KR20220131522A - 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 - Google Patents

완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 Download PDF

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KR20220131522A
KR20220131522A KR1020227025630A KR20227025630A KR20220131522A KR 20220131522 A KR20220131522 A KR 20220131522A KR 1020227025630 A KR1020227025630 A KR 1020227025630A KR 20227025630 A KR20227025630 A KR 20227025630A KR 20220131522 A KR20220131522 A KR 20220131522A
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스티븐 조셉 파콜라
파울로 팔라벨라
마리-로르 뉴보렛
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리젠엑스바이오 인크.
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KR1020227025630A 2020-01-22 2021-01-20 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 Pending KR20220131522A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062964351P 2020-01-22 2020-01-22
US62/964,351 2020-01-22
US202063086145P 2020-10-01 2020-10-01
US63/086,145 2020-10-01
PCT/US2021/014129 WO2021150570A1 (en) 2020-01-22 2021-01-20 Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)

Publications (1)

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KR20220131522A true KR20220131522A (ko) 2022-09-28

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KR1020227025630A Pending KR20220131522A (ko) 2020-01-22 2021-01-20 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료

Country Status (9)

Country Link
US (1) US20230059395A1 (https=)
EP (1) EP4093428A1 (https=)
JP (1) JP2023511382A (https=)
KR (1) KR20220131522A (https=)
AU (1) AU2021211416A1 (https=)
BR (1) BR112022014563A2 (https=)
CA (1) CA3167685A1 (https=)
IL (1) IL294625A (https=)
WO (1) WO2021150570A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA50016A (fr) 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
CA3167741A1 (en) * 2020-01-29 2021-08-05 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells

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PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
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EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
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
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
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HUE052676T2 (hu) 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
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EP3411488A1 (en) * 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
JP7042814B2 (ja) * 2016-11-01 2022-03-28 日本光電工業株式会社 患者の頭部に配置されて当該患者の脳の皮質組織の脈動を測定するためのトランスデューサを含むシステム
EP3541946A1 (en) * 2016-11-15 2019-09-25 Regents Of The University Of Minnesota Method for improving neurological function in mpsi and mpsii and other neurological disorders
CA3049915A1 (en) * 2017-01-31 2018-08-09 Stephen YOO Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
MA50016A (fr) * 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i

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JP2023511382A (ja) 2023-03-17
AU2021211416A1 (en) 2022-08-11
BR112022014563A2 (pt) 2022-09-13
WO2021150570A1 (en) 2021-07-29
US20230059395A1 (en) 2023-02-23
EP4093428A1 (en) 2022-11-30
IL294625A (en) 2022-09-01
CA3167685A1 (en) 2021-07-29

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