JP2022511348A5 - - Google Patents

Info

Publication number
JP2022511348A5
JP2022511348A5 JP2021516750A JP2021516750A JP2022511348A5 JP 2022511348 A5 JP2022511348 A5 JP 2022511348A5 JP 2021516750 A JP2021516750 A JP 2021516750A JP 2021516750 A JP2021516750 A JP 2021516750A JP 2022511348 A5 JP2022511348 A5 JP 2022511348A5
Authority
JP
Japan
Prior art keywords
sequence
nucleic acid
plasmid
seq
upstream
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
JP7560447B2 (ja
JP2022511348A (ja
Filing date
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
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 (3)

Publication Number Publication Date
JP2022511348A JP2022511348A (ja) 2022-01-31
JP2022511348A5 true JP2022511348A5 (enExample) 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 (enExample)
EP (1) EP3870148A4 (enExample)
JP (2) JP7560447B2 (enExample)
KR (1) KR20210086645A (enExample)
CN (1) CN112888426A (enExample)
AU (1) AU2019363593A1 (enExample)
CA (1) CA3112883A1 (enExample)
IL (1) IL281909A (enExample)
TW (1) TW202029957A (enExample)
WO (1) WO2020086881A1 (enExample)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
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
MX2023000318A (es) * 2020-07-07 2023-02-09 Univ Texas Vector de virus adenoasociado para marco de lectura abierto peque?o.
US12054738B2 (en) 2020-07-30 2024-08-06 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
EP4242310A4 (en) * 2020-11-04 2024-09-04 Synplogen Co., Ltd. PRODUCTION OF VIRAL 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
CN117529558A (zh) * 2021-04-21 2024-02-06 阿西莫夫公司 用于腺相关病毒产生的稳定产生系统
US12305188B2 (en) 2021-05-27 2025-05-20 Neuracle Genetics Inc. Dual helper plasmid
WO2022250491A1 (en) * 2021-05-27 2022-12-01 Neuracle Genetics Inc. Novel dual helper plasmid
AU2022319723A1 (en) * 2021-07-28 2024-02-22 Atsena Therapeutics, Inc. Aav transfer plasmids
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
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
EP4522729A1 (en) * 2022-05-12 2025-03-19 Aavnergene Inc. Compositions and methods for recombinant parvovirus production
KR20250040030A (ko) * 2022-08-31 2025-03-21 전지앙 프로바이오 바이오테크 컴퍼니 리미티드 아데노 관련 바이러스 역가를 향상시킬 수 있는 아데노 관련 바이러스 구조 플라스미드
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
WO2024211394A1 (en) * 2023-04-03 2024-10-10 Decibel Therapeutics, Inc. Modified rep-cap plasmids and uses thereof
EP4713344A2 (en) * 2023-05-16 2026-03-25 Oxford Biomedica (US) LLC Compositions and methods for increasing aav productivity
CN121794376A (zh) * 2023-09-07 2026-04-03 北京中因科技股份有限公司 Aav载体骨架优化及应用
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
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
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
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
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
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
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템
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
HK1220488A1 (zh) * 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015031686A1 (en) * 2013-08-30 2015-03-05 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
EP4678754A3 (en) * 2014-03-11 2026-04-08 Wayne State University A modified mglur6 promoter and methods of use
EP3218503A4 (en) * 2014-11-10 2018-06-06 Murdoch Childrens Research Institute Vectors and methods for targeted integration in loci comprising constitutively expressed genes
SI3265571T1 (sl) 2015-03-03 2022-10-28 Fondazione Telethon Več vektorski sistem in njegove uporabe
EP3294309A4 (en) 2015-05-14 2019-01-16 St. Jude Children's Research Hospital, Inc. NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF
WO2016196507A1 (en) 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
EP3533471B1 (en) 2016-10-31 2022-01-19 Kolon Life Science, Inc. Composition for alleviating or treating pain
CN113056561A (zh) 2018-04-05 2021-06-29 牛津大学科技创新有限公司 用于治疗黄斑营养不良的组合物和方法

Similar Documents

Publication Publication Date Title
JP2022511348A5 (enExample)
Nonnenmacher et al. Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning
US9896665B2 (en) Proviral plasmids and production of recombinant adeno-associated virus
JP7413256B2 (ja) 神経変性疾患の遺伝子治療
JP2020537544A5 (enExample)
US12104175B2 (en) High activity regulatory elements
JP2022530544A5 (enExample)
CA3162619A1 (en) Process for making adenoassociated viral vectors
JP2022530633A5 (enExample)
Nalbantoglu et al. Muscle-specific overexpression of the adenovirus primary receptor CAR overcomes low efficiency of gene transfer to mature skeletal muscle
JP2022530656A5 (enExample)
JP2022544004A (ja) 操作された核酸調節エレメントならびにその使用方法
JP2020537542A5 (enExample)
CN111886343A (zh) 使用闭合端dna(cedna)载体控制转基因的表达
US12600989B2 (en) Adeno-associated virus (AAV) vector and uses therefor
US20250066811A1 (en) DNA Amplification Method Using CARE Elements
EP3294891A1 (en) Polynucleotides, vectors and methods for insertion and expression of transgenes
JP7854125B2 (ja) 新規なデュアルヘルパープラスミド
US20250019667A1 (en) Adenoviral helper plasmid
WO2023147558A2 (en) Crispr methods for correcting bag3 gene mutations in vivo
Adachi et al. A PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging
EP4570260A1 (en) Viral vector for transduction of adipocytes
EP4667577A1 (en) Itr-rep gene complex
CN117264912A (zh) 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用
HK40061786A (en) Process for making adenoassociated viral vectors