JP2023544165A5 - - Google Patents

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Publication number
JP2023544165A5
JP2023544165A5 JP2023520053A JP2023520053A JP2023544165A5 JP 2023544165 A5 JP2023544165 A5 JP 2023544165A5 JP 2023520053 A JP2023520053 A JP 2023520053A JP 2023520053 A JP2023520053 A JP 2023520053A JP 2023544165 A5 JP2023544165 A5 JP 2023544165A5
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JP
Japan
Prior art keywords
aav
raav
acid sequence
nucleic acid
enhancer
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
JP2023520053A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023544165A (ja
JP7893807B2 (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/052913 external-priority patent/WO2022072657A1/en
Publication of JP2023544165A publication Critical patent/JP2023544165A/ja
Publication of JP2023544165A5 publication Critical patent/JP2023544165A5/ja
Application granted granted Critical
Publication of JP7893807B2 publication Critical patent/JP7893807B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2023520053A 2020-10-01 2021-09-30 肝臓に向けられた遺伝子補充療法によってpkuを処置するためのヒトpah発現カセット Active JP7893807B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063086537P 2020-10-01 2020-10-01
US63/086,537 2020-10-01
US202063121797P 2020-12-04 2020-12-04
US63/121,797 2020-12-04
PCT/US2021/052913 WO2022072657A1 (en) 2020-10-01 2021-09-30 Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy

Publications (3)

Publication Number Publication Date
JP2023544165A JP2023544165A (ja) 2023-10-20
JP2023544165A5 true JP2023544165A5 (enExample) 2024-10-07
JP7893807B2 JP7893807B2 (ja) 2026-07-22

Family

ID=78599166

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023520053A Active JP7893807B2 (ja) 2020-10-01 2021-09-30 肝臓に向けられた遺伝子補充療法によってpkuを処置するためのヒトpah発現カセット

Country Status (12)

Country Link
US (2) US12173308B2 (enExample)
EP (1) EP4222251A1 (enExample)
JP (1) JP7893807B2 (enExample)
KR (1) KR20230079172A (enExample)
AU (1) AU2021355481A1 (enExample)
BR (1) BR112023003929A2 (enExample)
CA (1) CA3193866A1 (enExample)
CO (1) CO2023003418A2 (enExample)
IL (1) IL301677A (enExample)
MX (1) MX2023003805A (enExample)
TW (1) TWI900667B (enExample)
WO (1) WO2022072657A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120349985A (zh) * 2018-10-12 2025-07-22 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
CN115896135B (zh) * 2022-11-02 2024-03-01 苏州诺洁贝生物技术有限公司 优化的pah基因和表达盒及其用途

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US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
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JP4559429B2 (ja) 2003-05-21 2010-10-06 ジェンザイム・コーポレーション 空キャプシドを実質的に含まない組換えaavビリオン調製物を生成するための方法
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US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
CA2660286A1 (en) * 2006-08-09 2008-02-21 Homestead Clinical Corporation Organ-specific proteins and methods of their use
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えaavベクターの改良された精製方法
CN103502458B (zh) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
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US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
WO2019152841A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
KR20210008501A (ko) 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법
JP7428664B2 (ja) * 2018-06-12 2024-02-06 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 合成肝臓指向性アデノ随伴ウイルスカプシドおよびその使用
CN120349985A (zh) 2018-10-12 2025-07-22 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
US12415002B2 (en) 2019-03-13 2025-09-16 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing phenylalanine hydroxylase (PAH) therapeutics
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
WO2021144649A2 (en) 2020-01-16 2021-07-22 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
TW202144575A (zh) 2020-04-03 2021-12-01 美商拜奧馬林製藥公司 使用aav及治療調配物之苯酮尿症治療
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
AU2021285812A1 (en) 2020-06-01 2023-01-05 Modernatx, Inc. Phenylalanine hydroxylase variants and uses thereof
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