KR20220008863A - 유전자 치료를 위한 변형된 아데노-연관 바이러스(aav) 입자들 - Google Patents

유전자 치료를 위한 변형된 아데노-연관 바이러스(aav) 입자들 Download PDF

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Publication number
KR20220008863A
KR20220008863A KR1020217040160A KR20217040160A KR20220008863A KR 20220008863 A KR20220008863 A KR 20220008863A KR 1020217040160 A KR1020217040160 A KR 1020217040160A KR 20217040160 A KR20217040160 A KR 20217040160A KR 20220008863 A KR20220008863 A KR 20220008863A
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South Korea
Prior art keywords
aav
binding site
disease
capsid
group
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KR1020217040160A
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English (en)
Korean (ko)
Inventor
폴 헵펜스톨
마리아노 마페이
페르난다 데 카스트로 레이스
카닌 모리스 포우
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유럽피안 몰레큘러 바이올로지 래보러토리
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Publication of KR20220008863A publication Critical patent/KR20220008863A/ko
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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
    • A61K48/0008—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
    • 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
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    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • 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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KR1020217040160A 2019-05-08 2020-05-07 유전자 치료를 위한 변형된 아데노-연관 바이러스(aav) 입자들 Pending KR20220008863A (ko)

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

Publications (1)

Publication Number Publication Date
KR20220008863A true KR20220008863A (ko) 2022-01-21

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Application Number Title Priority Date Filing Date
KR1020217040160A Pending KR20220008863A (ko) 2019-05-08 2020-05-07 유전자 치료를 위한 변형된 아데노-연관 바이러스(aav) 입자들

Country Status (12)

Country Link
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=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (16)

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Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
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
WO2014191128A1 (en) 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
CN104592364B (zh) * 2013-10-30 2018-05-01 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
US10179176B2 (en) 2016-02-16 2019-01-15 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
EP3448875A4 (en) * 2016-04-29 2020-04-08 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
FI3445773T3 (fi) * 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
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
MX2020000244A (es) * 2017-06-27 2020-09-28 Regeneron Pharma Vectores virales recombinantes con tropismo modificado y usos de estos para la introduccion dirigida de material genetico a celulas humanas.
EP3461836A1 (en) * 2017-09-28 2019-04-03 Universität zu Köln Mutated adeno-associated viral capsid proteins for chemical coupling of ligands, nanoparticles or drugs via thioether binding and production method thereof

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Publication number Publication date
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
JP7737312B2 (ja) 2025-09-10
JP2022531892A (ja) 2022-07-12
JP2025121993A (ja) 2025-08-20
US20220288234A1 (en) 2022-09-15
EP3736330A1 (en) 2020-11-11

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