KR102551733B1 - B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터 - Google Patents

B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터 Download PDF

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KR102551733B1
KR102551733B1 KR1020227012028A KR20227012028A KR102551733B1 KR 102551733 B1 KR102551733 B1 KR 102551733B1 KR 1020227012028 A KR1020227012028 A KR 1020227012028A KR 20227012028 A KR20227012028 A KR 20227012028A KR 102551733 B1 KR102551733 B1 KR 102551733B1
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sequence
polypeptide
polynucleotide
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프란지스카 홀링
요하네스 랑글레
팔코 군터 포크너
한스피터 로텐슈타이너
프리드리히 쉐이프린거
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다케다 야쿠힌 고교 가부시키가이샤
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KR1020227012028A 2017-05-22 2018-05-22 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터 Active KR102551733B1 (ko)

Applications Claiming Priority (4)

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US201762509616P 2017-05-22 2017-05-22
US62/509,616 2017-05-22
PCT/US2018/033866 WO2018217731A1 (en) 2017-05-22 2018-05-22 Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b
KR1020197037828A KR102386890B1 (ko) 2017-05-22 2018-05-22 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터

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KR102551733B1 true KR102551733B1 (ko) 2023-07-06

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KR1020197037828A Active KR102386890B1 (ko) 2017-05-22 2018-05-22 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터

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GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
MX2021004277A (es) 2018-10-18 2021-09-08 Intellia Therapeutics Inc Composiciones y metodos para expresar el factor ix.
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
US11596671B2 (en) 2019-02-01 2023-03-07 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression
CN117603949A (zh) * 2019-03-19 2024-02-27 杰特创新股份有限公司 因子ix变体及其在治疗中的用途
AR119271A1 (es) * 2019-06-11 2021-12-09 Shire Human Genetic Therapies Administración del vector viral adenoasociado para el tratamiento de enfermedades mediadas por la calicreína plasmática desregulada
US20200405883A1 (en) 2019-06-20 2020-12-31 Baxalta Incorporated Method of treatment with viral-based gene therapy
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
CA3165015A1 (en) * 2020-01-16 2021-07-22 Matthias Klugmann Adeno associated virus based gene therapy for phenylketonuria
TW202246505A (zh) * 2021-03-05 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途
AU2022298827A1 (en) * 2021-06-23 2024-02-08 Frontera Therapeutics, Inc. Composition and method for treating hemophilia b
JP2024523707A (ja) * 2021-07-08 2024-06-28 テナヤ セラピューティクス， インコーポレイテッド 遺伝子治療のための最適化された発現カセット
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
US20230149563A1 (en) * 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
WO2025143742A1 (ko) * 2023-12-26 2025-07-03 삼성바이오에피스 주식회사 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드

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