JP2021528407A - 組合せ遺伝子治療薬 - Google Patents

組合せ遺伝子治療薬 Download PDF

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JP2021528407A
JP2021528407A JP2020570467A JP2020570467A JP2021528407A JP 2021528407 A JP2021528407 A JP 2021528407A JP 2020570467 A JP2020570467 A JP 2020570467A JP 2020570467 A JP2020570467 A JP 2020570467A JP 2021528407 A JP2021528407 A JP 2021528407A
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transgene
vector
based vector
therapeutic agent
cells
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Japanese (ja)
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JP2021528407A5 (enExample
Inventor
サミル・エル・アンダロッシ
ペル・ルンディン
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エヴォックス・セラピューティクス・リミテッド
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    • 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
    • A61K48/0083—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 administration regime
    • 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
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0069—Oxidoreductases (1.) acting on single donors with incorporation of molecular oxygen, i.e. oxygenases (1.13)
    • 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
    • C12N2509/00—Methods for the dissociation of cells, e.g. specific use of enzymes
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Manufacturing & Machinery (AREA)
  • Developmental Biology & Embryology (AREA)
  • Rheumatology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2020570467A 2018-06-22 2019-06-21 組合せ遺伝子治療薬 Pending JP2021528407A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1810301.0 2018-06-22
GBGB1810301.0A GB201810301D0 (en) 2018-06-22 2018-06-22 Combinatorial gene therapy
PCT/EP2019/066471 WO2019243574A1 (en) 2018-06-22 2019-06-21 Combinatorial gene therapy

Publications (2)

Publication Number Publication Date
JP2021528407A true JP2021528407A (ja) 2021-10-21
JP2021528407A5 JP2021528407A5 (enExample) 2022-06-24

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ID=63042788

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020570467A Pending JP2021528407A (ja) 2018-06-22 2019-06-21 組合せ遺伝子治療薬

Country Status (14)

Country Link
US (1) US20210252164A1 (enExample)
EP (1) EP3810206B1 (enExample)
JP (1) JP2021528407A (enExample)
KR (1) KR20210024040A (enExample)
CN (1) CN112955185A (enExample)
AU (1) AU2019289998A1 (enExample)
BR (1) BR112020026260A2 (enExample)
CA (1) CA3104432A1 (enExample)
GB (1) GB201810301D0 (enExample)
IL (1) IL279584A (enExample)
MX (1) MX2020013865A (enExample)
SG (1) SG11202012727SA (enExample)
WO (1) WO2019243574A1 (enExample)
ZA (1) ZA202100244B (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3226019A1 (en) 2021-07-20 2023-01-26 Ags Therapeutics Sas Extracellular vesicles from microalgae, their preparation, and uses
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
JP2025535944A (ja) 2022-10-24 2025-10-30 エイジーエス・セラピューティクス・ソシエテ・パール・アクシオン・サンプリフィエ 微細藻類由来の細胞外小胞、鼻腔内投与時のそれらの生体内分布、及びそれらの使用
WO2025176843A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014513928A (ja) * 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法
WO2015002956A1 (en) * 2013-07-01 2015-01-08 Ohio State Innovation Foundation Exosome delivery system
JP2017526388A (ja) * 2014-09-05 2017-09-14 エクサーカイン コーポレイションＥｘｅｒｋｉｎｅ Ｃｏｒｐｏｒａｔｉｏｎ エキソソームの単離
WO2017172606A1 (en) * 2016-03-28 2017-10-05 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability
JP2021507682A (ja) * 2017-11-08 2021-02-25 エヴォックス・セラピューティクス・リミテッド Ｒｎａ治療薬を含むエクソソーム

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002008288A2 (en) * 2000-07-20 2002-01-31 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
CN102813942A (zh) * 2012-08-15 2012-12-12 钟志容 脂质超声微泡介导的腺相关病毒基因转染制剂及制备工艺
EP3090053B1 (en) * 2013-12-30 2018-11-21 CureVac AG Artificial nucleic acid molecules
WO2016044947A1 (en) * 2014-09-26 2016-03-31 Exerkine Corporation Exosomes useful to treat lysosomal storage disease

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014513928A (ja) * 2011-03-11 2014-06-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 無カプシドａａｖベクター、組成物ならびにベクター製造および遺伝子運搬のための方法
WO2015002956A1 (en) * 2013-07-01 2015-01-08 Ohio State Innovation Foundation Exosome delivery system
JP2017526388A (ja) * 2014-09-05 2017-09-14 エクサーカイン コーポレイションＥｘｅｒｋｉｎｅ Ｃｏｒｐｏｒａｔｉｏｎ エキソソームの単離
WO2017172606A1 (en) * 2016-03-28 2017-10-05 The Regents Of The University Of California Method and composition for treating neuronal hyper-excitability
JP2021507682A (ja) * 2017-11-08 2021-02-25 エヴォックス・セラピューティクス・リミテッド Ｒｎａ治療薬を含むエクソソーム

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
CELL MOL NEUROBIOL., vol. 36, JPN6023024462, 2016, pages 417427, ISSN: 0005268664 *
MOL THER: METHODS & CLINICAL DEVELOPMENT, vol. 9, JPN6023024463, June 2018 (2018-06-01), pages 278 - 287, ISSN: 0005268663 *

Also Published As

Publication number Publication date
GB201810301D0 (en) 2018-08-08
CA3104432A1 (en) 2019-12-26
MX2020013865A (es) 2021-03-25
WO2019243574A1 (en) 2019-12-26
EP3810206B1 (en) 2024-01-24
KR20210024040A (ko) 2021-03-04
IL279584A (en) 2021-03-01
US20210252164A1 (en) 2021-08-19
AU2019289998A1 (en) 2021-02-04
CN112955185A (zh) 2021-06-11
SG11202012727SA (en) 2021-01-28
EP3810206A1 (en) 2021-04-28
ZA202100244B (en) 2022-07-27
BR112020026260A2 (pt) 2021-03-23

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