JP7425066B2 - 核酸を臓器特異的送達するための組成物および方法 - Google Patents

核酸を臓器特異的送達するための組成物および方法 Download PDF

Info

Publication number
JP7425066B2
JP7425066B2 JP2021536681A JP2021536681A JP7425066B2 JP 7425066 B2 JP7425066 B2 JP 7425066B2 JP 2021536681 A JP2021536681 A JP 2021536681A JP 2021536681 A JP2021536681 A JP 2021536681A JP 7425066 B2 JP7425066 B2 JP 7425066B2
Authority
JP
Japan
Prior art keywords
lipid
sort
cationic
composition
cas9
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
JP2021536681A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021535227A (ja
JP2021535227A5 (enExample
Inventor
チャン チェン
トゥオ ウェイ
ダニエル ジェイ． シーグワート
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Texas System
Original Assignee
University of Texas System
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 University of Texas System filed Critical University of Texas System
Publication of JP2021535227A publication Critical patent/JP2021535227A/ja
Publication of JP2021535227A5 publication Critical patent/JP2021535227A5/ja
Priority to JP2023189771A priority Critical patent/JP7815196B2/ja
Application granted granted Critical
Publication of JP7425066B2 publication Critical patent/JP7425066B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/186—Quaternary ammonium compounds, e.g. benzalkonium chloride or cetrimide
    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic 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
    • A61K47/69—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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • 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
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • 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
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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]
    • 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nanotechnology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Optics & Photonics (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Manufacturing & Machinery (AREA)
  • Immunology (AREA)
  • Dispersion Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021536681A 2018-09-04 2019-09-04 核酸を臓器特異的送達するための組成物および方法 Active JP7425066B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023189771A JP7815196B2 (ja) 2018-09-04 2023-11-07 核酸を臓器特異的送達するための組成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862726741P 2018-09-04 2018-09-04
US62/726,741 2018-09-04
PCT/US2019/049565 WO2020051223A1 (en) 2018-09-04 2019-09-04 Compositions and methods for organ specific delivery of nucleic acids

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023189771A Division JP7815196B2 (ja) 2018-09-04 2023-11-07 核酸を臓器特異的送達するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2021535227A JP2021535227A (ja) 2021-12-16
JP2021535227A5 JP2021535227A5 (enExample) 2022-09-13
JP7425066B2 true JP7425066B2 (ja) 2024-01-30

Family

ID=69722750

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021536681A Active JP7425066B2 (ja) 2018-09-04 2019-09-04 核酸を臓器特異的送達するための組成物および方法
JP2023189771A Active JP7815196B2 (ja) 2018-09-04 2023-11-07 核酸を臓器特異的送達するための組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023189771A Active JP7815196B2 (ja) 2018-09-04 2023-11-07 核酸を臓器特異的送達するための組成物および方法

Country Status (8)

Country Link
US (9) US11229609B2 (enExample)
EP (1) EP3846822A4 (enExample)
JP (2) JP7425066B2 (enExample)
CN (2) CN112996519B (enExample)
AU (2) AU2019336679B2 (enExample)
CA (1) CA3111484A1 (enExample)
GB (3) GB2592505B (enExample)
WO (1) WO2020051223A1 (enExample)

Families Citing this family (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11752213B2 (en) 2015-12-21 2023-09-12 Duke University Surfaces having reduced non-specific binding and antigenicity
CN117018228A (zh) 2016-05-27 2023-11-10 创思瑞普泰克斯公司 用合成信使rna治疗原发性纤毛运动障碍
JP2020500150A (ja) 2016-09-23 2020-01-09 デューク ユニバーシティ 下限臨界溶液温度挙動を有する非反復かつ非構造的ポリペプチド
WO2019006374A1 (en) 2017-06-30 2019-01-03 Duke University ORDER AND DISORDER AS A DESIGN PRINCIPLE FOR STIMULI-SENSITIVE BIOPOLYMER NETWORKS
LT3675826T (lt) * 2017-08-31 2023-09-11 Life Technologies Corporation Katijoninės lipidų kompozicijos audiniams-specifiniam įterpimui
WO2019147954A1 (en) 2018-01-26 2019-08-01 Duke University Albumin binding peptide-drug (aibiped) conjugates and methods of making and using same
EP3788343B1 (en) 2018-04-30 2024-03-27 Duke University Stimuli-responsive peg-like polymer-based drug delivery platform
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
WO2020051223A1 (en) 2018-09-04 2020-03-12 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
EP3846857A4 (en) 2018-09-04 2022-10-12 The Board Of Regents Of The University Of Texas System COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS
US12594347B2 (en) 2018-09-07 2026-04-07 Duke University Nanoparticulate drug delivery systems
US11512314B2 (en) 2019-07-12 2022-11-29 Duke University Amphiphilic polynucleotides
KR20220044816A (ko) * 2019-08-12 2022-04-11 인티그레이티드 나노테라퓨틱스 아이엔씨. 하전 물질 전달을 위한 지질, 이의 제형 및 그 제조 방법
US20240293317A1 (en) * 2020-04-20 2024-09-05 Board Of Regents, The University Of Texas System Lipid nanoparticle (lnp) delivery systems and uses thereof
EP4153560A4 (en) * 2020-05-19 2024-04-24 Ramot at Tel-Aviv University Ltd. LIPID PARTICLES FOR NUCLEIC ACID DELIVERY AND CLINICAL APPLICATIONS THEREOF
EP4228658A4 (en) * 2020-10-14 2025-02-19 George Mason Research Foundation, Inc. IONIZABLE LIPIDS AND METHODS FOR THE PRODUCTION AND USE THEREOF
JP2024500879A (ja) * 2020-12-22 2024-01-10 コーネル ユニヴァーシティー 双性イオン性脂質ナノ粒子組成物およびその使用方法
WO2022204215A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204043A1 (en) * 2021-03-22 2022-09-29 The Board Of Regents Of The University Of Texas System Compositions and methods for targeted delivery to cells
JP2024511463A (ja) * 2021-03-22 2024-03-13 リコード セラピューティクス，インク． 細胞への標的送達のための組成物および方法
IL305998A (en) * 2021-03-23 2023-11-01 Recode Therapeutics Inc Formulations and methods for targeted systemic delivery to cells
CA3213107A1 (en) * 2021-03-23 2022-09-29 Mirko HENNIG Polynucleotide compositions, related formulations, and methods of use thereof
US20240207442A1 (en) * 2021-04-22 2024-06-27 The Board Of Regents Of The University Of Texas System All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo
KR20240027630A (ko) * 2021-06-01 2024-03-04 나노베이션 테라퓨틱스 인크. 지질 나노입자를 사용한 dna 벡터 전달
CN118201605A (zh) * 2021-10-25 2024-06-14 杜克大学 基于poegma的脂质纳米粒子
IL313876A (en) * 2021-12-27 2024-08-01 Univ Texas Compositions and methods for targeted delivery to cells
WO2023158738A2 (en) * 2022-02-16 2023-08-24 Trustees Of Tufts College Lipid nanoparticles for delivery to the eye or ear
WO2023165583A1 (zh) * 2022-03-04 2023-09-07 益杰立科(上海)生物科技有限公司 靶向眼部细胞的递送系统和方法
WO2023178425A1 (en) 2022-03-23 2023-09-28 Nanovation Therapeutics Inc. High sterol-containing lipid nanoparticles
CA3246723A1 (en) * 2022-03-30 2023-10-05 The Johns Hopkins University Lipid nanoparticle compositions for the delivery of plasma DNA to the liver and their preparation methods
WO2023184038A1 (en) * 2022-04-01 2023-10-05 Nanovation Therapeutics Inc. Mrna delivery method and composition thereof
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
CN114887071B (zh) * 2022-06-06 2023-09-22 郑州大学第一附属医院 一种靶向脾脏的纳米递送载体
CN114887070B (zh) * 2022-06-06 2023-09-22 郑州大学第一附属医院 一种脾脏靶向的纳米药物
CN114891808B (zh) * 2022-06-21 2023-10-27 珠海丽凡达生物技术有限公司 编码ALDH2多肽的mRNA分子、应用及mRNA药物
EP4547230A1 (en) * 2022-06-29 2025-05-07 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids
US20250312287A1 (en) 2022-07-08 2025-10-09 Polyplus Transfection Lipid nanoparticles comprising an imidazolium-based cationic lipid
WO2024040194A1 (en) 2022-08-17 2024-02-22 Capstan Therapeutics, Inc. Conditioning for in vivo immune cell engineering
US20250360083A1 (en) * 2022-09-21 2025-11-27 Board Of Regents Of The University Of Nebraska Lipid nanoparticle formulations and methods of use thereof
CN116236565B (zh) * 2022-11-21 2025-06-27 成都诺恩生物科技有限公司 适合于肌注给药的核酸-脂质纳米颗粒、制剂及其应用
JP2025538636A (ja) * 2022-11-24 2025-11-28 ザミディアム セラピューティクス カンパニー リミテッド 核酸送達のための新規脂質及び脂質ナノ粒子組成物
EP4630057A1 (en) 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
CN116270534B (zh) * 2022-12-13 2024-11-26 山东大学 一种组织选择性递送核酸药物的类脂质纳米颗粒及其制备方法和应用
EP4385523A1 (en) * 2022-12-14 2024-06-19 Beijing Jitai Pharmaceutical Technology Co., Ltd. Lipid-based topical injection formulations
CN116211831A (zh) * 2022-12-14 2023-06-06 北京剂泰医药科技有限公司 脂质基质局部注射制剂
CN121263401A (zh) 2022-12-22 2026-01-02 内诺维什疗法股份有限公司 用于递送核酸和其他治疗剂的含硫的可电离脂质
CN116199646B (zh) * 2023-03-07 2024-01-26 山东大学 一种基于Tris的可电离脂质及其制备方法与应用
CN116271105B (zh) * 2023-05-18 2023-08-25 上海贝斯昂科生物科技有限公司 一种适于rpe细胞转染的脂质纳米颗粒及其应用
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025029927A1 (en) * 2023-07-31 2025-02-06 The Board Of Regents Of The University Of Texas System Lipid nanoparticle
CN121752254A (zh) 2023-08-11 2026-03-27 浙江海昶生物医药股份有限公司 一种具有靶向性的脂质组合物及其制备方法
WO2025073054A1 (en) 2023-10-05 2025-04-10 Nanovation Therapeutics Inc. Method of delivery of dna or rna cargo using unshielded lipid nanoparticles
WO2025073056A1 (en) * 2023-10-05 2025-04-10 Nanovation Therapeutics Inc. Haematopoietic stem and/or progenitor cell delivery method and compositions for use thereof
WO2025081189A1 (en) * 2023-10-12 2025-04-17 Georgia Tech Research Corporation Methods and compositions for lipid nanoparticle (lnp) mediated delivery of mrna encoded synthetic antigens for universal tumor targeting by car t cells
WO2025088597A1 (en) * 2023-10-26 2025-05-01 New Phase Ltd. Nanoparticle combination therapy for tumors
WO2025108545A1 (en) * 2023-11-22 2025-05-30 Danmarks Tekniske Universitet Delivery of nucleotides from injectable biomaterial
US12553042B2 (en) * 2023-12-01 2026-02-17 Recode Therapeutics, Inc. Method for quantifying an amount of capped messenger RNA
US12364773B2 (en) * 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
WO2025133684A1 (en) * 2023-12-21 2025-06-26 Vilnius University Liposomal nanoparticles for crispr/cas9 delivery
CN120398741A (zh) * 2024-01-29 2025-08-01 江苏中天药业有限公司 一种用于递送核酸物质的脂肪酸衍生物及其合成方法
CN118001254B (zh) 2024-04-09 2024-07-26 北京悦康科创医药科技股份有限公司 靶向抗原呈递细胞的脂质组合物及其应用
WO2025213271A1 (en) * 2024-04-11 2025-10-16 Nanovation Therapeutics Inc. Method of delivering nucleic acid to macrophages and/or monocytes and compositions for use thereof
WO2025226642A1 (en) * 2024-04-25 2025-10-30 Eli Lilly And Company Lipid nano delivery particles for targeted transport of nucleic acids and methods of making and using the same
WO2025228975A1 (en) * 2024-04-29 2025-11-06 BioNTech SE Particles, compositions and methods
WO2025228979A1 (en) 2024-04-30 2025-11-06 Neovac Ltd. Lipids nanoparticle formulations suitable for lung targeting
WO2026025110A1 (en) * 2024-07-26 2026-01-29 Trevi Therapeutics, Inc. Treatment of refractory chronic cough
CN118750468B (zh) * 2024-09-05 2025-02-18 北京悦康科创医药科技股份有限公司 一种核酸-脂质纳米颗粒冻干组合物及其制备方法和应用
KR20260043575A (ko) * 2024-09-24 2026-03-31 주식회사 녹십자 선택적 장기 표적 지질을 포함하는 조성물
CN121610533B (zh) * 2026-02-03 2026-04-17 中国海洋大学三亚海洋研究院 用于鱼类在体皮肤基因编辑的脂质纳米颗粒及其应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014523411A (ja) 2011-06-08 2014-09-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法
JP2015519346A (ja) 2012-05-23 2015-07-09 ジ・オハイオ・ステート・ユニバーシティ アンチセンスオリゴヌクレオチドを送達するための脂質ナノ粒子組成物

Family Cites Families (64)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
AU733310C (en) 1997-05-14 2001-11-29 University Of British Columbia, The High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
US7314956B2 (en) 2001-08-08 2008-01-01 Vaxim, Inc. Multifunctional carrier for the delivery of a pharmacological agent or genetic material into a cell
US8871709B2 (en) * 2003-02-05 2014-10-28 Queensland University of Technolgy Synthetic chimeric proteins comprising epidermal growth factor and vitronectin
US7985424B2 (en) 2004-04-20 2011-07-26 Dendritic Nanotechnologies Inc. Dendritic polymers with enhanced amplification and interior functionality
MXPA06012722A (es) 2004-05-05 2007-02-14 Atugen Ag Lipidos, complejos de lipido y su uso.
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2006138380A2 (en) 2005-06-15 2006-12-28 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
US20090221684A1 (en) 2005-12-22 2009-09-03 Trustees Of Boston University Molecules for Gene Delivery and Gene Therapy, and Methods of Use Thereof
EP2046266A4 (en) 2006-07-21 2009-11-04 Massachusetts Inst Technology ENDMODICIFIED POLY (BETA AMINO ESTER) AND ITS USE
CA2710983A1 (en) * 2007-12-27 2009-10-01 The Ohio State University Research Foundation Lipid nanoparticle compositions and methods of making and using the same
WO2009111088A2 (en) 2008-01-02 2009-09-11 The Johns Hopkins University Antitumor immunization by liposomal delivery of vaccine to the spleen
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US8163861B2 (en) 2009-01-15 2012-04-24 Living Proof, Inc. Beta-amino ester compounds and uses thereof
CA3045126A1 (en) 2009-05-05 2010-11-11 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
BRPI1010175A2 (pt) 2009-06-02 2016-03-29 Keller Brian Charles "composição química que inclui um conjugado de lipídio-peg e lipossoma"
CN101591428B (zh) 2009-06-29 2011-05-25 浙江大学 一种聚酯树枝状大分子的制备方法
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
EP2618817B1 (en) * 2010-07-31 2018-06-13 The Scripps Research Institute Liposome targeting compounds and related uses
WO2012090223A1 (en) 2010-12-29 2012-07-05 Indian Institute Of Science Poly(ether imine) dendrimers and uses thereof
EP2718261B1 (en) 2011-06-08 2016-02-24 Nitto Denko Corporation Compounds for targeting drug delivery and enhancing sirna activity
CN107266391B (zh) 2011-10-18 2020-04-17 迪克纳制药公司 胺阳离子脂质及其用途
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US20160289674A1 (en) * 2012-04-02 2016-10-06 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
JP2015526434A (ja) 2012-08-14 2015-09-10 アンジオケム インコーポレーテッド ペプチド−デンドリマーコンジュゲート及びその使用
JP6241193B2 (ja) 2012-10-23 2017-12-06 コニカミノルタ株式会社 透明電極、電子デバイス及び有機エレクトロルミネッセンス素子
JP6118914B2 (ja) * 2012-12-28 2017-04-19 ターベダ セラピューティクス インコーポレイテッドＴａｒｖｅｄａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 放出制御型の固体ポリマーナノ粒子
US20140186332A1 (en) * 2012-12-28 2014-07-03 NX Pharmagen Biomarkers of preterm birth
US9820942B2 (en) 2013-03-06 2017-11-21 Biomics Biotechnologies Co., Ltd. Lipidosome preparation, preparation method and application thereof
RS57739B1 (sr) * 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
EP3388834B1 (en) 2013-03-15 2020-04-15 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
CN106413811A (zh) 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
US20170143848A1 (en) 2014-03-24 2017-05-25 Shire Human Genetic Therapies, Inc. Mrna therapy for the treatment of ocular diseases
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
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
EP3155116A4 (en) * 2014-06-10 2017-12-27 Massachusetts Institute Of Technology Method for gene editing
CN103999853B (zh) 2014-06-11 2015-07-22 中国农业大学 荧光树枝状纳米大分子在制备药物载体中的应用
CN106794141B (zh) * 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
CA2964234A1 (en) 2014-10-10 2016-04-14 Massachusetts Eye And Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ， インコーポレイテッド 関節疾患の治療のためのメッセンジャーｒｎａ治療法
WO2016094342A1 (en) 2014-12-08 2016-06-16 The Board Of Regents Of The University Of Texas System Lipocationic polymers and uses thereof
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
EP3247398A4 (en) * 2015-01-23 2018-09-26 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
US20180200186A1 (en) * 2015-07-09 2018-07-19 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
WO2017048789A1 (en) * 2015-09-14 2017-03-23 The Board Of Regents Of The University Of Texas System Lipocationic dendrimers and uses thereof
EP3353309A4 (en) 2015-09-25 2019-04-10 Tarveda Therapeutics, Inc. COMPOSITIONS AND METHOD FOR GENERIC EDITING
WO2017066793A1 (en) * 2015-10-16 2017-04-20 Modernatx, Inc. Mrna cap analogs and methods of mrna capping
JP7384512B2 (ja) * 2015-10-22 2023-11-21 モデルナティエックス インコーポレイテッド 広域インフルエンザウイルスワクチン
LT3436077T (lt) * 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2017180917A2 (en) * 2016-04-13 2017-10-19 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
PL3458074T3 (pl) 2016-05-16 2024-11-12 Board Of Regents Of The University Of Texas System KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA
SI3458083T1 (sl) * 2016-05-18 2023-03-31 Modernatx, Inc. Polinukleotidi, ki kodirajo interlevkin-12 (IL12) in njihova uporaba
CN118108847A (zh) * 2016-08-07 2024-05-31 诺华股份有限公司 mRNA介导的免疫方法
CA3040337A1 (en) * 2016-10-26 2018-05-03 Curevac Ag Lipid nanoparticle mrna vaccines
EP3315125A1 (en) * 2016-10-31 2018-05-02 Silence Therapeutics (London) Ltd Lipid nanoparticle formulation
CA3103528A1 (en) * 2018-06-19 2019-12-26 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mrna and long nucleic acids
WO2020051223A1 (en) 2018-09-04 2020-03-12 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
EP3846857A4 (en) 2018-09-04 2022-10-12 The Board Of Regents Of The University Of Texas System COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014523411A (ja) 2011-06-08 2014-09-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法
JP2015519346A (ja) 2012-05-23 2015-07-09 ジ・オハイオ・ステート・ユニバーシティ アンチセンスオリゴヌクレオチドを送達するための脂質ナノ粒子組成物
JP2015525209A (ja) 2012-05-23 2015-09-03 ジ・オハイオ・ステート・ユニバーシティ 脂質ナノ粒子組成物と、それを作製する方法、及びそれを使用する方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Advanced Materials，2017年，Vol.29, No.33，Article ID 1606944, p.1-7

Also Published As

Publication number Publication date
AU2025223878A1 (en) 2025-09-18
AU2019336679A1 (en) 2021-03-25
US20220133637A1 (en) 2022-05-05
US20230026843A1 (en) 2023-01-26
GB2606038B (en) 2023-05-03
US20210401762A1 (en) 2021-12-30
JP2021535227A (ja) 2021-12-16
GB202111271D0 (en) 2021-09-15
US11229609B2 (en) 2022-01-25
US11648209B2 (en) 2023-05-16
EP3846822A4 (en) 2022-07-06
GB2592505B (en) 2023-05-03
JP2024016183A (ja) 2024-02-06
US20220387337A1 (en) 2022-12-08
CN112996519B (zh) 2025-02-28
EP3846822A1 (en) 2021-07-14
US20240245620A1 (en) 2024-07-25
US11648210B2 (en) 2023-05-16
US11304911B2 (en) 2022-04-19
CA3111484A1 (en) 2020-03-12
US12133924B2 (en) 2024-11-05
CN112996519A (zh) 2021-06-18
US20240148666A1 (en) 2024-05-09
GB2617430A (en) 2023-10-11
GB2617430B (en) 2023-12-27
GB202219703D0 (en) 2023-02-08
CN120093931A (zh) 2025-06-06
GB2592505A (en) 2021-09-01
WO2020051223A1 (en) 2020-03-12
GB2606038A (en) 2022-10-26
JP7815196B2 (ja) 2026-02-17
US11510880B2 (en) 2022-11-29
AU2019336679B2 (en) 2025-05-29
US20250228788A1 (en) 2025-07-17
US20210259980A1 (en) 2021-08-26
US20220233460A1 (en) 2022-07-28
GB202104777D0 (en) 2021-05-19
US11590085B2 (en) 2023-02-28

Similar Documents

Publication Publication Date Title
JP7815196B2 (ja) 核酸を臓器特異的送達するための組成物および方法
US11766408B2 (en) Compositions and methods for organ specific delivery of nucleic acids
AU2022244355A1 (en) Compositions and methods for targeted systemic delivery to cells
HK40055120B (zh) 用於核酸的器官特异性递送的组合物和方法
HK40055120A (en) Compositions and methods for organ specific delivery of nucleic acids
HK40055907A (en) Compositions and methods for organ specific delivery of nucleic acids

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220905

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220905

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20220905

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221109

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230207

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230509

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230710

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231107

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231115

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20231205

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: 20231227

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20240118

R150 Certificate of patent or registration of utility model

Ref document number: 7425066

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150