JP7783628B2 - 多層rnaナノ粒子 - Google Patents

多層rnaナノ粒子

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Publication number
JP7783628B2
JP7783628B2 JP2022503898A JP2022503898A JP7783628B2 JP 7783628 B2 JP7783628 B2 JP 7783628B2 JP 2022503898 A JP2022503898 A JP 2022503898A JP 2022503898 A JP2022503898 A JP 2022503898A JP 7783628 B2 JP7783628 B2 JP 7783628B2
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Prior art keywords
rna
tumor
pharmaceutical composition
nanoparticle
nanoparticles
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JP2022503898A
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English (en)
Japanese (ja)
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JP2022541586A (ja
Inventor
セイヤー、エリアス
メンデス-ゴメス、ヘクター、ルーベン
ミッチェル、デュエイン
リナルディ、カルロス
Original Assignee
ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
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Publication of JP2022541586A publication Critical patent/JP2022541586A/ja
Priority to JP2025089202A priority Critical patent/JP2025138644A/ja
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/39Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0033Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53DNA (RNA) vaccination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dispersion Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022503898A 2019-07-19 2020-07-17 多層rnaナノ粒子 Active JP7783628B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025089202A JP2025138644A (ja) 2019-07-19 2025-05-28 多層rnaナノ粒子

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962876440P 2019-07-19 2019-07-19
US62/876,440 2019-07-19
US201962877598P 2019-07-23 2019-07-23
US62/877,598 2019-07-23
US201962884983P 2019-08-09 2019-08-09
US62/884,983 2019-08-09
US201962933326P 2019-11-08 2019-11-08
US62/933,326 2019-11-08
PCT/US2020/042606 WO2021016106A1 (en) 2019-07-19 2020-07-17 Multilamellar rna nanoparticles

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025089202A Division JP2025138644A (ja) 2019-07-19 2025-05-28 多層rnaナノ粒子

Publications (2)

Publication Number Publication Date
JP2022541586A JP2022541586A (ja) 2022-09-26
JP7783628B2 true JP7783628B2 (ja) 2025-12-10

Family

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Family Applications (2)

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JP2022503898A Active JP7783628B2 (ja) 2019-07-19 2020-07-17 多層rnaナノ粒子
JP2025089202A Pending JP2025138644A (ja) 2019-07-19 2025-05-28 多層rnaナノ粒子

Family Applications After (1)

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JP2025089202A Pending JP2025138644A (ja) 2019-07-19 2025-05-28 多層rnaナノ粒子

Country Status (8)

Country Link
US (1) US20220287969A1 (https=)
EP (1) EP3999034A4 (https=)
JP (2) JP7783628B2 (https=)
KR (1) KR20220035434A (https=)
AU (1) AU2020316335B2 (https=)
BR (1) BR112022000925A2 (https=)
CA (1) CA3144388A1 (https=)
WO (1) WO2021016106A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230346700A1 (en) * 2020-02-19 2023-11-02 University Of Florida Research Foundation, Incorporated Multilamellar RNA Nanoparticles and Methods of Sensitizing Tumors to Treatment with Immune Checkpoint Inhibitors
CA3174187A1 (en) * 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Multilamellar rna nanoparticle vaccine against sars-cov-2
JP2024517287A (ja) * 2021-05-07 2024-04-19 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド Car t細胞療法の方法
WO2024263713A1 (en) * 2023-06-21 2024-12-26 University Of Florida Research Foundation, Incorporated Materials and methods of using proinsulin mrna nanoparticles

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016537304A (ja) 2013-09-26 2016-12-01 バイオンテック アーゲー Rnaによるシェルを含む粒子
JP2017520549A (ja) 2014-06-26 2017-07-27 ラモット アット テル アビブ ユニバーシティ, リミテッド 核酸の送達のためのリポソーム製剤

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WO2011112597A1 (en) * 2010-03-08 2011-09-15 Northeastern University Magnetic nanoplatforms for theranostic and multi-modal imaging applications
PL3586861T3 (pl) * 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
US10245229B2 (en) * 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
ITRM20120480A1 (it) * 2012-10-09 2014-04-10 Uni Degli Studi Camerino Nanoparticelle lipidiche multicomponenti e procedimenti per la loro preparazione.
WO2017062502A1 (en) * 2015-10-05 2017-04-13 The Regents Of The University Of Colorgo, A Body Corporate Lipoplexes formulated for catalytic delivery
CA3099519A1 (en) * 2018-05-08 2019-11-14 University Of Florida Research Foundation, Incorporated Magnetic liposomes and related treatment and imaging methods
WO2020037102A1 (en) * 2018-08-15 2020-02-20 University Of Florida Research Foundation, Inc. Methods of sensitizing tumors to treatment with immune checkpoint inhibitors
EP3849597A1 (en) * 2018-09-12 2021-07-21 University of Florida Research Foundation, Inc. Slow-cycling cell-rna based nanoparticle vaccine to treat cancer
EP4099988A4 (en) * 2020-02-05 2024-03-13 University of Florida Research Foundation, Incorporated Rna-loaded nanoparticles and use thereof for the treatment of cancer
US20230346700A1 (en) * 2020-02-19 2023-11-02 University Of Florida Research Foundation, Incorporated Multilamellar RNA Nanoparticles and Methods of Sensitizing Tumors to Treatment with Immune Checkpoint Inhibitors
CA3174187A1 (en) * 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Multilamellar rna nanoparticle vaccine against sars-cov-2
WO2022140278A1 (en) * 2020-12-21 2022-06-30 University Of Florida Research Foundation, Inc. Multilamellar rna nanoparticle vaccine against cancer
JP2024517287A (ja) * 2021-05-07 2024-04-19 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド Car t細胞療法の方法
WO2023196232A1 (en) * 2022-04-04 2023-10-12 University Of Florida Research Foundation, Inc. Method of characterizing tumors

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JP2016537304A (ja) 2013-09-26 2016-12-01 バイオンテック アーゲー Rnaによるシェルを含む粒子
JP2017520549A (ja) 2014-06-26 2017-07-27 ラモット アット テル アビブ ユニバーシティ, リミテッド 核酸の送達のためのリポソーム製剤

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Also Published As

Publication number Publication date
CA3144388A1 (en) 2021-01-28
JP2022541586A (ja) 2022-09-26
EP3999034A4 (en) 2023-08-23
BR112022000925A2 (pt) 2022-05-17
KR20220035434A (ko) 2022-03-22
WO2021016106A8 (en) 2022-02-10
AU2020316335A1 (en) 2022-02-17
AU2020316335B2 (en) 2026-04-02
JP2025138644A (ja) 2025-09-25
US20220287969A1 (en) 2022-09-15
EP3999034A1 (en) 2022-05-25
WO2021016106A1 (en) 2021-01-28

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