JP2021514659A - Ａａｖキメラ - Google Patents

Ａａｖキメラ Download PDF

Info

Publication number
JP2021514659A
JP2021514659A JP2020546347A JP2020546347A JP2021514659A JP 2021514659 A JP2021514659 A JP 2021514659A JP 2020546347 A JP2020546347 A JP 2020546347A JP 2020546347 A JP2020546347 A JP 2020546347A JP 2021514659 A JP2021514659 A JP 2021514659A
Authority
JP
Japan
Prior art keywords
serotype
domains
serotypes
aav2
rep
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2020546347A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021514659A5 (https=
Inventor
メイヴィス アグバンジェ−マッケンナ，
メイヴィス アグバンジェ−マッケンナ，
マリオ メイチュ，
マリオ メイチュ，
ロバート マッケンナ，
ロバート マッケンナ，
Original Assignee
ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド
ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド, ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド filed Critical ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド
Publication of JP2021514659A publication Critical patent/JP2021514659A/ja
Publication of JP2021514659A5 publication Critical patent/JP2021514659A5/ja
Priority to JP2023175237A priority Critical patent/JP2023171525A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/861—Adenoviral 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2750/14161—Methods of inactivation or attenuation

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020546347A 2018-03-06 2019-03-06 Ａａｖキメラ Pending JP2021514659A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023175237A JP2023171525A (ja) 2018-03-06 2023-10-10 Ａａｖキメラ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862639466P 2018-03-06 2018-03-06
US62/639,466 2018-03-06
PCT/US2019/021048 WO2019173538A1 (en) 2018-03-06 2019-03-06 Aav chimeras

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023175237A Division JP2023171525A (ja) 2018-03-06 2023-10-10 Ａａｖキメラ

Publications (2)

Publication Number Publication Date
JP2021514659A true JP2021514659A (ja) 2021-06-17
JP2021514659A5 JP2021514659A5 (https=) 2022-03-14

Family

ID=67846340

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020546347A Pending JP2021514659A (ja) 2018-03-06 2019-03-06 Ａａｖキメラ
JP2023175237A Pending JP2023171525A (ja) 2018-03-06 2023-10-10 Ａａｖキメラ

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023175237A Pending JP2023171525A (ja) 2018-03-06 2023-10-10 Ａａｖキメラ

Country Status (8)

Country Link
US (2) US11905524B2 (https=)
EP (1) EP3762501A4 (https=)
JP (2) JP2021514659A (https=)
KR (1) KR20200130337A (https=)
CN (1) CN112166195A (https=)
AU (1) AU2019231711A1 (https=)
CA (1) CA3092871A1 (https=)
WO (1) WO2019173538A1 (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024143440A1 (ja) 2022-12-28 2024-07-04 富士フイルム株式会社 アデノ随伴ウイルスの製造方法、およびベクターのセット
WO2024172027A1 (ja) * 2023-02-17 2024-08-22 株式会社シンプロジェン ＩＴＲ－Ｒｅｐ遺伝子複合体

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
CA3092871A1 (en) 2018-03-06 2019-09-12 University Of Florida Research Foundation, Incorporated Aav chimeras
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
US12054738B2 (en) 2020-07-30 2024-08-06 Shape Therapeutics Inc. Stable cell lines for inducible production of rAAV virions
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CN116622908B (zh) * 2023-04-13 2024-02-06 武汉珈创生物技术股份有限公司 快速检测野生型腺相关病毒的引物探针、试剂盒及方法和应用
WO2025194042A1 (en) * 2024-03-14 2025-09-18 Affinia Therapeutics Inc. Plasmid system for production of recombinant adenoassociated virus
WO2026022064A1 (en) 2024-07-22 2026-01-29 F. Hoffmann-La Roche Ag Novel aav rep orfs and rep polypeptides

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003104392A2 (en) * 2001-12-18 2003-12-18 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
WO2011088081A1 (en) * 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1015619A1 (en) * 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
AU780231B2 (en) * 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6723551B2 (en) * 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
CN1461805A (zh) 2002-05-27 2003-12-17 本元正阳基因技术股份有限公司 一组重组单纯疱疹病毒的构建及其用途
CN101724608A (zh) 2008-10-10 2010-06-09 中国疾病预防控制中心病毒病预防控制所 用于重组aav2/8病毒载体生产的重组i型单纯疱疹病毒及其用途
WO2017100674A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
CA3092871A1 (en) 2018-03-06 2019-09-12 University Of Florida Research Foundation, Incorporated Aav chimeras

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003104392A2 (en) * 2001-12-18 2003-12-18 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
WO2011088081A1 (en) * 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
JOURNAL OF VIROLOGY, 2000年, vol. 74, no. 7, JPN6023002574, pages 3122 - 3129, ISSN: 0005240829 *
JOURNAL OF VIROLOGY, 2002年, vol. 76, no. 2, JPN6023002573, pages 791 - 801, ISSN: 0005240828 *
VIROLOGY, 2004年, vol. 330, JPN6023002572, pages 375 - 383, ISSN: 0004973802 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024143440A1 (ja) 2022-12-28 2024-07-04 富士フイルム株式会社 アデノ随伴ウイルスの製造方法、およびベクターのセット
EP4644556A1 (en) 2022-12-28 2025-11-05 FUJIFILM Corporation Method for producing adeno-associated virus, and vector set
WO2024172027A1 (ja) * 2023-02-17 2024-08-22 株式会社シンプロジェン ＩＴＲ－Ｒｅｐ遺伝子複合体

Also Published As

Publication number Publication date
CN112166195A (zh) 2021-01-01
US20230399658A1 (en) 2023-12-14
CA3092871A1 (en) 2019-09-12
US12163152B2 (en) 2024-12-10
US11905524B2 (en) 2024-02-20
JP2023171525A (ja) 2023-12-01
KR20200130337A (ko) 2020-11-18
US20200407753A1 (en) 2020-12-31
EP3762501A1 (en) 2021-01-13
WO2019173538A1 (en) 2019-09-12
EP3762501A4 (en) 2022-03-09
AU2019231711A1 (en) 2020-09-24

Similar Documents

Publication Publication Date Title
US12163152B2 (en) AAV chimeras
CA2985945C (en) Capsid
CN104520421B (zh) 用于生产腺伴随病毒的细胞系
Gonçalves Adeno-associated virus: from defective virus to effective vector
JP2024514956A (ja) 組織標的化された改変ａａｖカプシドおよびその使用方法
US20230159953A1 (en) Closed-ended, linear, duplex adenoassociated virus dna, and uses thereof
CN114150021B (zh) 一种包含重叠开放阅读框的基因的表达盒及其在昆虫细胞中的应用
CN110770344B (zh) shRNA表达框、携带其的多核苷酸序列及其应用
CN106701691B (zh) 一种可高效感染免疫细胞的aav病毒及其制备方法与应用
CN111718418A (zh) 一种增强基因编辑的融合蛋白及其应用
Coura et al. A role for adeno-associated viral vectors in gene therapy
Gao et al. Exploiting natural diversity of AAV for the design of vectors with novel properties
Adachi et al. A new recombinant adeno-associated virus (Aav)-based random peptide display library system: infection-defective Aav1. 9-3 as a novel detargeted platform for vector evolution
US20210292373A1 (en) Aav vp1u chimeras
WO2024050547A2 (en) Compact bidirectional promoters for gene expression
CN119569834A (zh) 靶向心肌细胞的aav衣壳蛋白变体及其用途
CN119331062A (zh) 靶向骨骼肌的aav衣壳蛋白变体及其用途
US20240425879A1 (en) Compositions and methods for adeno-associated (aav) virus dnase expression
CN119899246A (zh) Aav衣壳蛋白变体及其用途
CN119569833A (zh) 靶向骨骼肌的aav衣壳蛋白变体及其用途
HK40020047A (en) Capsid
Cho et al. Improved Production Efficiencies of Various Adeno-Associated Virus (AAV) Serotypes and a Novel Universal AAV Titration Method

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220304

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220304

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20230123

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230125

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230425

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230607

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231010

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20231120

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20240119