WO2018200419A1 - Vecteurs viraux comprenant des capsides d'aav modifié et compositions les contenant - Google Patents

Vecteurs viraux comprenant des capsides d'aav modifié et compositions les contenant Download PDF

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Publication number
WO2018200419A1
WO2018200419A1 PCT/US2018/028954 US2018028954W WO2018200419A1 WO 2018200419 A1 WO2018200419 A1 WO 2018200419A1 US 2018028954 W US2018028954 W US 2018028954W WO 2018200419 A1 WO2018200419 A1 WO 2018200419A1
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WO
WIPO (PCT)
Prior art keywords
hvr
aav
seq
capsid
aav3b
Prior art date
Application number
PCT/US2018/028954
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English (en)
Inventor
James M. Wilson
Qiang Wang
Original Assignee
The Trustees Of The University Of Pennsylvania
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Publication date
Application filed by The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of WO2018200419A1 publication Critical patent/WO2018200419A1/fr

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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
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La présente invention concerne une capside d'AAV artificiel permettant l'administration d'un génome à vecteur AAV portant un transgène présentant une aptitude à la translation améliorée entre espèces. La capside d'AAV artificiel est également utile dans la génération d'AAV recombinant la comprenant. L'invention concerne également des méthodes d'augmentation de la transduction de rAAV chez une espèce et d'administration de molécules thérapeutiques et autres à celle-ci.
PCT/US2018/028954 2017-04-23 2018-04-23 Vecteurs viraux comprenant des capsides d'aav modifié et compositions les contenant WO2018200419A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762488808P 2017-04-23 2017-04-23
US62/488,808 2017-04-23

Publications (1)

Publication Number Publication Date
WO2018200419A1 true WO2018200419A1 (fr) 2018-11-01

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ID=63919123

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/028954 WO2018200419A1 (fr) 2017-04-23 2018-04-23 Vecteurs viraux comprenant des capsides d'aav modifié et compositions les contenant

Country Status (1)

Country Link
WO (1) WO2018200419A1 (fr)

Cited By (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10414803B2 (en) 2015-05-11 2019-09-17 Ucl Business Plc Capsid
WO2021009805A1 (fr) * 2019-07-12 2021-01-21 株式会社遺伝子治療研究所 Virion de virus adéno-associé pour transfert de gènes vers le foie humain
WO2021041489A1 (fr) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Vecteurs viraux adéno-associés pour traverser la barrière hémato-encéphalique humaine
WO2021080991A1 (fr) * 2019-10-21 2021-04-29 The Trustees Of The University Of Pennsylvania Variants d'aav3b présentant un rendement de production et un tropisme hépatique améliorés
WO2021108468A1 (fr) * 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Nouveaux variants aav3b ciblant des hépatocytes humains dans le foie de souris humanisées
WO2021108467A1 (fr) * 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Nouveaux variants d'aav3b ciblant des hépatocytes humains et échappant à la réponse immunitaire humorale
CN113853209A (zh) * 2019-04-29 2021-12-28 宾夕法尼亚州大学信托人 新型aav衣壳和含有其的组合物
WO2022003211A1 (fr) * 2020-07-03 2022-01-06 Genethon Procédé d'ingénierie de nouveaux capsides aav hybrides par l'intermédiaire d'une permutation de régions hypervariables
WO2022165313A1 (fr) 2021-02-01 2022-08-04 Regenxbio Inc. Thérapie génique de céroïdes-lipofuscinoses neuronales
WO2022229703A3 (fr) * 2021-04-30 2022-12-15 Takeda Pharmaceutical Company, Ltd. Nouveaux variants de fuite immunitaire à base d'aav8
WO2023004416A1 (fr) * 2021-07-23 2023-01-26 California Institute Of Technology Vecteurs viraux pour administration améliorée induite par ultrasons au cerveau
EP3999120A4 (fr) * 2019-07-04 2024-02-21 Childrens Medical Res Institute Procédés et vecteurs de virus adéno-associé pour transduction in vivo
WO2024079249A1 (fr) * 2022-10-12 2024-04-18 Genethon Vecteur aav hybride améliorant l'expression d'un transgène dans le foie

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016065001A1 (fr) * 2014-10-21 2016-04-28 University Of Massachusetts Variants de vaa recombinants et leurs utilisations
WO2017015102A1 (fr) * 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions et procédés pour obtenir des niveaux élevés de transduction dans des cellules hépatiques humaines
US20170096683A1 (en) * 2014-05-02 2017-04-06 Genzyme Corporation Aav vectors for retinal and cns gene therapy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170096683A1 (en) * 2014-05-02 2017-04-06 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2016065001A1 (fr) * 2014-10-21 2016-04-28 University Of Massachusetts Variants de vaa recombinants et leurs utilisations
WO2017015102A1 (fr) * 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions et procédés pour obtenir des niveaux élevés de transduction dans des cellules hépatiques humaines

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GURDA, BL ET AL.: "Capsid Antibodies to Different Adeno-Associated Virus Serotypes Bind Common Regions", JOURNAL OF VIROLOGY, vol. 87, no. 16, 12 June 2013 (2013-06-12), pages 9111 - 9124, XP055534980 *

Cited By (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10414803B2 (en) 2015-05-11 2019-09-17 Ucl Business Plc Capsid
CN113853209A (zh) * 2019-04-29 2021-12-28 宾夕法尼亚州大学信托人 新型aav衣壳和含有其的组合物
EP3999120A4 (fr) * 2019-07-04 2024-02-21 Childrens Medical Res Institute Procédés et vecteurs de virus adéno-associé pour transduction in vivo
WO2021009805A1 (fr) * 2019-07-12 2021-01-21 株式会社遺伝子治療研究所 Virion de virus adéno-associé pour transfert de gènes vers le foie humain
JP7371954B2 (ja) 2019-07-12 2023-10-31 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
CN115087738A (zh) * 2019-08-29 2022-09-20 小利兰·斯坦福大学托管委员会 用于穿越人血脑屏障的腺相关病毒载体
WO2021041489A1 (fr) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Vecteurs viraux adéno-associés pour traverser la barrière hémato-encéphalique humaine
WO2021080991A1 (fr) * 2019-10-21 2021-04-29 The Trustees Of The University Of Pennsylvania Variants d'aav3b présentant un rendement de production et un tropisme hépatique améliorés
EP4048681A4 (fr) * 2019-10-21 2024-03-20 Univ Pennsylvania Variants d'aav3b présentant un rendement de production et un tropisme hépatique améliorés
WO2021108468A1 (fr) * 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Nouveaux variants aav3b ciblant des hépatocytes humains dans le foie de souris humanisées
WO2021108467A1 (fr) * 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Nouveaux variants d'aav3b ciblant des hépatocytes humains et échappant à la réponse immunitaire humorale
WO2022003211A1 (fr) * 2020-07-03 2022-01-06 Genethon Procédé d'ingénierie de nouveaux capsides aav hybrides par l'intermédiaire d'une permutation de régions hypervariables
WO2022165313A1 (fr) 2021-02-01 2022-08-04 Regenxbio Inc. Thérapie génique de céroïdes-lipofuscinoses neuronales
WO2022229703A3 (fr) * 2021-04-30 2022-12-15 Takeda Pharmaceutical Company, Ltd. Nouveaux variants de fuite immunitaire à base d'aav8
WO2023004416A1 (fr) * 2021-07-23 2023-01-26 California Institute Of Technology Vecteurs viraux pour administration améliorée induite par ultrasons au cerveau
WO2024079249A1 (fr) * 2022-10-12 2024-04-18 Genethon Vecteur aav hybride améliorant l'expression d'un transgène dans le foie

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