CN116940324A - 脂质纳米颗粒球形核酸 - Google Patents

脂质纳米颗粒球形核酸 Download PDF

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Publication number
CN116940324A
CN116940324A CN202280012579.6A CN202280012579A CN116940324A CN 116940324 A CN116940324 A CN 116940324A CN 202280012579 A CN202280012579 A CN 202280012579A CN 116940324 A CN116940324 A CN 116940324A
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CN
China
Prior art keywords
lnp
sna
oligonucleotide
lipid
toll
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Pending
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CN202280012579.6A
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English (en)
Chinese (zh)
Inventor
查德·A·米尔金
安德鲁·约瑟夫·西内格拉
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NORTHWEST UNIVERSITY
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NORTHWEST UNIVERSITY
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Publication of CN116940324A publication Critical patent/CN116940324A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
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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
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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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
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    • 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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    • A61B90/37—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
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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61B90/39—Markers, e.g. radio-opaque or breast lesions markers
    • A61B2090/3966—Radiopaque markers visible in an X-ray image
    • A—HUMAN NECESSITIES
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    • A61B90/50—Supports for surgical instruments, e.g. articulated arms
    • A61B2090/502—Headgear, e.g. helmet, spectacles
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • C12N2310/122—Hairpin
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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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  • Medicinal Chemistry (AREA)
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  • General Engineering & Computer Science (AREA)
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  • 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)
CN202280012579.6A 2021-01-12 2022-01-11 脂质纳米颗粒球形核酸 Pending CN116940324A (zh)

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
CN116940324A true CN116940324A (zh) 2023-10-24

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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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* Cited by examiner, † Cited by third party
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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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US20080020058A1 (en) * 2005-02-14 2008-01-24 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
WO2018067302A2 (en) * 2016-09-19 2018-04-12 North Western University Therapeutic effects of cellular delivery of small molecules and macromolecules with liposomal spherical nucleic acids
US20180214376A1 (en) * 2015-07-14 2018-08-02 Exicure, Inc. Spherical nucleic acid (sna)-mediated delivery of lipid-complexes to cells
US20190314291A1 (en) * 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
US20190336452A1 (en) * 2016-11-08 2019-11-07 Modernatx, Inc. Stabilized formulations of lipid nanoparticles

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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
US20200248183A1 (en) * 2017-04-03 2020-08-06 Subbarao Nallagatla Tlr9-targeted spherical nucleic acids having potent antitumor activity
MA49421A (fr) * 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
MX2020000387A (es) * 2017-07-13 2020-08-17 Univ Northwestern Método general y directo para preparar nanopartículas de estructura organometálica funcionalizadas con oligonucleotidos.
WO2019118883A1 (en) * 2017-12-15 2019-06-20 Northwestern University Structure-function relationships in the development of immunotherapeutic agents
WO2020168005A1 (en) * 2019-02-12 2020-08-20 Exicure, Inc. Combined spherical nucleic acid and checkpoint inhibitor for antitumor therapy
US20220362398A1 (en) * 2019-04-26 2022-11-17 Exicure Operating Company Administration of spherical nucleic acids for ophthalmological uses

Patent Citations (6)

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Publication number Priority date Publication date Assignee Title
US20080020058A1 (en) * 2005-02-14 2008-01-24 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
CN106536729A (zh) * 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
US20180214376A1 (en) * 2015-07-14 2018-08-02 Exicure, Inc. Spherical nucleic acid (sna)-mediated delivery of lipid-complexes to cells
WO2018067302A2 (en) * 2016-09-19 2018-04-12 North Western University Therapeutic effects of cellular delivery of small molecules and macromolecules with liposomal spherical nucleic acids
US20190336452A1 (en) * 2016-11-08 2019-11-07 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
US20190314291A1 (en) * 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells

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JP2024505151A (ja) 2024-02-05
EP4277590A1 (en) 2023-11-22
EP4277590A4 (en) 2026-01-28
US20240309367A1 (en) 2024-09-19

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