TW202403049A - 治療異染性白質失養症之方法 - Google Patents

治療異染性白質失養症之方法 Download PDF

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TW202403049A
TW202403049A TW112117950A TW112117950A TW202403049A TW 202403049 A TW202403049 A TW 202403049A TW 112117950 A TW112117950 A TW 112117950A TW 112117950 A TW112117950 A TW 112117950A TW 202403049 A TW202403049 A TW 202403049A
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arsa
raav
seq
aav
sequence
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TW112117950A
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Chinese (zh)
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希亞姆 拉馬契德藍
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美商健臻公司
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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TW112117950A 2022-05-16 2023-05-15 治療異染性白質失養症之方法 TW202403049A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202263342590P 2022-05-16 2022-05-16
US63/342,590 2022-05-16
US202363459564P 2023-04-14 2023-04-14
US63/459,564 2023-04-14

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TW202403049A true TW202403049A (zh) 2024-01-16

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US (1) US20230398192A1 (cg-RX-API-DMAC10.html)
EP (1) EP4526439A1 (cg-RX-API-DMAC10.html)
JP (1) JP2025521111A (cg-RX-API-DMAC10.html)
KR (1) KR20250011933A (cg-RX-API-DMAC10.html)
CN (1) CN119213120A (cg-RX-API-DMAC10.html)
AU (1) AU2023273809A1 (cg-RX-API-DMAC10.html)
CA (1) CA3253612A1 (cg-RX-API-DMAC10.html)
CO (1) CO2024016901A2 (cg-RX-API-DMAC10.html)
IL (1) IL316984A (cg-RX-API-DMAC10.html)
MX (1) MX2024014114A (cg-RX-API-DMAC10.html)
TW (1) TW202403049A (cg-RX-API-DMAC10.html)
WO (1) WO2023225481A1 (cg-RX-API-DMAC10.html)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN122206466A (zh) * 2023-11-15 2026-06-12 建新公司 治疗神经变性障碍的方法
WO2026142957A1 (en) * 2024-12-24 2026-07-02 Genzyme Corporation Aav genomes encoding engineered human frataxin transgene

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
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
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
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
JP7715640B2 (ja) * 2019-05-03 2025-07-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異染性白質ジストロフィーの治療に有用な組成物
PE20220233A1 (es) * 2019-06-10 2022-02-07 Homology Medicines Inc Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismas
CN115023242B (zh) 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体

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JP2025521111A (ja) 2025-07-08
KR20250011933A (ko) 2025-01-22
MX2024014114A (es) 2024-12-06
WO2023225481A1 (en) 2023-11-23
AU2023273809A1 (en) 2025-01-02
CO2024016901A2 (es) 2024-12-19
US20230398192A1 (en) 2023-12-14
EP4526439A1 (en) 2025-03-26
CA3253612A1 (en) 2023-11-23
IL316984A (en) 2025-01-01
CN119213120A (zh) 2024-12-27

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