KR102484553B1 - A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터 - Google Patents

A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터 Download PDF

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KR102484553B1
KR102484553B1 KR1020187015189A KR20187015189A KR102484553B1 KR 102484553 B1 KR102484553 B1 KR 102484553B1 KR 1020187015189 A KR1020187015189 A KR 1020187015189A KR 20187015189 A KR20187015189 A KR 20187015189A KR 102484553 B1 KR102484553 B1 KR 102484553B1
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factor viii
polynucleotide
sequence
nucleotide sequence
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팔코-귄터 팔크너
프란치스카 호링
조하네스 렝글러
한스페터 로텐스타이너
프리드리히 샤이프링거
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다케다 야쿠힌 고교 가부시키가이샤
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KR1020187015189A 2015-11-13 2016-11-11 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터 Active KR102484553B1 (ko)

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KR1020227046419A KR20230008256A (ko) 2015-11-13 2016-11-11 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562255317P 2015-11-13 2015-11-13
US62/255,317 2015-11-13
PCT/US2016/061684 WO2017083762A1 (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

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KR1020227046419A Division KR20230008256A (ko) 2015-11-13 2016-11-11 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터

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KR20180070699A KR20180070699A (ko) 2018-06-26
KR102484553B1 true KR102484553B1 (ko) 2023-01-04

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KR1020227046419A Ceased KR20230008256A (ko) 2015-11-13 2016-11-11 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터
KR1020187015189A Active KR102484553B1 (ko) 2015-11-13 2016-11-11 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터

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KR20210034013A (ko) * 2018-07-16 2021-03-29 박스알타 인코퍼레이티드 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법
WO2020028830A1 (en) * 2018-08-03 2020-02-06 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
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TWI851647B (zh) * 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
BR112021015776A2 (pt) * 2019-06-05 2021-11-09 Bayer Healthcare Llc Edição de gene para hemofilia a com expressão de fator viii melhorada
EP3986481A2 (en) * 2019-06-20 2022-04-27 Takeda Pharmaceutical Company Limited Method of treatment with viral-based gene therapy
CN114901686A (zh) * 2019-11-01 2022-08-12 自由行疗法有限公司 因子viii多肽
EP4073106A2 (en) * 2019-12-12 2022-10-19 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
WO2022211604A1 (ko) * 2021-04-02 2022-10-06 연세대학교 산학협력단 Fe-fviii 변이 유전자로 교정된 줄기세포, 이로부터 분화된 내피 세포 및 이를 포함하는 혈우병 예방 또는 치료용 약제학적 조성물
KR102421131B1 (ko) * 2021-05-28 2022-07-15 알엔에이진(주) 세포내에서 장시간동안 존재할 수 있는 외부 도입 mRNA를 선별하는 방법
JP2024544499A (ja) * 2021-11-25 2024-12-03 四川至善唯新生物科技有限公司 遺伝子工学的改変により得られた、分泌能と凝固活性を向上させた組換えヒト第ｖｉｉｉ因子
MA68586A1 (fr) * 2022-04-28 2025-04-30 Joint Stock Company "Biocad" Acide nucléique à codons optimisés codant pour fviii-bdd
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
WO2024191428A1 (en) 2023-03-14 2024-09-19 Sigilon Therapeutics, Inc. Hydrogel capsules comprising covalently photocrosslinked polysaccharides and islet cells
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

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