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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JP2019503649A (ja
JP2019503649A5 (https=
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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)

Priority Applications (2)

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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

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
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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JP2019503649A5 JP2019503649A5 (https=) 2019-12-05
JP7088831B2 true JP7088831B2 (ja) 2022-06-21

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JP2018521281A Active JP7088831B2 (ja) 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
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 (https=)
EP (1) EP3368560B1 (https=)
JP (3) JP7088831B2 (https=)
KR (1) KR20180090795A (https=)
CN (1) CN108368164A (https=)
AU (1) AU2016344030B2 (https=)
BR (1) BR112018008766A2 (https=)
CA (1) CA3002689A1 (https=)
CL (1) CL2018001107A1 (https=)
CO (1) CO2018005306A2 (https=)
IL (1) IL258959B2 (https=)
MX (2) MX2018005490A (https=)
MY (1) MY190926A (https=)
PE (2) PE20231949A1 (https=)
PH (1) PH12018501055A1 (https=)
RU (1) RU2745506C2 (https=)
SA (1) SA518391463B1 (https=)
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JP2022137029A (ja) * 2015-10-30 2022-09-21 スパーク セラピューティクス インコーポレイテッド 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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KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
TW202016298A (zh) 2018-05-14 2020-05-01 美商拜奧馬林製藥公司 Aav 載體於幼年受試者中之穩定表現
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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GB201900702D0 (en) 2019-01-18 2019-03-06 Univ Bristol Therapy
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SG11202109850SA (en) 2019-03-13 2021-10-28 Generation Bio Co Non-viral dna vectors and uses thereof for expressing fviii therapeutics
CN114929735A (zh) * 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
JP2023510392A (ja) * 2020-01-16 2023-03-13 武田薬品工業株式会社 フェニルケトン尿症に対するアデノ随伴ウイルスベースの遺伝子治療
GB202009039D0 (en) 2020-06-15 2020-07-29 Univ Bristol Therapy
WO2023036054A2 (en) * 2021-09-08 2023-03-16 Inspirar Limited Composition and method for treating hemophilia
KR20250007615A (ko) * 2022-04-28 2025-01-14 조인트 스탁 컴퍼니 "바이오케드" Fviii-bdd를 인코딩하는 코돈 최적화된 핵산
JP2025529195A (ja) * 2022-08-31 2025-09-04 ハイドロジーン セラピューティクス，インコーポレイテッド 流体力学的遺伝子送達のための方法および組成物
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
WO2025180508A1 (en) * 2024-02-29 2025-09-04 Shanghai Vitalgen Biopharma Co., Ltd. Human factor viii variant expression cassettes and uses thereof for treating hemophilia a

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