JP2020535828A5 - - Google Patents

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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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JP2020535828A (ja
JP7838779B2 (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 (https=) 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 (https=)
EP (1) EP3692157A4 (https=)
JP (2) JP7838779B2 (https=)
KR (1) KR20200057766A (https=)
CN (1) CN111433368A (https=)
AU (1) AU2018345745A1 (https=)
BR (1) BR112020006672A2 (https=)
CA (1) CA3077355A1 (https=)
IL (1) IL273670B2 (https=)
WO (1) WO2019070674A2 (https=)

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US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
EP3319631A4 (en) 2015-07-08 2019-01-09 American Gene Technologies International Inc. HIV PREMUNICATION AND IMMUNOTHERAPY
CN116064678A (zh) 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
WO2017156311A2 (en) 2016-03-09 2017-09-14 American Gene Technologies International Inc. Combination vectors and methods for treating cancer
EP3468617A4 (en) 2016-06-08 2020-01-22 American Gene Technologies International Inc. SYSTEM FOR THE NON-INTEGRAL VIRAL ISSUE AND PROCEDURE RELATING TO IT
AU2017292582C1 (en) 2016-07-08 2021-11-11 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
EP3487507A4 (en) 2016-07-21 2020-04-08 American Gene Technologies International, Inc. VIRAL VECTORS FOR THE TREATMENT OF PARKINSON'S DISEASE
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
JP2020524996A (ja) 2017-06-16 2020-08-27 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 ヒトガンマデルタt細胞を介する腫瘍細胞傷害性の活性化のための方法および組成物
BR112021024124A2 (pt) * 2019-05-31 2022-01-11 American Gene Tech Int Inc Expressão otimizada de fenilalanina hidroxilase
JP2023510392A (ja) * 2020-01-16 2023-03-13 武田薬品工業株式会社 フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療
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
MX2023003805A (es) 2020-10-01 2023-04-12 Genzyme Corp Casete de expresion de pah humana para el tratamiento de la pku mediante terapia de reemplazo genico dirigida al higado.
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)

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Publication number Priority date Publication date Assignee Title
ES2284545T3 (es) 1999-11-16 2007-11-16 Genzyme Corporation Vectores y transgenes con elementos reguladores para la administracion de genes del higado.
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
JP6188728B2 (ja) * 2012-02-07 2017-08-30 グローバル・バイオ・セラピューティクス・インコーポレイテッドGlobal Bio Therapeutics,Inc. 核酸送達の区画化方法ならびにその組成物および使用
HK1225258B (en) 2013-08-08 2017-09-08 Global Bio Therapeutics, Inc. Clamping device for minimally invasive surgery
CN114395559A (zh) * 2014-04-25 2022-04-26 吉尼松公司 高胆红素血症的治疗
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
CA3077651A1 (en) * 2017-10-03 2019-04-11 Joseph Lillegard Vectors and methods of use
TW202016314A (zh) * 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法

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