TWI695069B - 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 - Google Patents

用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 Download PDF

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TWI695069B
TWI695069B TW105136965A TW105136965A TWI695069B TW I695069 B TWI695069 B TW I695069B TW 105136965 A TW105136965 A TW 105136965A TW 105136965 A TW105136965 A TW 105136965A TW I695069 B TWI695069 B TW I695069B
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seq
factor viii
sequence
polynucleotide
amino acid
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TW105136965A
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TW201725266A (zh
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法科－甘特 福克諾
法蘭西斯卡 霍林
喬安斯 藍格勒
漢斯彼得 羅騰史戴納
弗利茲 許夫利格
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美商百克莎塔股份有限公司
瑞士商百克莎塔有限公司
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TW105136965A 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體 TWI695069B (zh)

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US201562255317P 2015-11-13 2015-11-13
US62/255,317 2015-11-13

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TW201725266A TW201725266A (zh) 2017-07-16
TWI695069B true TWI695069B (zh) 2020-06-01

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TW109118505A TWI777175B (zh) 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體
TW111137316A TW202302627A (zh) 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體
TW105136965A TWI695069B (zh) 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體

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TW109118505A TWI777175B (zh) 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體
TW111137316A TW202302627A (zh) 2015-11-13 2016-11-11 用於ａ型血友病基因治療之編碼表現增加之重組ｆｖｉｉｉ變異體之病毒載體

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Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115141836B (zh) 2015-10-28 2024-08-27 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法
MX2018005969A (es) 2015-11-13 2018-11-29 Baxalta Inc Vectores virales que codifican variantes del factor fviii recombinantes con mayor expresión para la terapia génica de hemofilia a.
EA202190827A1 (ru) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
WO2017180861A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsulvania Gene therapy for treating hemophilia b
KR102450833B1 (ko) * 2016-04-15 2022-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 혈우병 a 치료용 유전자 요법
WO2018128689A1 (en) 2016-11-04 2018-07-12 Baxalta Incorporated Adeno-associated virus formulations
WO2019067766A1 (en) * 2017-09-27 2019-04-04 Sigilon Therapeutics, Inc. METHODS, COMPOSITIONS AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS
US20200248179A1 (en) * 2017-10-02 2020-08-06 Research Institute At Nationwide Children's Hospital MiRNA Detargeting System for Tissue Specific Interference
KR102105145B1 (ko) * 2017-10-13 2020-05-13 영남대학교 산학협력단 촬영 장치 및 용기
KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
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
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150071883A1 (en) * 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
JPH0788399B2 (ja) 1985-04-12 1995-09-27 ジェネティックス・インスチチュ−ト・インコ−ポレ−テッド 新規プロコアギュラント蛋白質
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5543502A (en) 1986-06-24 1996-08-06 Novo Nordisk A/S Process for producing a coagulation active complex of factor VIII fragments
US6060447A (en) 1987-05-19 2000-05-09 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US6346513B1 (en) 1987-06-12 2002-02-12 Baxter Trading Gmbh Proteins with factor VIII activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
US5171844A (en) 1987-06-12 1992-12-15 Gist-Brocades N.W. Proteins with factor viii activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
CA2162497A1 (en) 1993-06-10 1994-12-22 Sheila Connelly Adenoviral vectors for treatment of hemophilia
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
US6818439B1 (en) 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
US5952198A (en) * 1995-05-04 1999-09-14 Bayer Corporation Production of recombinant Factor VIII in the presence of liposome-like substances of mixed composition
SE9503380D0 (sv) 1995-09-29 1995-09-29 Pharmacia Ab Protein derivatives
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
US6114148C1 (en) 1996-09-20 2012-05-01 Gen Hospital Corp High level expression of proteins
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6924365B1 (en) 1998-09-29 2005-08-02 Transkaryotic Therapies, Inc. Optimized messenger RNA
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US20060099685A1 (en) 1999-04-15 2006-05-11 Yallop Christopher A Recombinant expression of factor VIII in human cells
CN1179976C (zh) * 2000-12-29 2004-12-15 中国科学院上海生物化学研究所 产生凝血因子ⅷ的生产方法和宿主细胞
EP1572889B1 (en) 2001-10-05 2008-12-17 Expression Therapeutics, LLC Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
US7709224B2 (en) * 2003-06-03 2010-05-04 Biosante Pharmaceuticals, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
EP2037892B1 (en) 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
KR101542752B1 (ko) 2006-12-22 2015-08-10 체에스엘 베링 게엠베하 연장된 생체내 반감기를 갖는 변형된 응고 인자
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
JP5797551B2 (ja) 2008-04-22 2015-10-21 フエー・イー・ベー・フエー・ゼツト・ウエー 肝特異的核酸調節要素ならびにその方法および用途
EP2417155B1 (en) 2009-04-06 2013-06-19 Novo Nordisk A/S Targeted delivery of factor viii proteins to platelets
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
AU2011274423B2 (en) 2010-07-09 2016-02-11 Bioverativ Therapeutics Inc. Chimeric clotting factors
US20130017997A1 (en) 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
US10656167B2 (en) 2011-07-25 2020-05-19 Bioverativ Therapeutics Inc. Assays to monitor bleeding disorders
HUE041595T2 (hu) * 2012-01-12 2019-05-28 Bioverativ Therapeutics Inc A VIII-as faktorral szembeni immunogenitás csökkentése a VIII-as faktorterápiát kapó egyéneknél
HRP20191920T1 (hr) 2012-01-12 2020-01-10 Bioverativ Therapeutics Inc. Kimerni polipeptidi faktora viii i njihova uporaba
CN119192402A (zh) * 2012-02-15 2024-12-27 比奥贝拉蒂治疗公司 因子viii组合物及其制备和使用方法
CN104520428B (zh) 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
HK1206612A1 (en) * 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
GB201210357D0 (en) * 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
WO2014008172A2 (en) 2012-07-03 2014-01-09 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
JP6454643B2 (ja) 2012-10-26 2019-01-16 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用
EP3513802B1 (en) 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
PL2956477T5 (pl) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika viii
RU2015144076A (ru) 2013-03-15 2017-04-24 БАЙЕР ХелсКер ЛЛСи Композиции рекомбинантного фактора viii
LT3013855T (lt) 2013-06-24 2021-01-25 Xiao, Weidong Mutavusio viii faktoriaus kompozicijos ir metodai
CN107427557B (zh) 2014-08-13 2022-01-04 费城儿童医院 用于包装和表达变体因子viii以治疗血友病的改良表达组件
KR102554850B1 (ko) * 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
ES2764453T3 (es) 2015-03-17 2020-06-03 Univ Brussel Vrije Sistemas de expresión específica de hígado optimizados para FVIII y FIX
EA202190827A1 (ru) 2015-11-13 2021-11-30 Баксалта Инкорпорейтед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150071883A1 (en) * 2013-09-12 2015-03-12 Biomarin Pharmaceutical Inc. Adeno-Associated Virus Factor VIII Vectors

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