JP7317706B2 - 核酸およびタンパク質ペイロード送達のための方法および組成物 - Google Patents

核酸およびタンパク質ペイロード送達のための方法および組成物 Download PDF

Info

Publication number
JP7317706B2
JP7317706B2 JP2019531743A JP2019531743A JP7317706B2 JP 7317706 B2 JP7317706 B2 JP 7317706B2 JP 2019531743 A JP2019531743 A JP 2019531743A JP 2019531743 A JP2019531743 A JP 2019531743A JP 7317706 B2 JP7317706 B2 JP 7317706B2
Authority
JP
Japan
Prior art keywords
cells
payload
cases
targeting ligand
nucleic acid
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2019531743A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020502120A5 (enExample
JP2020502120A (ja
Inventor
アンドレ ロナルド ワトソン
クリスチャン フォスター
Original Assignee
リガンダル インコーポレイテッド
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 リガンダル インコーポレイテッド filed Critical リガンダル インコーポレイテッド
Publication of JP2020502120A publication Critical patent/JP2020502120A/ja
Publication of JP2020502120A5 publication Critical patent/JP2020502120A5/ja
Priority to JP2023117292A priority Critical patent/JP2023134730A/ja
Application granted granted Critical
Publication of JP7317706B2 publication Critical patent/JP7317706B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—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
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • 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
    • A61K47/62—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
    • 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
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • 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
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing 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
    • A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • 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
    • A61K47/6921—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 particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—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 particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—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 particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A61K47/6931—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 particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
    • A61K47/6935—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 particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol
    • A61K47/6937—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 particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol the polymer being PLGA, PLA or polyglycolic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • A61K9/5153—Polyesters, e.g. poly(lactide-co-glycolide)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing 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
    • C12N15/111—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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nanotechnology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Inorganic Chemistry (AREA)
  • Optics & Photonics (AREA)
  • Neurosurgery (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
JP2019531743A 2016-12-14 2017-12-14 核酸およびタンパク質ペイロード送達のための方法および組成物 Active JP7317706B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023117292A JP2023134730A (ja) 2016-12-14 2023-07-19 核酸およびタンパク質ペイロード送達のための方法および組成物

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662434344P 2016-12-14 2016-12-14
US62/434,344 2016-12-14
US201762443522P 2017-01-06 2017-01-06
US201762443567P 2017-01-06 2017-01-06
US62/443,522 2017-01-06
US62/443,567 2017-01-06
US201762517346P 2017-06-09 2017-06-09
US62/517,346 2017-06-09
PCT/US2017/066541 WO2018112278A1 (en) 2016-12-14 2017-12-14 Methods and compositions for nucleic acid and protein payload delivery

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023117292A Division JP2023134730A (ja) 2016-12-14 2023-07-19 核酸およびタンパク質ペイロード送達のための方法および組成物

Publications (3)

Publication Number Publication Date
JP2020502120A JP2020502120A (ja) 2020-01-23
JP2020502120A5 JP2020502120A5 (enExample) 2021-08-12
JP7317706B2 true JP7317706B2 (ja) 2023-07-31

Family

ID=62488586

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2019531743A Active JP7317706B2 (ja) 2016-12-14 2017-12-14 核酸およびタンパク質ペイロード送達のための方法および組成物
JP2019531758A Pending JP2020502124A (ja) 2016-12-14 2017-12-14 核酸および／またはタンパク質ペイロード送達のための組成物および方法
JP2023031534A Pending JP2023071853A (ja) 2016-12-14 2023-03-02 核酸および／またはタンパク質ペイロード送達のための組成物および方法
JP2023117292A Pending JP2023134730A (ja) 2016-12-14 2023-07-19 核酸およびタンパク質ペイロード送達のための方法および組成物

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2019531758A Pending JP2020502124A (ja) 2016-12-14 2017-12-14 核酸および／またはタンパク質ペイロード送達のための組成物および方法
JP2023031534A Pending JP2023071853A (ja) 2016-12-14 2023-03-02 核酸および／またはタンパク質ペイロード送達のための組成物および方法
JP2023117292A Pending JP2023134730A (ja) 2016-12-14 2023-07-19 核酸およびタンパク質ペイロード送達のための方法および組成物

Country Status (10)

Country Link
US (5) US20180179553A1 (enExample)
EP (2) EP3554555A4 (enExample)
JP (4) JP7317706B2 (enExample)
KR (4) KR20210035022A (enExample)
CN (2) CN110582301A (enExample)
AU (2) AU2017378427A1 (enExample)
CA (2) CA3045134A1 (enExample)
IL (2) IL267033B2 (enExample)
RU (2) RU2019121801A (enExample)
WO (2) WO2018112282A1 (enExample)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210069016A1 (en) * 2008-11-13 2021-03-11 Gholam A. Peyman Neurodegenerative Disorder Treatment Method
KR102712656B1 (ko) 2017-01-23 2024-10-04 리제너론 파마슈티칼스 인코포레이티드 Hsd17b13 변종 및 이것의 용도
US11479802B2 (en) 2017-04-11 2022-10-25 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family
KR102720855B1 (ko) 2017-06-15 2024-10-25 더 리전트 오브 더 유니버시티 오브 캘리포니아 표적화된 비-바이러스 dna 삽입
KR20200033259A (ko) 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
KR102780441B1 (ko) 2017-07-31 2025-03-17 리제너론 파마슈티칼스 인코포레이티드 Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
KR102503130B1 (ko) 2017-10-27 2023-02-24 더 리전트 오브 더 유니버시티 오브 캘리포니아 내인성 t 세포 수용체의 표적화된 대체
CN111885915B (zh) 2018-03-19 2023-04-28 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
EP3796894A4 (en) * 2018-04-24 2022-05-04 Ligandal, Inc. METHODS AND COMPOSITIONS FOR GENOMIC EDITING
US20210317436A1 (en) * 2018-09-08 2021-10-14 Blueallele, Llc Methods and compositions for modifying the von willebrand factor gene
EP3877526A4 (en) * 2018-11-08 2023-03-29 Summation Bio, Inc. Mini-nucleosome core proteins and use in nucleic acid delivery
KR20210102309A (ko) * 2018-12-05 2021-08-19 프레드 헛친슨 켄서 리서치 센터 유전적으로-변형된 세포를 제작하는 줄어든, 그리고 최소한의 조작
WO2020131862A1 (en) * 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US11249941B2 (en) * 2018-12-21 2022-02-15 Palo Alto Research Center Incorporated Exabyte-scale data storage using sequence-controlled polymers
CN111363013B (zh) * 2018-12-25 2021-11-19 深圳先进技术研究院 多组分纳米颗粒簇的构建方法
CN109735540B (zh) * 2019-01-22 2022-05-31 南京鼓楼医院 SH2D1A基因、sgRNA及其应用
US20210379192A1 (en) * 2019-01-25 2021-12-09 Mantra Bio, Inc. Skeletal muscle targeting moieties and uses thereof
KR20210134716A (ko) 2019-03-01 2021-11-10 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 폴리리보뉴클레오티드의 전달을 위한 조성물, 방법, 및 키트
AU2020232238B2 (en) 2019-03-01 2025-10-30 Flagship Pioneering Innovations Vi, Llc Polyribonucleotides and cosmetic uses thereof
WO2020223705A2 (en) * 2019-05-02 2020-11-05 Ligandal, Inc. Methods and compositions for diagnostically-responsive ligand-targeted delivery of therapeutic agents
PE20220902A1 (es) 2019-06-26 2022-05-30 Biorchestra Co Ltd Nanoparticulas micelares y sus usos
JP7630904B2 (ja) * 2019-07-23 2025-02-18 株式会社東芝 核酸導入キャリア、核酸導入キャリアセット、核酸導入組成物及び核酸導入方法
CN110628723B (zh) * 2019-09-05 2021-05-04 清华大学 基因修饰MSCs治疗2型糖尿病
US20210163935A1 (en) * 2019-09-05 2021-06-03 Trevor P. Castor Targeted Critical Fluid Nanoparticles Platform for Delivery of Nucleic Acids for Treatment of HIV-1 and Other Diseases
CN112779317A (zh) * 2019-11-11 2021-05-11 香港中文大学深圳研究院 一种用于小分子核糖核酸检测的试剂及其应用
US20230248658A1 (en) * 2019-11-15 2023-08-10 The Regents Of The University Of California Supramolecular nanosubstrate-mediated delivery system enables crispr/cas9 knockin of hemoglobin beta gene-a potential therapeutic solution for hemoglobinopathies
WO2021146184A1 (en) 2020-01-13 2021-07-22 Fluent Biosciences Inc. Single cell sequencing
JP7562682B2 (ja) 2020-01-27 2024-10-07 マントラ バイオ，インコーポレイテッド キメラ小胞局在化部分を含む非天然発生の小胞、その産生方法、およびその使用
KR20220158723A (ko) * 2020-02-25 2022-12-01 리간달 인코포레이티드 생체모방 바이러스 펩티드의 확인 및 그의 용도
WO2021168575A1 (en) * 2020-02-27 2021-09-02 Quadrumix Biotechnology Inc. Polypeptides directed against viral infection and uses thereof
CA3175931A1 (en) 2020-03-16 2021-09-23 Fluent Biosciences Inc. Multi-omic analysis in monodisperse droplets
US12428685B2 (en) 2020-03-24 2025-09-30 Illumina, Inc. Viral detection using template emulsification
WO2021211467A1 (en) * 2020-04-13 2021-10-21 Summation Bio, Inc. Modified mini-nucleosome core proteins and use in nucleic acid delivery
AU2021275213A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
JP2023534028A (ja) 2020-07-15 2023-08-07 フルーエント バイオサイエンシーズ インコーポレイテッド 段階的ライゲーション用オリゴ
CN111968707B (zh) * 2020-08-07 2022-06-17 上海交通大学 基于能量的原子结构与电子密度图多目标优化拟合预测方法
CN111888333A (zh) * 2020-08-11 2020-11-06 深圳大学 一种转铁蛋白受体靶向的纳米胶束及其制备方法与应用
WO2022051629A1 (en) 2020-09-03 2022-03-10 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
WO2022144812A1 (en) * 2020-12-30 2022-07-07 Biorchestra Co., Ltd. Micellar nanoparticles and uses thereof
US20240131048A1 (en) * 2020-12-30 2024-04-25 Biorchestra Co., Ltd. Micellar nanoparticles and uses thereof
EP4338267A4 (en) * 2021-05-14 2025-01-08 University of Rochester Variants of sirt6 for use in preventing and/or treating age-related diseases
EP4340947A4 (en) * 2021-05-18 2025-02-26 Illumina, Inc. Perturbed genomic expression in pretemplated instant partitions
WO2023023528A1 (en) * 2021-08-17 2023-02-23 California Institute Of Technology Cell-to-cell delivery of rna circuits
KR102775653B1 (ko) * 2021-09-15 2025-03-05 한국기초과학지원연구원 아르기닌 리치 펩타이드를 포함하는 단백질 분해제 및 이를 이용한 단백질 이상질환의 예방 또는 치료용 약학적 조성물
CA3232968A1 (en) 2021-10-14 2023-04-20 Jasper Williams Immune cells having co-expressed shrnas and logic gate systems
CN115304756B (zh) * 2022-01-30 2023-05-09 上海科技大学 一种五元脂质纳米颗粒及其制备方法和应用
CN114480672B (zh) * 2022-02-22 2023-08-15 青岛农业大学 一种通过miR-145筛选高产肉率碱地黑牛的方法
CN114933658B (zh) * 2022-04-24 2023-07-11 深圳市鹏泰生物科技有限公司 一种短肽元件及其应用方法
CN115184480B (zh) * 2022-05-24 2024-07-09 南京中医药大学 一种用于鉴定华蟾素制剂或蟾皮的特征多肽及其含量测定方法与应用
CN115177734B (zh) * 2022-07-05 2025-03-14 中国人民解放军空军军医大学 一种靶向干预线粒体基因表达的工程化外泌体及其构建方法和应用
CN115350330B (zh) * 2022-09-01 2023-10-20 北京化工大学 一种负电性小分子调控的表面在蛋白差异性黏附上的应用
WO2024098238A1 (en) * 2022-11-08 2024-05-16 Pulsar Therapeutics Inc. Methods for treating hemoglobinopathies
WO2025049677A1 (en) * 2023-08-31 2025-03-06 Tufts Medical Center, Inc. Use of nanoparticles in therapeutic and imaging methods
WO2025090678A1 (en) * 2023-10-23 2025-05-01 The Board Of Regents Of The University Of Texas System Novel endoplasmic reticulum targeting signal peptides and uses thereof
CN117599200A (zh) * 2023-10-27 2024-02-27 华中科技大学 一种新型的双链rna递送载体及应用
WO2025102075A1 (en) * 2023-11-09 2025-05-15 The Board Of Regents Of The University Of Texas System Endogenous synapse targeting signal peptides and uses thereof
KR20250160320A (ko) * 2024-05-03 2025-11-12 한양대학교 산학협력단 엔도좀 탈출 효율이 향상된 항 인간 트랜스페린 수용체 항체, 및 이를 이용한 다중특이적 항체 및 약학 조성물

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002544127A (ja) 1999-04-30 2002-12-24 アミリン・ファーマシューティカルズ，インコーポレイテッド 修飾されたエキセンジンおよびエキセンジン・アゴニスト
US20080312410A1 (en) 2000-06-06 2008-12-18 Backer Marina V Cysteine-containing peptide tag for site-specific conjugation of proteins
JP2009530395A (ja) 2006-03-21 2009-08-27 アミリン・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−ぺプチダーゼ阻害剤及びその使用
JP2011006447A (ja) 2000-12-07 2011-01-13 Eli Lilly & Co Ｇｌｐ−１融合タンパク質
JP2012531467A (ja) 2009-07-02 2012-12-10 アンジオケム インコーポレーテッド 多量体ペプチドコンジュゲートおよびその使用
JP2013506628A (ja) 2009-09-30 2013-02-28 グラクソ グループ リミテッド 延長された半減期を有する薬物融合物及びコンジュゲート
WO2015042585A1 (en) 2013-09-23 2015-03-26 Rensselaer Polytechnic Institute Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations

Family Cites Families (104)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US6379966B2 (en) 1999-02-26 2002-04-30 Mirus Corporation Intravascular delivery of non-viral nucleic acid
JP3937727B2 (ja) 1997-10-29 2007-06-27 株式会社カネカ 生分解性を有する発泡体用樹脂組成物
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7098032B2 (en) 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
AU2001257331A1 (en) 2000-04-28 2001-11-12 Sangamo Biosciences, Inc. Methods for designing exogenous regulatory molecules
US20030082561A1 (en) 2000-07-21 2003-05-01 Takashi Sera Zinc finger domain recognition code and uses thereof
ATE320792T1 (de) 2000-09-28 2006-04-15 Chiron Corp Mikropartikel zur verabreichung von heterologen nukleinsäure
CA2435394C (en) 2001-01-22 2018-01-09 Sangamo Biosciences, Inc. Modified zinc finger binding proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040102606A1 (en) 2001-04-24 2004-05-27 Danuta Balicki Histone H2A -derived peptides useful in gene delivery
US6805904B2 (en) 2002-02-20 2004-10-19 International Business Machines Corporation Process of forming a multilayer nanoparticle-containing thin film self-assembly
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
CN100419426C (zh) 2002-04-22 2008-09-17 佛罗里达州立大学 功能化纳米微粒及其使用方法
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
WO2004085998A2 (en) 2003-03-28 2004-10-07 The Children's Hospital Of Philadelphia Biomimetic hierarchies using functionalized nanoparticles as building blocks
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP3222715A1 (en) 2003-08-08 2017-09-27 Sangamo BioSciences, Inc. Methods and compositions for targeted cleavage and recombination
JP2007502833A (ja) 2003-08-21 2007-02-15 サウスウエスト・リサーチ・インスティチュート 骨格に標的とされるナノ粒子
EP1748796A4 (en) 2004-03-05 2008-08-27 Univ Texas METHOD AND SYSTEM FOR ADMINISTERING MULTILAYER NANOMEDICAMENTS
CA2565633C (en) 2004-05-05 2016-04-26 Atugen Ag Lipids, lipid complexes and use thereof
WO2005123142A1 (en) 2004-06-10 2005-12-29 Abraxis Bioscience, Inc. A method using inorganic nanoparticles as non-viral vectors for gene therapy
US20060088599A1 (en) 2004-08-02 2006-04-27 Prasad Paras N Amino functionalized ORMOSIL nanoparticles as delivery vehicles
US20090220587A1 (en) * 2005-02-01 2009-09-03 United State Army Liposomal drug delivery constructs targeted by lipid-conjugated peptide ligands
SI2019683T2 (sl) 2006-04-25 2022-10-28 The Regents Of The University Of California Dajanje rastnih faktorjev za zdravljenje motenj CŽS
US7928186B2 (en) * 2006-08-02 2011-04-19 Phoenix Pharmaceuticals, Inc. Cell permeable bioactive peptide conjugates
WO2008021207A2 (en) 2006-08-11 2008-02-21 Dow Agrosciences Llc Zinc finger nuclease-mediated homologous recombination
US20100015218A1 (en) 2007-02-16 2010-01-21 Vasant Jadhav Compositions and methods for potentiated activity of biologically active molecules
WO2008109806A2 (en) 2007-03-08 2008-09-12 Massachusetts Institute Of Technology Electrostatic coating of particles for drug delivery
TWI428135B (zh) 2007-03-26 2014-03-01 Hirofumi Takeuchi And a carrier composition for quick-acting nucleic acid delivery
CA2700170A1 (en) 2007-09-27 2009-04-02 Sangamo Biosciences, Inc. Genomic editing in zebrafish using zinc finger nucleases
AU2008317354B2 (en) 2007-10-25 2014-04-10 Ospedale San Raffaele S.R.L. Methods and compositions for targeted integration
JP2011517279A (ja) 2007-10-29 2011-06-02 ユニバーシティ オブ マサチューセッツ 核酸（ｓｉＲＮＡ）送達用の酵母細胞壁粒子（ＹＣＷＰ）多層状ナノ粒子
WO2009059309A2 (en) 2007-11-01 2009-05-07 Panacea Pharmaceuticals, Inc. Furin-cleavable peptide linkers for drug-ligand conjugates
US8323618B2 (en) 2007-11-07 2012-12-04 University Of Houston System Ultrasmall superparamagnetic iron oxide nanoparticles and uses thereof
US20100285111A1 (en) 2007-11-09 2010-11-11 Northeastern University Self-assembling micelle-like nanoparticles for systemic gene delivery
GB0725321D0 (en) 2007-12-31 2008-02-06 Syntaxin Ltd Delivery vehicles
US7999025B2 (en) 2008-01-28 2011-08-16 University Of Utah Research Foundation Asymmetrically-functionalized nanoparticles organized on one-dimensional chains
US8344116B2 (en) 2008-03-17 2013-01-01 Case Western Reserve University Polymers and complexes for delivery of nucleic acids to intracellular targets
US8324333B2 (en) 2008-06-05 2012-12-04 Wisconsin Alumni Research Foundation Anionic charge-dynamic polymers for release of cationic agents
US8404636B2 (en) 2008-07-17 2013-03-26 George Mason Intellectual Properties, Inc. Targeted delivery of antimicrobial agents
ES2595364T3 (es) * 2008-10-07 2016-12-29 The Regents Of The University Of California Producción de proteínas NELL recombinantes
CN105152939A (zh) 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
CN102625655B (zh) 2008-12-04 2016-07-06 桑格摩生物科学股份有限公司 使用锌指核酸酶在大鼠中进行基因组编辑
TWI576432B (zh) 2009-04-07 2017-04-01 陶氏農業科學公司 序列特異性核酸酶之奈米粒子媒介式遞送技術
US20110077581A1 (en) 2009-09-25 2011-03-31 Georgia Tech Research Corporation Targeted cellular delivery of nanoparticles
US20120277158A1 (en) * 2009-10-06 2012-11-01 Angiochem Inc. Compositions and methods for the transport of therapeutic agents
CA2783351C (en) 2009-12-10 2021-09-07 Regents Of The University Of Minnesota Tal effector-mediated dna modification
WO2011096408A1 (ja) 2010-02-02 2011-08-11 国立大学法人 東京大学 複合体微粒子およびその製造方法、ならびに該複合体微粒子を用いた薬学組成物
KR101198715B1 (ko) 2010-05-14 2012-11-13 한국생명공학연구원 핵산 및 친수성 음이온 화합물의 고효율 포획을 위한 비대칭 리포솜 및 이의 제조방법
WO2012012352A2 (en) * 2010-07-19 2012-01-26 Amidebio, Llc Modified peptides and proteins
US9233163B2 (en) * 2010-12-28 2016-01-12 The Children's Hospital Of Philadelphia Hydrolytically releasable prodrugs for sustained release nanoparticle formulations
US9074187B2 (en) 2011-03-21 2015-07-07 Board Of Trustees Of The University Of Arkansas Nanostructural materials that increase mineralization in bone cells and affect gene expression through miRNA regulation and applications of same
WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
EP2694091B1 (en) 2011-04-05 2019-03-13 Cellectis Method for the generation of compact tale-nucleases and uses thereof
PT2702160T (pt) 2011-04-27 2020-07-30 Amyris Inc Métodos para modificação genómica
US8916696B2 (en) * 2011-06-12 2014-12-23 City Of Hope Aptamer-mRNA conjugates for targeted protein or peptide expression and methods for their use
WO2013058812A1 (en) * 2011-10-19 2013-04-25 President And Fellows Of Harvard College Targeted delivery to pancreatic islet endothelial cells
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
CZ303963B6 (cs) 2012-01-13 2013-07-17 Ústav organické chemie a biochemie Akademie ved CR, v.v.i. Lipopolyaminy sperminového typu pro konstrukci liposomálních transfekcních systému
WO2013163234A1 (en) 2012-04-23 2013-10-31 Massachusetts Institute Of Technology Stable layer-by-layer coated particles
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
RS62288B9 (sr) 2012-06-08 2021-12-31 Nitto Denko Corp Lipidi za formulacije za dostavu terapeutskih agenasa
US20140005379A1 (en) 2012-06-20 2014-01-02 Frank GU Nanoparticle delivery system and components thereof
KR20150105956A (ko) 2012-12-12 2015-09-18 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
PT2784162E (pt) 2012-12-12 2015-08-27 Broad Inst Inc Engenharia de sistemas, métodos e composições guia otimizadas para a manipulação de sequências
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
BR102013032129B1 (pt) 2012-12-13 2022-06-07 Dow Agrosciences Llc Método para identificar a presença de um polinucleotídeo de dna doador exógeno inserido dentro de um único locus genômico eucariótico alvo
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
US9504747B2 (en) 2013-03-08 2016-11-29 Novartis Ag Lipids and lipid compositions for the delivery of active agents
WO2014140113A1 (en) * 2013-03-13 2014-09-18 Biocrine Ab Methods for treating and/or limiting development of diabetes
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US20140364333A1 (en) 2013-03-15 2014-12-11 President And Fellows Of Harvard College Methods for Live Imaging of Cells
US20140273235A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota ENGINEERING PLANT GENOMES USING CRISPR/Cas SYSTEMS
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
WO2014165612A2 (en) 2013-04-05 2014-10-09 Dow Agrosciences Llc Methods and compositions for integration of an exogenous sequence within the genome of plants
EP3456831B1 (en) 2013-04-16 2021-07-14 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
EP2994531B1 (en) 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US20140349405A1 (en) 2013-05-22 2014-11-27 Wisconsin Alumni Research Foundation Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis
US9873907B2 (en) 2013-05-29 2018-01-23 Agilent Technologies, Inc. Method for fragmenting genomic DNA using CAS9
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
PL3057604T3 (pl) 2013-10-14 2021-12-20 Nanosphere Health Sciences Inc. Kompozycje nanocząstek i sposoby jako nośniki czynników nutraceutycznych przez błony komórkowe i bariery biologiczne
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3079726B1 (en) 2013-12-12 2018-12-05 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
SG10201805073VA (en) 2013-12-13 2018-07-30 Univ Nanyang Tech Multilayered nanoparticle and methods of manufacturing and using the same
WO2015187651A1 (en) * 2014-06-02 2015-12-10 Li-Cor, Inc. Therapeutic and diagnostic probes
HK1244479A1 (zh) 2014-11-18 2018-08-10 阿克丘勒斯治疗公司 用於rna递送的可电离阳离子脂质
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002544127A (ja) 1999-04-30 2002-12-24 アミリン・ファーマシューティカルズ，インコーポレイテッド 修飾されたエキセンジンおよびエキセンジン・アゴニスト
US20080312410A1 (en) 2000-06-06 2008-12-18 Backer Marina V Cysteine-containing peptide tag for site-specific conjugation of proteins
JP2011006447A (ja) 2000-12-07 2011-01-13 Eli Lilly & Co Ｇｌｐ−１融合タンパク質
JP2009530395A (ja) 2006-03-21 2009-08-27 アミリン・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−ぺプチダーゼ阻害剤及びその使用
JP2012531467A (ja) 2009-07-02 2012-12-10 アンジオケム インコーポレーテッド 多量体ペプチドコンジュゲートおよびその使用
JP2013506628A (ja) 2009-09-30 2013-02-28 グラクソ グループ リミテッド 延長された半減期を有する薬物融合物及びコンジュゲート
WO2015042585A1 (en) 2013-09-23 2015-03-26 Rensselaer Polytechnic Institute Nanoparticle-mediated gene delivery, genomic editing and ligand-targeted modification in various cell populations

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Bioconjugate Chem.，2008年，Vol.19，p.2156-2162

Also Published As

Publication number Publication date
RU2019121992A3 (enExample) 2021-01-26
EP3554546A1 (en) 2019-10-23
US10975388B2 (en) 2021-04-13
JP2020502120A (ja) 2020-01-23
RU2019121801A (ru) 2021-01-15
KR102641844B1 (ko) 2024-02-28
EP3554546A4 (en) 2021-04-14
AU2017378427A1 (en) 2019-06-20
WO2018112278A1 (en) 2018-06-21
US20180179553A1 (en) 2018-06-28
KR20240027888A (ko) 2024-03-04
CA3045134A1 (en) 2018-06-21
RU2019121992A (ru) 2021-01-15
JP2023071853A (ja) 2023-05-23
JP2023134730A (ja) 2023-09-27
US20200095605A1 (en) 2020-03-26
KR20240172763A (ko) 2024-12-10
US12123012B2 (en) 2024-10-22
CN110582302A (zh) 2019-12-17
CA3045131A1 (en) 2018-06-21
IL267034B1 (en) 2024-11-01
IL267034A (en) 2019-07-31
US20210324406A1 (en) 2021-10-21
KR20210035021A (ko) 2021-03-31
EP3554555A4 (en) 2021-02-24
EP3554555A1 (en) 2019-10-23
JP2020502124A (ja) 2020-01-23
CN110582301A (zh) 2019-12-17
IL267033B2 (en) 2024-12-01
IL267034B2 (en) 2025-03-01
IL267033A (en) 2019-07-31
KR20210035022A (ko) 2021-03-31
AU2017378431A1 (en) 2019-06-20
RU2019121801A3 (enExample) 2021-10-12
WO2018112282A1 (en) 2018-06-21
IL267033B1 (en) 2024-08-01
US20180161447A1 (en) 2018-06-14
US20240026375A1 (en) 2024-01-25

Similar Documents

Publication Publication Date Title
JP7317706B2 (ja) 核酸およびタンパク質ペイロード送達のための方法および組成物
US20230323401A1 (en) Methods and compositions for genome editing
RS62863B1 (sr) Konstruisane vanćelijske vezikule i njihove upotrebe
US20200208177A1 (en) Methods and compositions for genome editing
US20230059921A1 (en) Methods and compositions for diagnostically-responsive ligand-targeted delivery of therapeutic agents

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20191224

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20201211

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20201211

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210628

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20210630

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211227

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220217

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220627

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221031

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230127

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230424

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230524

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230608

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230719

R150 Certificate of patent or registration of utility model

Ref document number: 7317706

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150