.g., for gene delivery in vivo.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
10.
LOCALIZED EXPRESSION OF THERAPEUTIC NUCLEIC ACIDS IN LUNG EPITHELIAL CELLS
Provided herein are methods and compositions for the treatment of lung disorders comprising the expression of therapeutic nucleic acid(s) in human airway epithelial cells, including the treatment of cystic fibrosis and disorders caused by expression of a mutated CFTR gene comprising the expression of functional CFTR in human airway epithelial cells.
A61K 47/36 - PolysaccharidesDerivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
05 - Pharmaceutical, veterinary and sanitary products
40 - Treatment of materials; recycling, air and water treatment,
42 - Scientific, technological and industrial services, research and design
Goods & Services
Preparations and substances for use in the treatment of genetic deficiencies or disorders, diseases, infectious diseases, immune system-related diseases or immune system-mediated disorders and metabolic diseases and disorders, cancers, cardiovascular diseases and disorders, hematologic diseases and disorders, neurologic diseases and disorders, skeletal muscular diseases and disorders, pulmonary diseases and disorders, gastrointestinal diseases and disorders and aging Custom manufacturing of pharmaceutical preparations and substances for others; manufacturing services in the field of cell and gene therapy, namely, manufacturing and processing of viral and non-viral gene vectors, proteins, peptides, antibodies and DNA to order and/or specification of others Medical research and development services in the field of gene therapy and genetic medicine; scientific research and development; and pharmaceutical research and development
12.
REVERSIBLE COATING OF CHITOSAN-NUCLEIC ACID NANOPARTICLES AND METHODS OF THEIR USE
Chitosan-nucleic acid polyplex compositions containing a reversibly bound polymer coat comprising linear block copolymers with a polyanionic anchor region and at least one polyethylene glycol tail region are described herein. In some cases, the compositions exhibit improved stability and/or mucosal diffusion as compared to uncoated particles. In some cases, the reversibly bound polymer coat does not interfere with, or enhances, transfection of target cells or tissues as compared to uncoated particles.
A61K 47/36 - PolysaccharidesDerivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/87 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
13.
CHITOSAN POLYPLEX-BASED LOCALIZED EXPRESSION OF IL-12 ALONE OR IN COMBINATION WITH TYPE-I IFN INDUCERS FOR TREATMENT OF MUCOSAL CANCERS
The present disclosure relates to methods and compositions comprising derivatized-chitosan polyplexes reversibly coated with a polyanion-containing block co-polymer for the localized expression of IL-12 in mucosal tissues, preferably in combination with an IFN- 1 activator/inducer, for use in cancer immunotherapy.
A61K 47/36 - PolysaccharidesDerivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 47/30 - Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
A61K 47/34 - Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/117 - Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
Provided herein is chitosan-derivative nanoparticle comprising chitosan functionalized with a cationic amino acid and a hydrophilic polyol; and methods of making and using same, e.g., for gene delivery in vivo.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
15.
Dually derivatized chitosan nanoparticles and methods of making and using the same for gene transfer in vivo
Provided herein is chitosan dually derivatized with arginine and gluconic acid; and methods of making and using the same, e.g., for gene delivery in vivo.
A61K 47/61 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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 the organic macromolecular compound being a polysaccharide or a derivative thereof
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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
C07H 13/04 - Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids by carboxylic acids having the esterifying carboxyl radicals attached to acyclic carbon atoms
Provided herein are methods and compositions for the amelioration of inflammatory disorders comprising the intestinal expression of programmed death ligand 1.
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
A61P 1/00 - Drugs for disorders of the alimentary tract or the digestive system
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
A61P 1/04 - Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
A61K 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61P 1/06 - Anti-spasmodics, e.g. drugs for colics, esophagic dyskinesia
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
17.
Dually derivatized chitosan nanoparticles and methods of making and using the same for gene transfer in vivo
Provided herein is chitosan-derivative nanoparticle comprising chitosan functionalized with a cationic amino acid and a hydrophilic polyol; and methods of making and using same, e.g., for gene delivery in vivo.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
18.
High concentration chitosan-nucleic acid polyplex compositions
The invention provides highly concentrated chitosan-nucleic acid polyplex compositions and dispersions, and methods for producing the compositions and dispersions. Methods of mixing the chitosan-nucleic acid polyplexes include an inline mixing of chitosan solution and nucleic acid solution, followed by further concentrating the dispersion of chitosan-nucleic acid polyplexes, optionally with an aggregation inhibitor. Further provides are methods for altering the diameter of chitosan-nucleic acid polyplexes.
Provided herein are methods and compositions for the amelioration of inflammatory disorders comprising the intestinal expression of programmed death ligand 1.
Provided herein is chitosan dually derivatized with arginine and gluconic acid; and methods of making and using the same, e.g., for gene delivery in vivo.
A61K 47/61 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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 the organic macromolecular compound being a polysaccharide or a derivative thereof
C12N 15/87 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
C07H 13/04 - Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids by carboxylic acids having the esterifying carboxyl radicals attached to acyclic carbon atoms
A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting 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
Provided herein is chitosan-derivative nanoparticle comprising chitosan functionalized with a cationic amino acid and a hydrophilic polyol; and methods of making and using same, e.g., for gene delivery in vivo.
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
22.
DUALLY DERIVATIZED CHITOSAN NANOPARTICLES AND METHODS OF MAKING AND USING THE SAME FOR GENE TRANSFER IN VIVO
Provided herein is chitosan-derivative nanoparticle comprising chitosan functionalized with a cationic amino acid and a hydrophilic polyol; and methods of making and using same, e.g., for gene delivery in vivo.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/87 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
23.
High concentration chitosan-nucleic acid polyplex compositions
The invention provides highly concentrated chitosan-nucleic acid polyplex compositions and dispersions, and methods for producing the compositions and dispersions. Methods of mixing the chitosan-nucleic acid polyplexes include an inline mixing of chitosan solution and nucleic acid solution, followed by further concentrating the dispersion of chitosan-nucleic acid polyplexes, optionally with an aggregation inhibitor. Further provides are methods for altering the diameter of chitosan-nucleic acid polyplexes.
Provided herein is chitosan dually derivatized with arginine and gluconic acid; and methods of making and using the same, e.g., for gene delivery in vivo.
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/87 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
25.
Chitosan-based non-viral methods for transfecting gut cells in vivo
The present invention provides chitosan-based nanoparticles that can protect nucleic acids and deliver the same into gut mucosal cells. Compositions and methods for the expression of therapeutic nucleic acids in cells of the gut mucosa are provided. Compositions and methods for delivering therapeutic proteins systemically from cells of the gut mucosa are also provided.
The invention provides highly acidic chitosan-nucleic acid polyplex compositions. The compositions may be used to transfect cells in vitro and in vivo, and are particularly useful for transfecting cells of mucosal epithelia.
C12N 15/87 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
27.
HIGH CONCENTRATION CHITOSAN-NUCLEIC ACID POLYPLEX COMPOSITIONS
The invention provides highly concentrated chitosan-nucleic acid polyplex compositions and dispersions, and methods for producing the compositions and dispersions. Methods of mixing the chitosan-nucleic acid polyplexes include an inline mixing of chitosan solution and nucleic acid solution, followed by further concentrating the dispersion of chitosan-nucleic acid polyplexes, optionally with an aggregation inhibitor. Further provided are methods for altering the diameter of chitosan-nucleic acid polyplexes.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
C12N 15/87 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
28.
NON-VIRAL COMPOSITIONS AND METHODS FOR TRANSFECTING GUT CELLS IN VIVO
The present invention provides chitosan-based nanoparticles that can protect nucleic acids and deliver the same into gut mucosal cells. Compositions and methods for the expression of therapeutic nucleic acids in cells of the gut mucosa are provided. Compositions and methods for delivering therapeutic proteins systemically from cells of the gut mucosa are also provided.
05 - Pharmaceutical, veterinary and sanitary products
42 - Scientific, technological and industrial services, research and design
Goods & Services
(1) Preparations and substances for use in the treatment of genetic deficiencies or disorders, diseases, infectious diseases, immune system-related diseases or immune system-mediated disorders and metabolic diseases and disorders, cancers, cardiovascular diseases and disorders, hematologic diseases and disorders, neurologic diseases and disorders, skeletal muscular diseases and disorders, pulmonary diseases and disorders, gastrointestinal diseases and disorders and aging. (1) Medical, scientific and pharmaceutical research and development; preparation of pharmaceutical preparations and substances; preparation of viral and non-viral gene vectors, proteins, peptides, antibodies and DNA
