JP2020535828A5 - - Google Patents

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Publication number
JP2020535828A5
JP2020535828A5 JP2020518812A JP2020518812A JP2020535828A5 JP 2020535828 A5 JP2020535828 A5 JP 2020535828A5 JP 2020518812 A JP2020518812 A JP 2020518812A JP 2020518812 A JP2020518812 A JP 2020518812A JP 2020535828 A5 JP2020535828 A5 JP 2020535828A5
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Japan
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viral vector
seq
vector according
cells
item
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JP2020518812A
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Japanese (ja)
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JP7838779B2 (ja
JP2020535828A (ja
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Priority claimed from PCT/US2018/053919 external-priority patent/WO2019070674A2/en
Publication of JP2020535828A publication Critical patent/JP2020535828A/ja
Publication of JP2020535828A5 publication Critical patent/JP2020535828A5/ja
Priority to JP2023101880A priority Critical patent/JP2023115125A/ja
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JP2020518812A 2017-10-02 2018-10-02 フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター Active JP7838779B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023101880A JP2023115125A (ja) 2017-10-02 2023-06-21 フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762566979P 2017-10-02 2017-10-02
US62/566,979 2017-10-02
PCT/US2018/053919 WO2019070674A2 (en) 2017-10-02 2018-10-02 VECTORS COMPRISING PROMOTER AND ACTIVATOR COMBINATIONS FOR THE TREATMENT OF PHENYLCETONURIA

Related Child Applications (1)

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JP2023101880A Division JP2023115125A (ja) 2017-10-02 2023-06-21 フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター

Publications (3)

Publication Number Publication Date
JP2020535828A JP2020535828A (ja) 2020-12-10
JP2020535828A5 true JP2020535828A5 (enExample) 2021-11-04
JP7838779B2 JP7838779B2 (ja) 2026-04-01

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ID=65994855

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020518812A Active JP7838779B2 (ja) 2017-10-02 2018-10-02 フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター
JP2023101880A Pending JP2023115125A (ja) 2017-10-02 2023-06-21 フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023101880A Pending JP2023115125A (ja) 2017-10-02 2023-06-21 フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター

Country Status (10)

Country Link
US (1) US20200318081A1 (enExample)
EP (1) EP3692157A4 (enExample)
JP (2) JP7838779B2 (enExample)
KR (1) KR20200057766A (enExample)
CN (1) CN111433368A (enExample)
AU (1) AU2018345745A1 (enExample)
BR (1) BR112020006672A2 (enExample)
CA (1) CA3077355A1 (enExample)
IL (1) IL273670B2 (enExample)
WO (1) WO2019070674A2 (enExample)

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WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
JP7153332B2 (ja) 2016-02-08 2022-10-14 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Hivワクチン接種および免疫療法
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
WO2017213697A1 (en) 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
BR112021024124A2 (pt) * 2019-05-31 2022-01-11 American Gene Tech Int Inc Expressão otimizada de fenilalanina hidroxilase
WO2021144649A2 (en) * 2020-01-16 2021-07-22 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
TW202208632A (zh) * 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
KR20230079172A (ko) 2020-10-01 2023-06-05 젠자임 코포레이션 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트
WO2023225569A1 (en) * 2022-05-17 2023-11-23 Umoja Biopharma, Inc. Manufacturing viral particles
CN115772503B (zh) * 2022-08-16 2023-10-17 广东省南山医药创新研究院 一种表达pah的基因修饰细胞药物及其制备方法与应用
TWI893802B (zh) * 2023-05-11 2025-08-11 大陸商四川至善唯新生物科技有限公司 工程化的肝臟特異性增強子、核心啟動子及其應用

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001036620A2 (en) * 1999-11-16 2001-05-25 Genzyme Corporation Vectors and transgenies with regulatory elements for gene delivery to the liver
US20030198620A1 (en) * 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US20050138677A1 (en) * 2003-09-16 2005-06-23 Pfister Herbert J. Transgenic animal model for the treatment of skin tumors
EP1854455B1 (en) 2006-05-10 2009-10-07 Biocompatibles UK Limited Spherical microcapsules comprising GLP-1 peptides, their production and use
EP2008656A1 (en) * 2007-06-28 2008-12-31 Bergen Teknologioverforing AS Compositions for the treatment of hyperphenylalaninemia
SG10201604810UA (en) * 2012-02-07 2016-08-30 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
CN105813579B (zh) 2013-08-08 2019-05-07 全球生物疗法有限公司 用于微创手术过程的夹具装置和其应用
ES2744565T3 (es) * 2014-04-25 2020-02-25 Genethon Tratamiento de la hiperbilirrubinemia
WO2017100551A1 (en) * 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
US11820999B2 (en) * 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
EP3692141A1 (en) * 2017-10-03 2020-08-12 Children's Hospital and Clinics of Minnesota Vectors and methods of use
KR20210008501A (ko) * 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법

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