JP7854125B2 - 新規なデュアルヘルパープラスミド - Google Patents

新規なデュアルヘルパープラスミド

Info

Publication number
JP7854125B2
JP7854125B2 JP2023573241A JP2023573241A JP7854125B2 JP 7854125 B2 JP7854125 B2 JP 7854125B2 JP 2023573241 A JP2023573241 A JP 2023573241A JP 2023573241 A JP2023573241 A JP 2023573241A JP 7854125 B2 JP7854125 B2 JP 7854125B2
Authority
JP
Japan
Prior art keywords
gene
rep
cap
plasmid
aav
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.)
Active
Application number
JP2023573241A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024519175A (ja
JP2024519175A5 (https=
Inventor
ソク ハン，ジュ
ヨン コン，フン
ヒョン ファン，ユン
Original Assignee
エリシジェン インコーポレイテッド
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
Priority claimed from US17/750,241 external-priority patent/US12305188B2/en
Application filed by エリシジェン インコーポレイテッド filed Critical エリシジェン インコーポレイテッド
Publication of JP2024519175A publication Critical patent/JP2024519175A/ja
Publication of JP2024519175A5 publication Critical patent/JP2024519175A5/ja
Application granted granted Critical
Publication of JP7854125B2 publication Critical patent/JP7854125B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2023573241A 2021-05-27 2022-05-27 新規なデュアルヘルパープラスミド Active JP7854125B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
KR20210068364 2021-05-27
KR10-2021-0068364 2021-05-27
US17/750,241 US12305188B2 (en) 2021-05-27 2022-05-20 Dual helper plasmid
US17/750,241 2022-05-20
PCT/KR2022/007553 WO2022250491A1 (en) 2021-05-27 2022-05-27 Novel dual helper plasmid

Publications (3)

Publication Number Publication Date
JP2024519175A JP2024519175A (ja) 2024-05-08
JP2024519175A5 JP2024519175A5 (https=) 2024-08-05
JP7854125B2 true JP7854125B2 (ja) 2026-05-01

Family

ID=84228978

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023573241A Active JP7854125B2 (ja) 2021-05-27 2022-05-27 新規なデュアルヘルパープラスミド

Country Status (6)

Country Link
US (1) US20260092291A1 (https=)
EP (1) EP4347850A4 (https=)
JP (1) JP7854125B2 (https=)
KR (1) KR102685652B1 (https=)
AU (1) AU2022282057B2 (https=)
WO (1) WO2022250491A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024211780A1 (en) * 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025083617A1 (en) * 2023-10-18 2025-04-24 Nanocell Therapeutics Holdings B.V. Methods and compositions for nucleic construct delivery

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003501042A (ja) 1999-05-27 2003-01-14 ジェノボ， インコーポレイテッド 非哺乳動物ウイルス由来のキャリアベクターを使用する、組換えウイルスの産生のための組成物および方法
JP2008200043A (ja) 1994-11-03 2008-09-04 Cell Genesys Inc 新規なアデノウイルスベクター、パッケージング細胞系、組換えアデノウイルスおよび方法
JP2020517238A (ja) 2017-04-18 2020-06-18 グラクソスミスクライン、インテレクチュアル、プロパティー、ディベロップメント、リミテッドＧｌａｘｏｓｍｉｔｈｋｌｉｎｅ Ｉｎｔｅｌｌｅｃｔｕａｌ Ｐｒｏｐｅｒｔｙ Ｄｅｖｅｌｏｐｍｅｎｔ Ｌｉｍｉｔｅｄ アデノ随伴ウイルスベクターの産生方法
JP2021510496A (ja) 2018-01-19 2021-04-30 オックスフォード ジェネティクス リミテッドＯｘｆｏｒｄ Ｇｅｎｅｔｉｃｓ Ｌｉｍｉｔｅｄ Ａａｖ粒子の産生を目的としたベクター
US20210147877A1 (en) 2017-03-15 2021-05-20 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors for the treatment of disease
WO2022097647A1 (ja) 2020-11-04 2022-05-12 株式会社シンプロジェン 統合型プラスミド

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR100483597B1 (ko) * 2002-10-15 2005-04-15 주식회사 삼양제넥스 재조합 아데노부속바이러스 유전자 전달체 생산용 벡터시스템
DK2368903T3 (da) * 2010-03-26 2014-05-05 Deutsches Krebsforsch Adenovirusafledt hjælpevirus til fremme af produktion af rekombinant parvovirus
GB201706090D0 (en) * 2017-04-18 2017-05-31 Glaxosmithkline Ip Dev Ltd Methods for adeno-associated viral vector production
CA3112883A1 (en) * 2018-10-25 2020-04-30 Baxalta Incorporated Aav triple-plasmid system
DK3722434T3 (da) * 2019-04-12 2022-10-24 Freeline Therapeutics Ltd Plasmidsystem

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008200043A (ja) 1994-11-03 2008-09-04 Cell Genesys Inc 新規なアデノウイルスベクター、パッケージング細胞系、組換えアデノウイルスおよび方法
JP2003501042A (ja) 1999-05-27 2003-01-14 ジェノボ， インコーポレイテッド 非哺乳動物ウイルス由来のキャリアベクターを使用する、組換えウイルスの産生のための組成物および方法
US20210147877A1 (en) 2017-03-15 2021-05-20 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors for the treatment of disease
JP2020517238A (ja) 2017-04-18 2020-06-18 グラクソスミスクライン、インテレクチュアル、プロパティー、ディベロップメント、リミテッドＧｌａｘｏｓｍｉｔｈｋｌｉｎｅ Ｉｎｔｅｌｌｅｃｔｕａｌ Ｐｒｏｐｅｒｔｙ Ｄｅｖｅｌｏｐｍｅｎｔ Ｌｉｍｉｔｅｄ アデノ随伴ウイルスベクターの産生方法
JP2021510496A (ja) 2018-01-19 2021-04-30 オックスフォード ジェネティクス リミテッドＯｘｆｏｒｄ Ｇｅｎｅｔｉｃｓ Ｌｉｍｉｔｅｄ Ａａｖ粒子の産生を目的としたベクター
WO2022097647A1 (ja) 2020-11-04 2022-05-12 株式会社シンプロジェン 統合型プラスミド

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DEFINITION: Human adenovirus 5, complete genome, ACCESSION: AC_000008，Database GenBank [online]，2018年08月13日，<URL: https://www.ncbi.nlm.nih.gov/nuccore/AC_000008>，[令和6年10月10日検索], インターネット

Also Published As

Publication number Publication date
EP4347850A1 (en) 2024-04-10
US20260092291A1 (en) 2026-04-02
AU2022282057A1 (en) 2023-12-21
JP2024519175A (ja) 2024-05-08
KR102685652B1 (ko) 2024-07-19
AU2022282057A9 (en) 2024-01-25
WO2022250491A1 (en) 2022-12-01
KR20230152768A (ko) 2023-11-03
AU2022282057B2 (en) 2025-01-23
EP4347850A4 (en) 2025-04-02

Similar Documents

Publication Publication Date Title
AU2018372235B2 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
US20250121092A1 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
JP2025102825A (ja) アデノ随伴ウイルスベクター変種
JP2021514659A (ja) Ａａｖキメラ
AU2016259976A1 (en) Promoters for expression of heterologous genes
JP7785389B2 (ja) 新規なイントロン断片
US20260092291A1 (en) Novel dual helper plasmid
CN120641136A (zh) 用于额颞叶痴呆的基因疗法
CN116096904A (zh) 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
CA3219287C (en) Dual helper plasmid
US12305188B2 (en) Dual helper plasmid
CN117940446A (zh) 衣壳变体及其使用方法
US10105450B2 (en) Promoter compositions
EP4534097A1 (en) Composition for treating retinal or macular diseases
EA049452B1 (ru) Новые интронные фрагменты
KR20260046127A (ko) 뇌 지향성 aav 변이체
HK40121156A (en) Composition for treating retinal or macular diseases
WO2025128817A1 (en) Artificial micrornas targeting tau
CN119264217A (zh) 具有变异衣壳蛋白的腺相关病毒及其应用
BR122025008690A2 (pt) Polinucleotídeo, vetor, célula in vitro, método para produzir uma partícula de virus recombinante, partícula de virus recombinante e uso
BR112022024345B1 (pt) Polinucleotídeo isolado, vetor, célula procariótica in vitro, método para produzir uma partícula de virus recombinante, partícula de virus recombinante adenoassociado e uso

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231127

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20231127

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240726

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20240726

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20241031

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241105

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250127

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250507

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250701

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250926

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251014

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260109

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20260224

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20260325