JPWO2020086881A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2020086881A5
JPWO2020086881A5 JP2021516750A JP2021516750A JPWO2020086881A5 JP WO2020086881 A5 JPWO2020086881 A5 JP WO2020086881A5 JP 2021516750 A JP2021516750 A JP 2021516750A JP 2021516750 A JP2021516750 A JP 2021516750A JP WO2020086881 A5 JPWO2020086881 A5 JP WO2020086881A5
Authority
JP
Japan
Prior art keywords
nucleic acid
plasmid
sequence
seq
downstream
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.)
Granted
Application number
JP2021516750A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022511348A (ja
JP7560447B2 (ja
JP2022511348A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/057916 external-priority patent/WO2020086881A1/en
Publication of JP2022511348A publication Critical patent/JP2022511348A/ja
Publication of JP2022511348A5 publication Critical patent/JP2022511348A5/ja
Publication of JPWO2020086881A5 publication Critical patent/JPWO2020086881A5/ja
Priority to JP2024161633A priority Critical patent/JP2025000711A/ja
Application granted granted Critical
Publication of JP7560447B2 publication Critical patent/JP7560447B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021516750A 2018-10-25 2019-10-24 Aavトリプルプラスミドシステム Active JP7560447B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024161633A JP2025000711A (ja) 2018-10-25 2024-09-19 Aavトリプルプラスミドシステム

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862750603P 2018-10-25 2018-10-25
US62/750,603 2018-10-25
PCT/US2019/057916 WO2020086881A1 (en) 2018-10-25 2019-10-24 Aav triple-plasmid system

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024161633A Division JP2025000711A (ja) 2018-10-25 2024-09-19 Aavトリプルプラスミドシステム

Publications (4)

Publication Number Publication Date
JP2022511348A JP2022511348A (ja) 2022-01-31
JP2022511348A5 JP2022511348A5 (https=) 2022-10-19
JPWO2020086881A5 true JPWO2020086881A5 (https=) 2022-10-19
JP7560447B2 JP7560447B2 (ja) 2024-10-02

Family

ID=70331899

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021516750A Active JP7560447B2 (ja) 2018-10-25 2019-10-24 Aavトリプルプラスミドシステム
JP2024161633A Pending JP2025000711A (ja) 2018-10-25 2024-09-19 Aavトリプルプラスミドシステム

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024161633A Pending JP2025000711A (ja) 2018-10-25 2024-09-19 Aavトリプルプラスミドシステム

Country Status (10)

Country Link
US (1) US12383587B2 (https=)
EP (1) EP3870148A4 (https=)
JP (2) JP7560447B2 (https=)
KR (1) KR20210086645A (https=)
CN (1) CN112888426A (https=)
AU (1) AU2019363593A1 (https=)
CA (1) CA3112883A1 (https=)
IL (1) IL281909A (https=)
TW (1) TW202029957A (https=)
WO (1) WO2020086881A1 (https=)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019144061A1 (en) 2018-01-19 2019-07-25 Duke University Genome engineering with crispr-cas systems in eukaryotes
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
EP4189077A1 (en) 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
IL302578A (en) * 2020-11-04 2023-07-01 Synplogen Co Ltd Production of a virus vector plasmid in Bacillus Subtilis
US11926842B2 (en) * 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
US20240360422A1 (en) * 2021-04-21 2024-10-31 Michael T. Leonard Stable production systems for adeno-associated virus production
US12305188B2 (en) 2021-05-27 2025-05-20 Neuracle Genetics Inc. Dual helper plasmid
KR102685652B1 (ko) * 2021-05-27 2024-07-19 주식회사 뉴라클제네틱스 신규한 듀얼 헬퍼 플라스미드
EP4377471A4 (en) * 2021-07-28 2025-10-15 Atsena Therapeutics Inc AAV TRANSFER PLASMIDS
CN114196705B (zh) * 2021-12-07 2024-09-06 中国科学院深圳先进技术研究院 一种重组腺相关病毒包装质粒、重组腺相关病毒及其应用
WO2023102736A1 (zh) * 2021-12-07 2023-06-15 中国科学院深圳先进技术研究院 一种重组腺相关病毒包装质粒、重组腺相关病毒及其应用
KR20250009975A (ko) * 2022-05-12 2025-01-20 아브너진 인코포레이티드 재조합 파르보바이러스 생산을 위한 조성물 및 방법
CN119866377A (zh) * 2022-08-31 2025-04-22 镇江蓬勃生物科技有限公司 能够提高腺相关病毒滴度的腺相关病毒结构质粒
CN116064672B (zh) * 2022-09-15 2025-02-14 思鹏生物科技(苏州)有限公司 利用ires基因提升aav包装效率的方法及其功能基因
WO2024098035A2 (en) * 2022-11-04 2024-05-10 National Resilience, Inc. Methods and compositions for preparing recombinant adeno associated viruses and uses thereof
WO2024160232A1 (zh) * 2023-02-01 2024-08-08 纽福斯(苏州)生物科技有限公司 质粒包装系统及其应用
CN116411024A (zh) * 2023-03-10 2023-07-11 上海泰昶生物技术有限公司 用于生产重组腺相关病毒的质粒系统
US20240352483A1 (en) * 2023-04-03 2024-10-24 Decibel Therapeutics, Inc. Modified rep-cap plasmids and uses thereof
WO2024238742A2 (en) * 2023-05-16 2024-11-21 Oxford Biomedica (Us) Llc Compositions and methods for increasing aav productivity
WO2025050347A1 (en) * 2023-09-07 2025-03-13 Chigenovo Co., Ltd. Aav vector backbone optimization and use thereof
CN117683797B (zh) * 2023-12-04 2024-07-05 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
CN117925663A (zh) * 2023-12-04 2024-04-26 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
CN117778431B (zh) * 2023-12-04 2024-10-25 广州派真生物技术有限公司 一种用于重组腺相关病毒包装的质粒系统及其应用
CN121780618A (zh) * 2023-12-13 2026-04-03 广州派真生物技术有限公司 一种高产重组腺相关病毒的辅助质粒及应用
CN117660534B (zh) * 2023-12-13 2024-05-07 广州派真生物技术有限公司 一种降低重组腺相关病毒中宿主细胞dna残留的辅助质粒及应用
US20250276095A1 (en) 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025217308A1 (en) * 2024-04-10 2025-10-16 Solid Biosciences Inc. Method of aav production
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
CN119662733B (zh) * 2024-12-09 2025-10-28 广州派真生物技术有限公司 一种高产rAAV的质粒系统及应用

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6004797A (en) 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
CA2369985A1 (en) 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
MX384795B (es) * 2013-03-15 2025-03-14 Childrens Hospital Philadelphia Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso.
KR20240090694A (ko) 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
EP3039129B1 (en) 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
AU2015229464B2 (en) * 2014-03-11 2021-07-22 Wayne State University A modified mGluR6 promoter and methods of use
WO2016074016A1 (en) 2014-11-10 2016-05-19 Murdoch Childrens Research Institute Vectors and methods for targeted integration in loci comprising constitutively expressed genes
RS63416B1 (sr) 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
WO2016183422A1 (en) 2015-05-14 2016-11-17 St. Jude Children's Research Hospital, Inc. Nucleic acid molecules containing spacers and methods of use thereof
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
KR102445192B1 (ko) 2016-10-31 2022-09-20 코오롱생명과학 주식회사 통증 완화 또는 치료용 조성물
JP2021520232A (ja) 2018-04-05 2021-08-19 オックスフォード ユニバーシティ イノベーション リミテッドOxford University Innovation Limited 黄斑ジストロフィーを処置するための組成物及び方法

Similar Documents

Publication Publication Date Title
JPWO2020086881A5 (https=)
CN115298316B (zh) 用于制备腺相关病毒载体的方法
US9896665B2 (en) Proviral plasmids and production of recombinant adeno-associated virus
JP6093358B2 (ja) アデノ随伴ウイルスベクターの産生細胞
JP7530431B2 (ja) Dna増幅方法
JP2020537544A5 (https=)
EP4200428B1 (en) Method of making recombinant aavs
JP2024517104A (ja) Careエレメントを用いたdna増幅方法
CN118460614A (zh) 一种腺相关病毒Cap蛋白的表达框
US20250019667A1 (en) Adenoviral helper plasmid
JP7853958B2 (ja) 組み換えaavの作製方法
WO2025262205A1 (en) Nucleic acid sequences
WO2025262203A1 (en) Nucleic acid sequences
HK40061786A (en) Process for making adenoassociated viral vectors
JPWO2022079083A5 (https=)
KR20260052122A (ko) 아데노-연관 바이러스(AAV) 기반 벡터의 생산을 위한 아데노바이러스 E2a 유전자의 변형된 서열 (변이체)
WO2022251096A1 (en) Promoter sequence and related products and uses thereof
HK40061787A (en) Dna amplification method
HK40096036A (en) Method of making recombinant aavs
JPWO2023060113A5 (https=)
JPWO2022241215A5 (https=)