JP7737312B2 - 遺伝子治療のための改変型アデノ随伴ウイルス （ａａｖ） 粒子 - Google Patents

遺伝子治療のための改変型アデノ随伴ウイルス （ａａｖ） 粒子

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Publication number
JP7737312B2
JP7737312B2 JP2021566142A JP2021566142A JP7737312B2 JP 7737312 B2 JP7737312 B2 JP 7737312B2 JP 2021566142 A JP2021566142 A JP 2021566142A JP 2021566142 A JP2021566142 A JP 2021566142A JP 7737312 B2 JP7737312 B2 JP 7737312B2
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aav
ligand
binding site
disease
capsid
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JPWO2020225363A5 (https=
JP2022531892A5 (https=
JP2022531892A (ja
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ポール ヘッペンスタール
マリアーノ マッフェイ
カストロ レイス フェルナンダ デ
カニン モリス パウー
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Europaisches Laboratorium fuer Molekularbiologie EMBL
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Europaisches Laboratorium fuer Molekularbiologie EMBL
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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
    • 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
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14161—Methods of inactivation or attenuation
    • C12N2750/14162—Methods of inactivation or attenuation by genetic engineering

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JP2021566142A 2019-05-08 2020-05-07 遺伝子治療のための改変型アデノ随伴ウイルス （ａａｖ） 粒子 Active JP7737312B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025077923A JP2025121993A (ja) 2019-05-08 2025-05-08 遺伝子治療のための改変型アデノ随伴ウイルス （ａａｖ） 粒子

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19173365.8 2019-05-08
EP19173365.8A EP3736330A1 (en) 2019-05-08 2019-05-08 Modified adeno-associated virus (aav) particles for gene therapy
PCT/EP2020/062713 WO2020225363A2 (en) 2019-05-08 2020-05-07 Modified adeno-associated virus (aav) particles for gene therapy

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JP2025077923A Division JP2025121993A (ja) 2019-05-08 2025-05-08 遺伝子治療のための改変型アデノ随伴ウイルス （ａａｖ） 粒子

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JP2022531892A JP2022531892A (ja) 2022-07-12
JPWO2020225363A5 JPWO2020225363A5 (https=) 2023-05-15
JP2022531892A5 JP2022531892A5 (https=) 2023-05-15
JP7737312B2 true JP7737312B2 (ja) 2025-09-10

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JP2025077923A Withdrawn JP2025121993A (ja) 2019-05-08 2025-05-08 遺伝子治療のための改変型アデノ随伴ウイルス （ａａｖ） 粒子

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US (1) US20220288234A1 (https=)
EP (2) EP3736330A1 (https=)
JP (2) JP7737312B2 (https=)
KR (1) KR20220008863A (https=)
CN (1) CN114008194A (https=)
AU (1) AU2020268759A1 (https=)
BR (1) BR112021022385A2 (https=)
CA (1) CA3139183A1 (https=)
IL (1) IL287614A (https=)
MX (1) MX2021013619A (https=)
SG (1) SG11202112029XA (https=)
WO (1) WO2020225363A2 (https=)

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CA3201533A1 (en) 2020-11-11 2022-05-19 European Molecular Biology Laboratory Modified viral particles for gene therapy
CN118109515A (zh) * 2022-11-29 2024-05-31 中国科学院深圳先进技术研究院 一种基于生物正交技术的病毒载体及其制备方法和应用
CN121889172A (zh) 2023-07-21 2026-04-17 意大利商博雷亚医疗有限责任公司 制备表面修饰病毒衣壳的方法

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WO2018035503A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system

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KR102437522B1 (ko) 2012-05-25 2022-08-26 셀렉티스 면역요법을 위한 동종이형 및 면역억제제 저항성인 ｔ 세포의 조작 방법
KR102234672B1 (ko) * 2013-05-21 2021-04-06 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
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BR112021022385A2 (pt) 2022-03-22
WO2020225363A2 (en) 2020-11-12
CN114008194A (zh) 2022-02-01
AU2020268759A1 (en) 2021-12-16
IL287614A (en) 2021-12-01
SG11202112029XA (en) 2021-11-29
WO2020225363A3 (en) 2020-12-17
MX2021013619A (es) 2022-02-22
EP3966319A2 (en) 2022-03-16
CA3139183A1 (en) 2020-11-12
JP2022531892A (ja) 2022-07-12
JP2025121993A (ja) 2025-08-20
US20220288234A1 (en) 2022-09-15
KR20220008863A (ko) 2022-01-21
EP3736330A1 (en) 2020-11-11

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