JP2023511382A - 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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JP2023511382A
JP2023511382A JP2022544318A JP2022544318A JP2023511382A JP 2023511382 A JP2023511382 A JP 2023511382A JP 2022544318 A JP2022544318 A JP 2022544318A JP 2022544318 A JP2022544318 A JP 2022544318A JP 2023511382 A JP2023511382 A JP 2023511382A
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ジョセフ パコーラ スティーブン
ファラベーラ パウロ
ネボレ マリー‐ロール
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レジェンクスバイオ インコーポレーテッド
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JP2022544318A 2020-01-22 2021-01-20 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 JP2023511382A (ja)

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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)

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JP2023511382A true JP2023511382A (ja) 2023-03-17
JPWO2021150570A5 JPWO2021150570A5 (https=) 2024-02-16
JP2023511382A5 JP2023511382A5 (https=) 2024-02-16

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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=)

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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
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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WO2019010335A1 (en) * 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
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JP2020500928A (ja) * 2016-11-15 2020-01-16 リジェネクスバイオ インコーポレイテッド Ｍｐｓｉおよびｍｐｓｉｉならびに他の神経障害において神経機能を改善するための方法
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BR112022014563A2 (pt) 2022-09-13
WO2021150570A1 (en) 2021-07-29
US20230059395A1 (en) 2023-02-23
KR20220131522A (ko) 2022-09-28
EP4093428A1 (en) 2022-11-30
IL294625A (en) 2022-09-01
CA3167685A1 (en) 2021-07-29

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