KR20250004778A - mRNA 제제의 엑소좀-매개 전달을 위한 조성물 및 방법 - Google Patents

mRNA 제제의 엑소좀-매개 전달을 위한 조성물 및 방법 Download PDF

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Publication number
KR20250004778A
KR20250004778A KR1020247037694A KR20247037694A KR20250004778A KR 20250004778 A KR20250004778 A KR 20250004778A KR 1020247037694 A KR1020247037694 A KR 1020247037694A KR 20247037694 A KR20247037694 A KR 20247037694A KR 20250004778 A KR20250004778 A KR 20250004778A
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South Korea
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mrna
cells
cell
human
mrna preparation
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Korean (ko)
Inventor
케네스 치엔
존 폴 셸
랜 양
에두아르데 로너
안나 마리아니
예스퍼 솔머
카일리 푸
카일리 푸
네빈 위트먼
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스마트셀라 솔루션즈 아베
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Publication of KR20250004778A publication Critical patent/KR20250004778A/ko
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    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • 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
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1858—Platelet-derived growth factor [PDGF]
    • A61K38/1866—Vascular endothelial growth factor [VEGF]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
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    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
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    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/555—Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511—Organic adjuvants
    • A61K2039/55555—Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/65—MicroRNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0662—Stem cells
    • C12N5/0668—Mesenchymal stem cells from other natural sources

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Wood Science & Technology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Organic Chemistry (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Vascular Medicine (AREA)
  • Mycology (AREA)
  • General Engineering & Computer Science (AREA)
  • Cell Biology (AREA)
  • Rheumatology (AREA)
  • Botany (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020247037694A 2022-04-15 2023-04-13 mRNA 제제의 엑소좀-매개 전달을 위한 조성물 및 방법 Pending KR20250004778A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263331532P 2022-04-15 2022-04-15
US63/331,532 2022-04-15
PCT/IB2023/000204 WO2023199113A1 (en) 2022-04-15 2023-04-13 COMPOSITIONS AND METHODS FOR EXOSOME-MEDIATED DELIVERY OF mRNA AGENTS

Publications (1)

Publication Number Publication Date
KR20250004778A true KR20250004778A (ko) 2025-01-08

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KR1020247037694A Pending KR20250004778A (ko) 2022-04-15 2023-04-13 mRNA 제제의 엑소좀-매개 전달을 위한 조성물 및 방법

Country Status (8)

Country Link
US (1) US20230330264A1 (https=)
EP (1) EP4507701A1 (https=)
JP (1) JP2025512066A (https=)
KR (1) KR20250004778A (https=)
AU (1) AU2023254488A1 (https=)
CA (1) CA3247667A1 (https=)
IL (1) IL316370A (https=)
WO (1) WO2023199113A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026017857A1 (en) 2024-07-18 2026-01-22 Smartcella Solutions Ab Treatment of osteoarthritis and/or rheumatoid arthritis with pro-chondrogenic and/or chondrocyte protective factors
WO2026017864A1 (en) 2024-07-18 2026-01-22 Smartcella Solutions Ab Treatment of osteoarthritis and/or rheumatoid arthritis with pro-chondrogenic and/or chondrocyte protective factors

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5486359A (en) 1990-11-16 1996-01-23 Osiris Therapeutics, Inc. Human mesenchymal stem cells
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
US5994619A (en) 1996-04-01 1999-11-30 University Of Massachusetts, A Public Institution Of Higher Education Of The Commonwealth Of Massachusetts, As Represented By Its Amherst Campus Production of chimeric bovine or porcine animals using cultured inner cell mass cells
US5945577A (en) 1997-01-10 1999-08-31 University Of Massachusetts As Represented By Its Amherst Campus Cloning using donor nuclei from proliferating somatic cells
WO2003062404A1 (en) 2002-01-25 2003-07-31 Gamida-Cell Ltd. Methods of expanding stem and progenitor cells and expanded cell populations obtained thereby
EP2291213B1 (en) 2008-04-08 2015-08-19 SmartWise Sweden AB Endoluminal medical access device
WO2012004165A1 (en) 2010-07-08 2012-01-12 Karolinska Institutet Innovations Ab Novel endoluminal medical access device
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
WO2013103659A1 (en) 2012-01-04 2013-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Stabilizing rna by incorporating chain-terminating nucleosides at the 3'-terminus
SG11201408717XA (en) 2012-06-26 2015-02-27 Seraxis Inc Stem cells and pancreatic cells useful for the treatment of insulin-dependent diabetes mellitus
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
CN120796168A (zh) 2013-06-11 2025-10-17 哈佛学院校长同事会 SC-β细胞以及用于产生其的组合物和方法
MA40589A (fr) 2014-08-22 2017-06-28 Procella Therapeutics Ab Utilisation de jagged 1/frizzled 4 à titre de marqueurs de surface cellulaire pour isoler des cellules progénitrices ventriculaires cardiaques humaines
US10596200B2 (en) 2014-08-22 2020-03-24 Procella Therapeutics Ab Use of LIFR or FGFR3 as a cell surface marker for isolating human cardiac ventricular progenitor cells
WO2016100921A1 (en) 2014-12-18 2016-06-23 President And Fellows Of Harvard College METHODS FOR GENERATING STEM CELL-DERIVED β CELLS AND USES THEREOF
CN107614678B (zh) 2014-12-18 2021-04-30 哈佛学院校长同事会 干细胞来源的β细胞的产生方法及其使用方法
WO2016100898A1 (en) 2014-12-18 2016-06-23 President And Fellows Of Harvard College Serum-free in vitro directed differentiation protocol for generating stem cell-derived b cells and uses thereof
WO2016100925A1 (en) 2014-12-18 2016-06-23 President And Fellows Of Harvard College METHODS FOR GENERATING AUGMENTED STEM CELL-DERIVED β CELLS AND USES THEREOF
US11160834B2 (en) * 2015-04-28 2021-11-02 The Texas A&M University System Scalable production of standardized extracellular vesicles, extracellular vesicle preparations and uses thereof
EP3291822A1 (en) 2015-05-05 2018-03-14 Katholieke Universiteit Leuven Improved methods for pancreatic islet transplantation
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
WO2017075708A1 (en) * 2015-11-05 2017-05-11 Exerkine Corporation Exosomes useful to treat cystic fibrosis
US10508263B2 (en) 2016-11-29 2019-12-17 Procella Therapeutics Ab Methods for isolating human cardiac ventricular progenitor cells
WO2019038587A1 (en) 2017-08-23 2019-02-28 Procella Therapeutics Ab USE OF NEUROPILIN-1 (NRP1) AS A CELL SURFACE MARKER FOR ISOLATING HUMAN CARDIAC VENTRICULAR PROGENITOR CELLS

Also Published As

Publication number Publication date
CA3247667A1 (en) 2023-10-19
IL316370A (en) 2024-12-01
JP2025512066A (ja) 2025-04-16
AU2023254488A1 (en) 2024-12-05
EP4507701A1 (en) 2025-02-19
US20230330264A1 (en) 2023-10-19
WO2023199113A1 (en) 2023-10-19

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