JP2022535745A5 - - Google Patents

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Publication number
JP2022535745A5
JP2022535745A5 JP2021570364A JP2021570364A JP2022535745A5 JP 2022535745 A5 JP2022535745 A5 JP 2022535745A5 JP 2021570364 A JP2021570364 A JP 2021570364A JP 2021570364 A JP2021570364 A JP 2021570364A JP 2022535745 A5 JP2022535745 A5 JP 2022535745A5
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JP
Japan
Prior art keywords
sequence
seq
percent
codon
viral vector
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.)
Pending
Application number
JP2021570364A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022535745A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/035584 external-priority patent/WO2020243717A1/en
Publication of JP2022535745A publication Critical patent/JP2022535745A/ja
Publication of JP2022535745A5 publication Critical patent/JP2022535745A5/ja
Pending legal-status Critical Current

Links

JP2021570364A 2019-05-31 2020-06-01 最適化されたフェニルアラニンヒドロキシラーゼ発現 Pending JP2022535745A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962855506P 2019-05-31 2019-05-31
US62/855,506 2019-05-31
PCT/US2020/035584 WO2020243717A1 (en) 2019-05-31 2020-06-01 Optimized phenylalanine hydroxylase expression

Publications (2)

Publication Number Publication Date
JP2022535745A JP2022535745A (ja) 2022-08-10
JP2022535745A5 true JP2022535745A5 (enExample) 2023-06-08

Family

ID=73553945

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021570364A Pending JP2022535745A (ja) 2019-05-31 2020-06-01 最適化されたフェニルアラニンヒドロキシラーゼ発現

Country Status (10)

Country Link
US (1) US20220162643A1 (enExample)
EP (1) EP3976076A4 (enExample)
JP (1) JP2022535745A (enExample)
KR (1) KR20220068954A (enExample)
CN (1) CN113905768A (enExample)
AU (1) AU2020283069A1 (enExample)
BR (1) BR112021024124A2 (enExample)
CA (1) CA3137698A1 (enExample)
IL (1) IL288400A (enExample)
WO (1) WO2020243717A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
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
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
MX2023003019A (es) * 2020-09-16 2023-05-08 Generation Bio Co Vectores de adn de extremo cerrado y usos de estos para expresar fenilalanina hidroxilasa (pah).
EP4402267A4 (en) * 2021-09-16 2025-11-26 Generation Bio Co Liver-specific expression cassettes, vectors, and their uses for expressing therapeutic proteins
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
IL327036A (en) * 2023-09-19 2026-05-01 Poseida Therapeutics Inc Compositions and methods for combining viral vectors

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
EP3574923A1 (en) * 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
CN115094062A (zh) * 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
WO2017106326A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
AU2017248189B2 (en) * 2016-04-08 2021-04-29 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
EP3562494A4 (en) * 2016-12-30 2020-08-19 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS
US11820999B2 (en) * 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
KR20200057766A (ko) * 2017-10-02 2020-05-26 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 페닐케톤뇨증을 치료하기 위한 프로모터 및 인핸서 조합을 포함하는 벡터

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