JP2023544165A5 - - Google Patents

Info

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
Authority
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.)
Pending
Application number
JP2023520053A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023544165A (ja
Filing date
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
Pending legal-status Critical Current

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 Pending JP2023544165A (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 (2)

Publication Number Publication Date
JP2023544165A JP2023544165A (ja) 2023-10-20
JP2023544165A5 true JP2023544165A5 (https=) 2024-10-07

Family

ID=78599166

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023520053A Pending JP2023544165A (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 (https=)
EP (1) EP4222251A1 (https=)
JP (1) JP2023544165A (https=)
KR (1) KR20230079172A (https=)
AU (1) AU2021355481A1 (https=)
BR (1) BR112023003929A2 (https=)
CA (1) CA3193866A1 (https=)
CO (1) CO2023003418A2 (https=)
IL (1) IL301677A (https=)
MX (1) MX2023003805A (https=)
TW (1) TWI900667B (https=)
WO (1) WO2022072657A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019357039B2 (en) * 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy
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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CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
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KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 aav 벡터에 대한 개선된 정제 방법
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
ES2857773T5 (es) * 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのmRNA療法
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CA3077355A1 (en) 2017-10-02 2019-04-11 American Gene Technologies International Inc. Vectors with promoter and enhancer combinations for treating phenylketonuria
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US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
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
AU2019287468B2 (en) * 2018-06-12 2024-10-10 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
AU2019357039B2 (en) * 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy
EP3938515A4 (en) 2019-03-13 2022-12-28 Generation Bio Co. NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS
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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
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