CN116390934A - 功能性aav衣壳的高通量工程化 - Google Patents

功能性aav衣壳的高通量工程化 Download PDF

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Publication number
CN116390934A
CN116390934A CN202180058369.6A CN202180058369A CN116390934A CN 116390934 A CN116390934 A CN 116390934A CN 202180058369 A CN202180058369 A CN 202180058369A CN 116390934 A CN116390934 A CN 116390934A
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seq
capsid polypeptide
capsid
amino acid
aav
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CN202180058369.6A
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Chinese (zh)
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弗朗索瓦·维格纳尔特
托马斯·帕卡德
大卫·杰弗里·赫斯
凯文·克里斯多佛·斯坦
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Shape Therapy Co
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Shape Therapy Co
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    • 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
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    • C40B40/02—Libraries contained in or displayed by microorganisms, e.g. bacteria or animal cells; Libraries contained in or displayed by vectors, e.g. plasmids; Libraries containing only microorganisms or vectors
    • 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
    • A61K48/0008—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
    • A61K48/0041—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 the non-active part being polymeric
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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
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    • 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
    • C12N2750/14123—Virus like particles [VLP]
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    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule

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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202180058369.6A 2020-05-26 2021-05-26 功能性aav衣壳的高通量工程化 Pending CN116390934A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202063030038P 2020-05-26 2020-05-26
US63/030,038 2020-05-26
US202063119554P 2020-11-30 2020-11-30
US63/119,554 2020-11-30
US202163134885P 2021-01-07 2021-01-07
US63/134,885 2021-01-07
US202163181037P 2021-04-28 2021-04-28
US63/181,037 2021-04-28
PCT/US2021/034329 WO2021242909A1 (en) 2020-05-26 2021-05-26 High throughput engineering of functional aav capsids

Publications (1)

Publication Number Publication Date
CN116390934A true CN116390934A (zh) 2023-07-04

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CN202180058369.6A Pending CN116390934A (zh) 2020-05-26 2021-05-26 功能性aav衣壳的高通量工程化

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US (2) US20230048732A1 (https=)
EP (1) EP4158037A1 (https=)
JP (1) JP2023529315A (https=)
CN (1) CN116390934A (https=)
AU (1) AU2021282257A1 (https=)
CA (1) CA3173450A1 (https=)
WO (1) WO2021242909A1 (https=)

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CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CA3249551A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
WO2023152229A1 (en) 2022-02-09 2023-08-17 Teitur Trophics Aps Novel peptides
CA3259902A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. AAV Capsid Variants and Their Uses
US20250388626A1 (en) 2022-07-06 2025-12-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4626906A1 (en) * 2022-11-29 2025-10-08 Vacino Biotech Co., Ltd. Transporter peptides and application thereof
CN118307648A (zh) * 2023-01-09 2024-07-09 广东克冠达医药科技有限公司 腺相关病毒衣壳蛋白衍生多肽及其用途
EP4410988A1 (en) * 2023-01-31 2024-08-07 Universitätsklinikum Hamburg-Eppendorf An aav2-vector variant for targeted transfer of genes
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202523846A (zh) 2023-08-16 2025-06-16 美商航海家醫療公司 用於治療與共濟蛋白(frataxin)不足有關之疾病之組合物及方法
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025038795A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
EP3003391B1 (en) * 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2015048534A1 (en) * 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3200830B1 (en) * 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
SG11201808812RA (en) * 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
JP2020534788A (ja) * 2017-08-28 2020-12-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルスカプシド変異体及びその使用方法
EP3833745A1 (en) 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production

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US20260002177A1 (en) 2026-01-01
EP4158037A1 (en) 2023-04-05
WO2021242909A1 (en) 2021-12-02
US20230048732A1 (en) 2023-02-16
JP2023529315A (ja) 2023-07-10
AU2021282257A1 (en) 2022-12-01
CA3173450A1 (en) 2021-12-02

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