JP2020508289A - 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 - Google Patents

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 Download PDF

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JP2020508289A
JP2020508289A JP2019541264A JP2019541264A JP2020508289A JP 2020508289 A JP2020508289 A JP 2020508289A JP 2019541264 A JP2019541264 A JP 2019541264A JP 2019541264 A JP2019541264 A JP 2019541264A JP 2020508289 A JP2020508289 A JP 2020508289A
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idua
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ヨー ステファン
ヨー ステファン
ロバート ラインハルト リッキー
ロバート ラインハルト リッキー
マシュー シンプソン クラン
マシュー シンプソン クラン
ウー ズフチュン
ウー ズフチュン
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レジェンクスバイオ インコーポレーテッド
レジェンクスバイオ インコーポレーテッド
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)

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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)

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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

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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)

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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

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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=)

Cited By (1)

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JP2023552443A (ja) * 2020-12-07 2023-12-15 スペースクラフト セブン リミテッド ライアビリティ カンパニー ダノン病の治療

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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)
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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JP2023552443A (ja) * 2020-12-07 2023-12-15 スペースクラフト セブン リミテッド ライアビリティ カンパニー ダノン病の治療

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IL268076A (en) 2019-09-26
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CA3049915A1 (en) 2018-08-09
WO2018144441A1 (en) 2018-08-09
UY37587A (es) 2018-08-31
EP3576768A1 (en) 2019-12-11
MA47436A (fr) 2019-12-11
KR20250022877A (ko) 2025-02-17
SG11201906452PA (en) 2019-08-27
US20240050537A1 (en) 2024-02-15
AU2018216807A1 (en) 2019-08-08
EP3576768A4 (en) 2020-11-04
KR20190109506A (ko) 2019-09-25
JP2024112876A (ja) 2024-08-21
BR112019015482A2 (pt) 2020-03-31

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