CN116249772A - 具有变体衣壳的腺相关病毒病毒体及其使用方法 - Google Patents

具有变体衣壳的腺相关病毒病毒体及其使用方法 Download PDF

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Publication number
CN116249772A
CN116249772A CN202180041153.9A CN202180041153A CN116249772A CN 116249772 A CN116249772 A CN 116249772A CN 202180041153 A CN202180041153 A CN 202180041153A CN 116249772 A CN116249772 A CN 116249772A
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amino acids
ala
thr
gly
ser
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Chinese (zh)
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J·G·弗兰纳里
S·F·基勒
K·I·盖林
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Novartis AG
University of California San Diego UCSD
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Novartis AG
University of California San Diego UCSD
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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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
    • C12N2750/14122—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
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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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202180041153.9A 2020-05-29 2021-05-27 具有变体衣壳的腺相关病毒病毒体及其使用方法 Pending CN116249772A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063032206P 2020-05-29 2020-05-29
US63/032,206 2020-05-29
US202163187154P 2021-05-11 2021-05-11
US63/187,154 2021-05-11
PCT/US2021/034624 WO2021243085A2 (en) 2020-05-29 2021-05-27 Adeno-associated virus virions with variant capsids and methods of use thereof

Publications (1)

Publication Number Publication Date
CN116249772A true CN116249772A (zh) 2023-06-09

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CN202180041153.9A Pending CN116249772A (zh) 2020-05-29 2021-05-27 具有变体衣壳的腺相关病毒病毒体及其使用方法

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US (1) US12031147B2 (enExample)
EP (1) EP4157299A4 (enExample)
JP (1) JP2023528801A (enExample)
KR (1) KR20230043794A (enExample)
CN (1) CN116249772A (enExample)
AU (1) AU2021280317A1 (enExample)
CA (1) CA3179597A1 (enExample)
MX (1) MX2022015048A (enExample)
WO (1) WO2021243085A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118955654A (zh) * 2024-10-15 2024-11-15 朗信启昇(苏州)生物制药有限公司 具有变异衣壳的腺相关病毒病毒体及其用途

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WO2022150776A1 (en) 2021-01-11 2022-07-14 Vedere Bio Ii, Inc. OPTOGENETIC COMPOSITIONS COMPRISING A CBh PROMOTER SEQUENCE AND METHODS FOR USE
AU2022358779A1 (en) 2021-10-08 2024-04-18 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
AU2024234833A1 (en) 2023-03-10 2025-10-23 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN118791624A (zh) * 2023-04-11 2024-10-18 苏州星明优健生物技术有限公司 用于治疗视网膜神经退行性疾病的新型gpcr嵌合视蛋白
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (2)

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Publication number Priority date Publication date Assignee Title
CN110709511A (zh) * 2017-08-28 2020-01-17 加利福尼亚大学董事会 腺相关病毒衣壳变体及其使用方法
US20200121746A1 (en) * 2017-06-30 2020-04-23 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof

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US8663624B2 (en) * 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
SMT202400520T1 (it) 2011-04-22 2025-01-14 Univ California Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo
CA2978068C (en) * 2015-03-02 2024-05-28 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP6836999B2 (ja) 2015-03-24 2021-03-03 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Ｔｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
EP4209501A1 (en) * 2016-05-13 2023-07-12 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
CA3040179A1 (en) * 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
WO2018134168A1 (en) * 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
UY39772A (es) * 2021-05-21 2023-01-31 Novartis Ag Composiciones y métodos para mejorar la función visual

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200121746A1 (en) * 2017-06-30 2020-04-23 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
CN110709511A (zh) * 2017-08-28 2020-01-17 加利福尼亚大学董事会 腺相关病毒衣壳变体及其使用方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DAY, TIMOTHY PAUL: "Expanding the Potential of Adeno-Associated Virus for the Treatment of Intractable Inherited Retinal Degenerations", pages 16 - 28, Retrieved from the Internet <URL:https://escholarship.org/uc/item/3846w1bs> *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118955654A (zh) * 2024-10-15 2024-11-15 朗信启昇(苏州)生物制药有限公司 具有变异衣壳的腺相关病毒病毒体及其用途

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WO2021243085A2 (en) 2021-12-02
KR20230043794A (ko) 2023-03-31
MX2022015048A (es) 2023-03-08
EP4157299A4 (en) 2024-10-02
AU2021280317A1 (en) 2023-01-05
JP2023528801A (ja) 2023-07-06
US20210371879A1 (en) 2021-12-02
US12031147B2 (en) 2024-07-09
WO2021243085A3 (en) 2022-01-06
EP4157299A2 (en) 2023-04-05
CA3179597A1 (en) 2021-12-02

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