JP2024505151A - 脂質ナノ粒子球状核酸 - Google Patents

脂質ナノ粒子球状核酸 Download PDF

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Publication number
JP2024505151A
JP2024505151A JP2023541965A JP2023541965A JP2024505151A JP 2024505151 A JP2024505151 A JP 2024505151A JP 2023541965 A JP2023541965 A JP 2023541965A JP 2023541965 A JP2023541965 A JP 2023541965A JP 2024505151 A JP2024505151 A JP 2024505151A
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lnp
sna
oligonucleotide
lipid
toll
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JP2023541965A
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Japanese (ja)
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JP2024505151A5 (https=
Inventor
エー．マーキン チャド
ジョセフ シングラ アンドリュー
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ノースウェスタン ユニバーシティ
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • 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
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    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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
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    • A61B90/36—Image-producing devices or illumination devices not otherwise provided for
    • A61B2090/364—Correlation of different images or relation of image positions in respect to the body
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    • A61B90/36—Image-producing devices or illumination devices not otherwise provided for
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    • A61B2090/371—Surgical systems with images on a monitor during operation with simultaneous use of two cameras
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61B90/36—Image-producing devices or illumination devices not otherwise provided for
    • A61B90/37—Surgical systems with images on a monitor during operation
    • A61B2090/376—Surgical systems with images on a monitor during operation using X-rays, e.g. fluoroscopy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61B90/36—Image-producing devices or illumination devices not otherwise provided for
    • A61B90/37—Surgical systems with images on a monitor during operation
    • A61B2090/378—Surgical systems with images on a monitor during operation using ultrasound
    • A—HUMAN NECESSITIES
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    • A61B90/00—Instruments, implements or accessories specially adapted for surgery or diagnosis and not covered by any of the groups A61B1/00 - A61B50/00, e.g. for luxation treatment or for protecting wound edges
    • A61B90/39—Markers, e.g. radio-opaque or breast lesions markers
    • A61B2090/3966—Radiopaque markers visible in an X-ray image
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61B90/00—Instruments, implements or accessories specially adapted for surgery or diagnosis and not covered by any of the groups A61B1/00 - A61B50/00, e.g. for luxation treatment or for protecting wound edges
    • A61B90/50—Supports for surgical instruments, e.g. articulated arms
    • A61B2090/502—Headgear, e.g. helmet, spectacles
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • General Engineering & Computer Science (AREA)
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  • Biotechnology (AREA)
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  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Biophysics (AREA)
  • Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dispersion Chemistry (AREA)
  • Medical Informatics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Robotics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023541965A 2021-01-12 2022-01-11 脂質ナノ粒子球状核酸 Pending JP2024505151A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163136501P 2021-01-12 2021-01-12
US63/136,501 2021-01-12
PCT/US2022/012023 WO2022155149A1 (en) 2021-01-12 2022-01-11 Lipid nanoparticle spherical nucleic acids

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Publication Number Publication Date
JP2024505151A true JP2024505151A (ja) 2024-02-05
JP2024505151A5 JP2024505151A5 (https=) 2025-01-21

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JP2023541965A Pending JP2024505151A (ja) 2021-01-12 2022-01-11 脂質ナノ粒子球状核酸

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US (1) US20240309367A1 (https=)
EP (1) EP4277590A4 (https=)
JP (1) JP2024505151A (https=)
CN (1) CN116940324A (https=)
WO (1) WO2022155149A1 (https=)

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US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
KR20250112318A (ko) * 2022-10-27 2025-07-23 씨울프 테라퓨틱스, 인코포레이티드 핵 표적 dna 전달 및 이를 실시하는데 사용하기 위한 조성물
EP4630061A1 (en) * 2022-12-08 2025-10-15 Nanovation Therapeutics Inc. Lipid nanoparticles comprising elevated neutral lipid and a targeting moiety for targeted delivery of nucleic acid
WO2026006618A1 (en) * 2024-06-26 2026-01-02 Northwestern University Lipid nanoparticle spherical nucleic acids for genome engineering

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WO2017011662A1 (en) * 2015-07-14 2017-01-19 Exicure, Inc. Spherical nucleic acid (sna)-mediated delivery of lipid-complexes to cells
JP2020526558A (ja) * 2017-07-13 2020-08-31 ノースウェスタン ユニバーシティ オリゴヌクレオチド官能化金属有機構造体ナノ粒子を調製するための一般的かつ直接的な方法
WO2020219985A1 (en) * 2019-04-26 2020-10-29 Exicure, Inc. Administration of spherical nucleic acids for ophthalmological uses

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IT1397011B1 (it) * 2009-10-14 2012-12-20 Univ Ferrara Nanoparticella del tipo core-shell idonea per la veicolazione di oligonucleotidi terapeutici in tessuti bersaglio e suo impiego per la preparazione di un medicamento per il trattamento della distrofia muscolare di duchenne.
EP2860255A1 (en) * 2013-10-14 2015-04-15 Technische Universität Graz Compositions comprising cationic and neutral lipids for transfecting nucleic acid molecules into eukaryotic cells
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JP2020526558A (ja) * 2017-07-13 2020-08-31 ノースウェスタン ユニバーシティ オリゴヌクレオチド官能化金属有機構造体ナノ粒子を調製するための一般的かつ直接的な方法
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WO2022155149A1 (en) 2022-07-21
CN116940324A (zh) 2023-10-24
EP4277590A1 (en) 2023-11-22
EP4277590A4 (en) 2026-01-28
US20240309367A1 (en) 2024-09-19

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