JP2020508289A5 - - Google Patents

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Publication number
JP2020508289A5
JP2020508289A5 JP2019541264A JP2019541264A JP2020508289A5 JP 2020508289 A5 JP2020508289 A5 JP 2020508289A5 JP 2019541264 A JP2019541264 A JP 2019541264A JP 2019541264 A JP2019541264 A JP 2019541264A JP 2020508289 A5 JP2020508289 A5 JP 2020508289A5
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JP
Japan
Prior art keywords
human
idua
pharmaceutical composition
subject
expression 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
JP2019541264A
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English (en)
Japanese (ja)
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JP2020508289A (ja
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Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/015910 external-priority patent/WO2018144441A1/en
Publication of JP2020508289A publication Critical patent/JP2020508289A/ja
Publication of JP2020508289A5 publication Critical patent/JP2020508289A5/ja
Priority to JP2024079088A priority Critical patent/JP2024112876A/ja
Pending legal-status Critical Current

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JP2019541264A 2017-01-31 2018-01-30 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 Pending JP2020508289A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024079088A JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201762452769P 2017-01-31 2017-01-31
US62/452,769 2017-01-31
US201762485655P 2017-04-14 2017-04-14
US62/485,655 2017-04-14
US201762529366P 2017-07-06 2017-07-06
US62/529,366 2017-07-06
US201762579690P 2017-10-31 2017-10-31
US62/579,690 2017-10-31
US201862616234P 2018-01-11 2018-01-11
US62/616,234 2018-01-11
PCT/US2018/015910 WO2018144441A1 (en) 2017-01-31 2018-01-30 Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024079088A Division JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Publications (2)

Publication Number Publication Date
JP2020508289A JP2020508289A (ja) 2020-03-19
JP2020508289A5 true JP2020508289A5 (https=) 2021-03-18

Family

ID=63041038

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019541264A Pending JP2020508289A (ja) 2017-01-31 2018-01-30 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
JP2024079088A Pending JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024079088A Pending JP2024112876A (ja) 2017-01-31 2024-05-15 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療

Country Status (12)

Country Link
US (2) US20190358303A1 (https=)
EP (1) EP3576768A4 (https=)
JP (2) JP2020508289A (https=)
KR (2) KR20250022877A (https=)
AU (1) AU2018216807A1 (https=)
BR (1) BR112019015482A2 (https=)
CA (1) CA3049915A1 (https=)
IL (1) IL268076B2 (https=)
MA (1) MA47436A (https=)
SG (1) SG11201906452PA (https=)
UY (1) UY37587A (https=)
WO (1) WO2018144441A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
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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
IL280198B2 (en) * 2018-07-18 2026-01-01 Regenxbio Inc Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus)
WO2021150570A1 (en) * 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
CA3201247A1 (en) * 2020-12-07 2022-06-16 Gaurav Shah Treatment of danon disease
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CA3236188A1 (en) * 2021-10-27 2023-05-04 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i

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