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 (https=
JPWO2019243574A5 (https=
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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Publication of JP2021528407A5 publication Critical patent/JP2021528407A5/ja
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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)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (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 (3)

Publication Number Publication Date
JP2021528407A true JP2021528407A (ja) 2021-10-21
JP2021528407A5 JP2021528407A5 (https=) 2022-06-24
JPWO2019243574A5 JPWO2019243574A5 (https=) 2022-06-24

Family

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

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
CN121038801A (zh) 2022-10-24 2025-11-28 Ags治疗简易股份公司 来自微藻的细胞外囊泡、它们在鼻内施用时的生物分布及其用途
WO2025176847A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Ocular delivery of active agents via microalgae extracellular vesicles

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
AU2001273150A1 (en) * 2000-07-20 2002-02-05 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
EP3495486B1 (en) * 2013-12-30 2020-12-16 CureVac AG Artificial nucleic acid molecules
CA2962081A1 (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
CA3104432A1 (en) 2019-12-26
EP3810206B1 (en) 2024-01-24
MX2020013865A (es) 2021-03-25
CN112955185A (zh) 2021-06-11
EP3810206A1 (en) 2021-04-28
BR112020026260A2 (pt) 2021-03-23
US20210252164A1 (en) 2021-08-19
GB201810301D0 (en) 2018-08-08
SG11202012727SA (en) 2021-01-28
AU2019289998A1 (en) 2021-02-04
KR20210024040A (ko) 2021-03-04
WO2019243574A1 (en) 2019-12-26
IL279584A (en) 2021-03-01
ZA202100244B (en) 2022-07-27

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