JP2022508182A5 - - Google Patents

Info

Publication number
JP2022508182A5
JP2022508182A5 JP2021529002A JP2021529002A JP2022508182A5 JP 2022508182 A5 JP2022508182 A5 JP 2022508182A5 JP 2021529002 A JP2021529002 A JP 2021529002A JP 2021529002 A JP2021529002 A JP 2021529002A JP 2022508182 A5 JP2022508182 A5 JP 2022508182A5
Authority
JP
Japan
Prior art keywords
sequence
nucleic acid
aav vector
acid according
recombinant 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.)
Granted
Application number
JP2021529002A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022508182A (ja
JP7704998B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/062531 external-priority patent/WO2020106916A1/en
Publication of JP2022508182A publication Critical patent/JP2022508182A/ja
Publication of JP2022508182A5 publication Critical patent/JP2022508182A5/ja
Priority to JP2025034621A priority Critical patent/JP2025102783A/ja
Application granted granted Critical
Publication of JP7704998B2 publication Critical patent/JP7704998B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021529002A 2018-11-21 2019-11-21 組換えウイルスベクター及びそれの産生のための核酸 Active JP7704998B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025034621A JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 2018-11-21
US62/770,202 2018-11-21
PCT/US2019/062531 WO2020106916A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025034621A Division JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Publications (3)

Publication Number Publication Date
JP2022508182A JP2022508182A (ja) 2022-01-19
JP2022508182A5 true JP2022508182A5 (enExample) 2022-11-30
JP7704998B2 JP7704998B2 (ja) 2025-07-09

Family

ID=70773595

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021529002A Active JP7704998B2 (ja) 2018-11-21 2019-11-21 組換えウイルスベクター及びそれの産生のための核酸
JP2025034621A Pending JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025034621A Pending JP2025102783A (ja) 2018-11-21 2025-03-05 組換えウイルスベクター及びそれの産生のための核酸

Country Status (20)

Country Link
US (1) US20210324418A1 (enExample)
EP (1) EP3883954A4 (enExample)
JP (2) JP7704998B2 (enExample)
KR (1) KR20210103469A (enExample)
CN (1) CN113302201A (enExample)
AR (1) AR117145A1 (enExample)
AU (1) AU2019385506A1 (enExample)
BR (1) BR112021009733A2 (enExample)
CA (1) CA3120289A1 (enExample)
CL (1) CL2021001327A1 (enExample)
CO (1) CO2021008120A2 (enExample)
EA (1) EA202191418A1 (enExample)
EC (1) ECSP21044840A (enExample)
IL (1) IL283344A (enExample)
MX (1) MX2021005997A (enExample)
PE (1) PE20211419A1 (enExample)
PH (1) PH12021551155A1 (enExample)
SG (1) SG11202105326WA (enExample)
TW (1) TW202039533A (enExample)
WO (1) WO2020106916A1 (enExample)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107686842A (zh) * 2016-08-03 2018-02-13 南京大学 一种靶多核苷酸编辑方法及其应用
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
US12173304B2 (en) * 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
US20230285596A1 (en) * 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
MX2023002016A (es) * 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
MX2023003656A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector dlx2.
EP4222270A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. NEUROD1-VECTOR
US20220098617A1 (en) * 2020-09-29 2022-03-31 NeuExcell Therapeutics Inc. Ascl1 vector
MX2023003654A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector de combinación de neurod1.
EP4232152A4 (en) * 2020-10-21 2025-04-23 Duke University Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy
CN116887866A (zh) 2020-12-03 2023-10-13 巴特尔纪念研究院 聚合物纳米颗粒和dna纳米结构组合物及用于非病毒递送的方法
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
JP2024516108A (ja) 2021-04-07 2024-04-12 バテル・メモリアル・インスティテュート 非ウイルス性担体を同定および使用するための迅速な設計、構築、試験、および学習技術
US20240376497A1 (en) * 2021-09-17 2024-11-14 Astellas Gene Therapies, Inc. Frataxin gene therapy
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
US20250361525A1 (en) * 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
CN118715025A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于增强基因表达的构建体
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
US12441996B2 (en) 2023-12-08 2025-10-14 Battelle Memorial Institute Use of DNA origami nanostructures for molecular information based data storage systems
CN117701638B (zh) * 2023-12-18 2024-10-18 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002081632A2 (en) * 2001-04-04 2002-10-17 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services Nucleic acids for transgene expression
US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
EP2986635B1 (en) * 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
WO2015044704A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
US10947596B2 (en) 2014-05-06 2021-03-16 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of cancer using NFS1 biomarkers and modulators
WO2016094783A1 (en) * 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US20180021364A1 (en) 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US10450563B2 (en) * 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
WO2016150964A1 (en) * 2015-03-23 2016-09-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
WO2016164642A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
RU2021132397A (ru) * 2015-05-16 2022-02-24 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
PT3313991T (pt) * 2015-06-23 2024-09-27 Childrens Hospital Philadelphia Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos
US10266844B2 (en) * 2015-07-31 2019-04-23 California Institute Of Technology Activity-dependent expression of nucleic acids
PE20181156A1 (es) * 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica
RU2742612C2 (ru) * 2015-12-15 2021-02-09 Джензим Корпорейшн Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii
US11564997B2 (en) * 2016-06-29 2023-01-31 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US20200056206A1 (en) * 2016-12-01 2020-02-20 UNIVERSITé LAVAL Crispr-based treatment of friedreich ataxia
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
MX2020003042A (es) * 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.

Similar Documents

Publication Publication Date Title
JP2024009857A5 (enExample)
Grieger et al. Adeno-associated virus vectorology, manufacturing, and clinical applications
JP2021106619A5 (enExample)
CA2915124C (en) A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
RU2018116076A (ru) Модифицированные гены атаксии фридрейха и векторы для генной терапии
JP2019116492A5 (enExample)
JP2022549380A5 (enExample)
JP2021512649A5 (enExample)
JP2021523718A5 (enExample)
JP2020503869A5 (enExample)
JP2021511803A5 (enExample)
JP2022535749A5 (enExample)
JP2018522529A5 (enExample)
JP2022551744A5 (enExample)
JP7541027B2 (ja) 筋肉発現のためのハイブリッドプロモーター
JP2017509632A5 (enExample)
JP2023537625A5 (enExample)
JP2023514242A5 (enExample)
JP2022541070A5 (enExample)
JP2023537358A5 (enExample)
JP2023524024A5 (enExample)
IL292264B1 (en) Aav transfer cassette
JP2024508696A5 (enExample)
JP2021520811A5 (enExample)
JP2023541443A5 (enExample)