KR20220035434A - 다중층 rna 나노입자 - Google Patents

다중층 rna 나노입자 Download PDF

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Publication number
KR20220035434A
KR20220035434A KR1020227004840A KR20227004840A KR20220035434A KR 20220035434 A KR20220035434 A KR 20220035434A KR 1020227004840 A KR1020227004840 A KR 1020227004840A KR 20227004840 A KR20227004840 A KR 20227004840A KR 20220035434 A KR20220035434 A KR 20220035434A
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Prior art keywords
rna
tumor
clause
cells
nanoparticles
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Pending
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KR1020227004840A
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English (en)
Korean (ko)
Inventor
엘리아스 세이유어
헥토르 루벤 멘데즈-고메즈
듀에인 미첼
카를로스 리날디
Original Assignee
유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드
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Publication of KR20220035434A publication Critical patent/KR20220035434A/ko
Pending legal-status Critical Current

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    • 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
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-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
    • 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
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/39—Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
    • 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
    • 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
    • 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
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Dispersion Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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)
KR1020227004840A 2019-07-19 2020-07-17 다중층 rna 나노입자 Pending KR20220035434A (ko)

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

Publications (1)

Publication Number Publication Date
KR20220035434A true KR20220035434A (ko) 2022-03-22

Family

ID=74193752

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227004840A Pending KR20220035434A (ko) 2019-07-19 2020-07-17 다중층 rna 나노입자

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
EP4106730A4 (en) * 2020-02-19 2024-03-27 University of Florida Research Foundation, Incorporated MULTILAMELLAR RNA NANOPARTICLES AND METHODS FOR SENSITIZING TUMORS TO TREATMENT WITH IMMUNE CHECKPOINT INHIBITORS
EP4126030A1 (en) * 2020-04-01 2023-02-08 University Of Florida Research Foundation, Incorporated Multilamellar rna nanoparticle vaccine against sars-cov-2
US20240238418A1 (en) * 2021-05-07 2024-07-18 University Of Florida Research Foundation, Inc. Car t cell therapy method
WO2024263713A1 (en) * 2023-06-21 2024-12-26 University Of Florida Research Foundation, Incorporated Materials and methods of using proinsulin mrna nanoparticles

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130323165A1 (en) * 2010-03-08 2013-12-05 Northeastern University Magnetic Nanoplatforms for Theranostic and Multi-Modal Imaging Applications
RU2013154295A (ru) * 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
EP2858679B2 (en) * 2012-06-08 2024-06-05 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.
US9950065B2 (en) * 2013-09-26 2018-04-24 Biontech Rna Pharmaceuticals Gmbh Particles comprising a shell with RNA
EP3160448B1 (en) * 2014-06-26 2025-08-06 Ramot at Tel-Aviv University Ltd. Liposomal formulations for delivery of nucleic acids
WO2017062502A1 (en) * 2015-10-05 2017-04-13 The Regents Of The University Of Colorgo, A Body Corporate Lipoplexes formulated for catalytic delivery
EP3790533A1 (en) * 2018-05-08 2021-03-17 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
US20220054610A1 (en) * 2018-09-12 2022-02-24 University Of Florida Research Foundation, Inc. Slow-cycling cell-rna based nanoparticle vaccine to treat cancer
US20230096704A1 (en) * 2020-02-05 2023-03-30 University Of Florida Research Foundation, Incorporated Rna-loaded nanoparticles and use thereof for the treatment of cancer
EP4106730A4 (en) * 2020-02-19 2024-03-27 University of Florida Research Foundation, Incorporated MULTILAMELLAR RNA NANOPARTICLES AND METHODS FOR SENSITIZING TUMORS TO TREATMENT WITH IMMUNE CHECKPOINT INHIBITORS
EP4126030A1 (en) * 2020-04-01 2023-02-08 University Of Florida Research Foundation, Incorporated Multilamellar rna nanoparticle vaccine against sars-cov-2
US20240299516A1 (en) * 2020-12-21 2024-09-12 University Of Florida Research Foundaton, Inc. Multilamellar rna nanoparticle vaccine against cancer
US20240238418A1 (en) * 2021-05-07 2024-07-18 University Of Florida Research Foundation, Inc. Car t cell therapy method
WO2023196232A1 (en) * 2022-04-04 2023-10-12 University Of Florida Research Foundation, Inc. Method of characterizing tumors

Also Published As

Publication number Publication date
JP7783628B2 (ja) 2025-12-10
BR112022000925A2 (pt) 2022-05-17
JP2022541586A (ja) 2022-09-26
JP2025138644A (ja) 2025-09-25
WO2021016106A1 (en) 2021-01-28
AU2020316335A1 (en) 2022-02-17
US20220287969A1 (en) 2022-09-15
EP3999034A1 (en) 2022-05-25
AU2020316335B2 (en) 2026-04-02
CA3144388A1 (en) 2021-01-28
WO2021016106A8 (en) 2022-02-10
EP3999034A4 (en) 2023-08-23

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