JP7088831B2 - 核酸変種、アデノ随伴ウイルスベクター及び医薬組成物 - Google Patents

核酸変種、アデノ随伴ウイルスベクター及び医薬組成物 Download PDF

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JP7088831B2
JP7088831B2 JP2018521281A JP2018521281A JP7088831B2 JP 7088831 B2 JP7088831 B2 JP 7088831B2 JP 2018521281 A JP2018521281 A JP 2018521281A JP 2018521281 A JP2018521281 A JP 2018521281A JP 7088831 B2 JP7088831 B2 JP 7088831B2
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aav
fviii
nucleic acid
vector
cpg
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JP2019503649A5 (enExample
JP2019503649A (ja
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シャビエル アングエラ，
サム シエン－イ シェン，
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Spark Therapeutics Inc
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JP2018521281A 2015-10-30 2016-10-31 核酸変種、アデノ随伴ウイルスベクター及び医薬組成物 Active JP7088831B2 (ja)

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JP2022093877A JP2022137029A (ja) 2015-10-30 2022-06-09 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用
JP2024131729A JP2024170416A (ja) 2015-10-30 2024-08-08 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用

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US201562249001P 2015-10-30 2015-10-30
US62/249,001 2015-10-30
US201662331872P 2016-05-04 2016-05-04
US62/331,872 2016-05-04
US201662349532P 2016-06-13 2016-06-13
US62/349,532 2016-06-13
US201662357874P 2016-07-01 2016-07-01
US62/357,874 2016-07-01
PCT/US2016/059793 WO2017075619A1 (en) 2015-10-30 2016-10-31 Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders

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JP2022093877A Pending JP2022137029A (ja) 2015-10-30 2022-06-09 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用
JP2024131729A Pending JP2024170416A (ja) 2015-10-30 2024-08-08 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用

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JP2024131729A Pending JP2024170416A (ja) 2015-10-30 2024-08-08 ＣｐＧ減少第ＶＩＩＩ因子変種、組成物並びに止血障害の処置のための方法及び使用

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US (3) US20180312571A1 (enExample)
EP (1) EP3368560A4 (enExample)
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KR (1) KR20180090795A (enExample)
CN (1) CN108368164A (enExample)
AU (1) AU2016344030B2 (enExample)
BR (1) BR112018008766A2 (enExample)
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TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
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MA54951A (fr) 2019-02-15 2021-12-22 Bayer Healthcare Llc Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
MA55313A (fr) 2019-03-13 2022-01-19 Generation Bio Co Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii
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CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2025164792A1 (ja) * 2024-02-02 2025-08-07 学校法人自治医科大学 高比活性／高分泌発現型の第ｖｉｉｉ因子改変体、高発現型の第ｖｉｉｉ因子コード核酸及びそれらの用途

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