JP2023529054A - Ａａｖキャプシドバリアントおよびその使用 - Google Patents

Ａａｖキャプシドバリアントおよびその使用 Download PDF

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Publication number
JP2023529054A
JP2023529054A JP2022559694A JP2022559694A JP2023529054A JP 2023529054 A JP2023529054 A JP 2023529054A JP 2022559694 A JP2022559694 A JP 2022559694A JP 2022559694 A JP2022559694 A JP 2022559694A JP 2023529054 A JP2023529054 A JP 2023529054A
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mir
hsa
aavv66
aav
raav
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Japanese (ja)
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JP2023529054A5 (https=
JPWO2021202494A5 (https=
Inventor
グアンピン ガオ，
グアンチャオ シュー，
フィリップ タイ，
ユーチュアン ウェイ，
リー ルオ，
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ユニバーシティ オブ マサチューセッツ
スーチュワン ユニバーシティ
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Publication of JP2023529054A publication Critical patent/JP2023529054A/ja
Publication of JP2023529054A5 publication Critical patent/JP2023529054A5/ja
Publication of JPWO2021202494A5 publication Critical patent/JPWO2021202494A5/ja
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    • 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
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2022559694A 2020-03-31 2021-03-30 Ａａｖキャプシドバリアントおよびその使用 Pending JP2023529054A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063003143P 2020-03-31 2020-03-31
US63/003,143 2020-03-31
PCT/US2021/024837 WO2021202494A1 (en) 2020-03-31 2021-03-30 Aav capsids variants and uses thereof

Publications (3)

Publication Number Publication Date
JP2023529054A true JP2023529054A (ja) 2023-07-07
JP2023529054A5 JP2023529054A5 (https=) 2024-04-10
JPWO2021202494A5 JPWO2021202494A5 (https=) 2024-04-10

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ID=75870702

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JP2022559694A Pending JP2023529054A (ja) 2020-03-31 2021-03-30 Ａａｖキャプシドバリアントおよびその使用

Country Status (14)

Country Link
US (1) US20230138766A1 (https=)
EP (1) EP4126911A1 (https=)
JP (1) JP2023529054A (https=)
KR (1) KR20230113689A (https=)
CN (1) CN115698039A (https=)
AR (1) AR122404A1 (https=)
AU (1) AU2021248577A1 (https=)
BR (1) BR112022019304A2 (https=)
CA (1) CA3177182A1 (https=)
CO (1) CO2022015313A2 (https=)
IL (1) IL296765A (https=)
MX (1) MX2022012279A (https=)
TW (1) TW202204377A (https=)
WO (1) WO2021202494A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
AU2019237514A1 (en) 2018-03-23 2020-10-08 University Of Massachusetts Gene therapeutics for treating bone disorders
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
WO2024020376A1 (en) * 2022-07-22 2024-01-25 University Of Massachusetts Aav-mediated delivery of osteoblast/osteoclast-regulating mirnas for osteoporosis therapy

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019530463A (ja) * 2016-10-13 2019-10-24 ユニバーシティ オブ マサチューセッツ Ａａｖカプシド設計

Family Cites Families (24)

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US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
EP3134522B1 (en) 2014-04-25 2021-10-06 University of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
US20200316221A1 (en) * 2016-10-13 2020-10-08 University Of Massachusetts Aav capsid designs

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019530463A (ja) * 2016-10-13 2019-10-24 ユニバーシティ オブ マサチューセッツ Ａａｖカプシド設計

Also Published As

Publication number Publication date
EP4126911A1 (en) 2023-02-08
KR20230113689A (ko) 2023-08-01
CA3177182A1 (en) 2021-10-07
US20230138766A1 (en) 2023-05-04
IL296765A (en) 2022-11-01
TW202204377A (zh) 2022-02-01
CO2022015313A2 (es) 2023-02-16
WO2021202494A1 (en) 2021-10-07
MX2022012279A (es) 2023-02-23
AR122404A1 (es) 2022-09-07
BR112022019304A2 (pt) 2022-12-06
CN115698039A (zh) 2023-02-03
AU2021248577A1 (en) 2022-09-29

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