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EA201891137A1 - Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a - Google Patents

Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a

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Publication number
EA201891137A1
EA201891137A1 EA201891137A EA201891137A EA201891137A1 EA 201891137 A1 EA201891137 A1 EA 201891137A1 EA 201891137 A EA201891137 A EA 201891137A EA 201891137 A EA201891137 A EA 201891137A EA 201891137 A1 EA201891137 A1 EA 201891137A1
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EA
Eurasian Patent Office
Prior art keywords
gene therapy
hemophilia
options
viral vectors
increased expression
Prior art date
Application number
EA201891137A
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English (en)
Other versions
EA038288B1 (ru
Inventor
Фалько-Гюнтер Фалькнер
Франциска Хорлинг
Йоханнес Ленглер
Ханспетер Роттенштайнер
Фридрих Шайфлингер
Original Assignee
Баксалта Инкорпорейтед
Баксалта Гмбх
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Баксалта Инкорпорейтед, Баксалта Гмбх filed Critical Баксалта Инкорпорейтед
Publication of EA201891137A1 publication Critical patent/EA201891137A1/ru
Publication of EA038288B1 publication Critical patent/EA038288B1/ru

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Peptides Or Proteins (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Данное описание предлагает, среди прочего, полинуклеотиды с измененными кодонами, кодирующие варианты фактора VIII для экспрессии в клетках млекопитающих. В некоторых вариантах реализации изобретения в описании также представлены векторы для генной терапии млекопитающих и способы лечения гемофилии A.
EA201891137A 2015-11-13 2016-11-11 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a EA038288B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562255317P 2015-11-13 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

Publications (2)

Publication Number Publication Date
EA201891137A1 true EA201891137A1 (ru) 2018-12-28
EA038288B1 EA038288B1 (ru) 2021-08-05

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Application Number Title Priority Date Filing Date
EA201891137A EA038288B1 (ru) 2015-11-13 2016-11-11 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
EA202190827A EA202190827A1 (ru) 2015-11-13 2016-11-11 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a

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EA202190827A EA202190827A1 (ru) 2015-11-13 2016-11-11 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a

Country Status (18)

Country Link
US (4) US10189888B2 (ru)
EP (1) EP3374387A1 (ru)
JP (4) JP6768798B2 (ru)
KR (2) KR102484553B1 (ru)
CN (3) CN116949052A (ru)
AU (4) AU2016353353B2 (ru)
BR (1) BR112018009717B1 (ru)
CA (1) CA3005334A1 (ru)
CL (1) CL2018001299A1 (ru)
CO (1) CO2018005377A2 (ru)
EA (2) EA038288B1 (ru)
IL (3) IL281038B2 (ru)
MX (1) MX2018005982A (ru)
MY (1) MY190067A (ru)
SG (2) SG11201804070XA (ru)
TW (3) TWI777175B (ru)
WO (1) WO2017083762A1 (ru)
ZA (3) ZA201803443B (ru)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6909212B2 (ja) 2015-10-28 2021-07-28 サンガモ セラピューティクス， インコーポレイテッド 肝臓特異的コンストラクト、第ｖｉｉｉ因子発現カセット、およびその使用の方法
TWI850587B (zh) 2015-11-13 2024-08-01 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變異體之病毒載體
CN116949052A (zh) 2015-11-13 2023-10-27 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
US11779656B2 (en) * 2016-04-15 2023-10-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A
IL266264B2 (en) 2016-11-04 2023-11-01 Baxalta Inc Adeno-associated virus formulations
CA3077380A1 (en) * 2017-09-27 2019-04-04 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
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
CA3106590A1 (en) * 2018-07-16 2020-01-23 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
AU2019314513A1 (en) * 2018-08-03 2021-01-28 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
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
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
WO2020257586A2 (en) * 2019-06-20 2020-12-24 Baxalta Incorporated Method of treatment with viral-based gene therapy
WO2021084275A1 (en) * 2019-11-01 2021-05-06 Freeline Therapeutics Limited Factor viii polypeptide
US20230023826A1 (en) * 2019-12-12 2023-01-26 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
WO2023211316A1 (en) * 2022-04-28 2023-11-02 Joint Stock Company "Biocad" Codon-optimized nucleic acid encoding the 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

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
AU5772886A (en) 1985-04-12 1986-11-05 Genetics Institute Inc. Novel procoagulant proteins
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
US5610278A (en) 1986-06-24 1997-03-11 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
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
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
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
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
ES2319745T3 (es) 2001-10-05 2009-05-12 Expression Therapeutics, Llc Secuencias de acidos nucleicos y aminoacidos que codifican polipeptidos de factor viii de alto nivel de expresion, y metodos de uso.
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
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
US8198421B2 (en) 2006-06-19 2012-06-12 Asklepios Biopharmaceutical, Inc. Modified factor VIII and factor IX genes and vectors for gene therapy
CA2673459C (en) 2006-12-22 2016-09-13 Stefan Schulte Modified coagulation factors with prolonged in vivo half-life
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
EP3586868B1 (en) 2008-04-22 2023-11-08 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
JP5770161B2 (ja) 2009-04-06 2015-08-26 ノヴォ ノルディスク アー／エス 血小板への第ｖｉｉｉ因子タンパク質の標的送達
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP3560962A1 (en) 2010-07-09 2019-10-30 Bioverativ Therapeutics Inc. Processable single chain molecules and polypeptides made using same
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
NZ626945A (en) * 2012-01-12 2016-10-28 Biogen Ma Inc Chimeric factor viii polypeptides and uses thereof
EP3970737A1 (en) * 2012-01-12 2022-03-23 Bioverativ Therapeutics Inc. Reducing immunogenicity against factor viii in individuals undergoing factor viii therapy
DK3564260T5 (da) * 2012-02-15 2024-09-02 Bioverativ Therapeutics Inc Faktor viii-sammensætninger og fremgangsmåder til fremstilling og anvendelse deraf
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
JP2015518705A (ja) * 2012-04-02 2015-07-06 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ヒト疾患に関連する生物製剤およびタンパク質の産生のための修飾ポリヌクレオチド
GB201210357D0 (en) * 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
US8986991B2 (en) 2012-07-03 2015-03-24 Expression Therapeutics, Llc High yield suspension cell line, system and method for making same
US10398787B2 (en) 2012-10-26 2019-09-03 Vrije Universiteit Brussel Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
ES2959747T3 (es) 2013-02-15 2024-02-28 Bioverativ Therapeutics Inc Gen del factor VIII optimizado
CA2905739A1 (en) 2013-03-15 2014-09-25 Bayer Healthcare Llc Recombinant factor viii formulations
CN105431451B (zh) * 2013-06-24 2024-08-23 肖卫东 突变型因子viii组合物和方法
RU2710307C2 (ru) * 2013-09-12 2019-12-26 Байомарин Фармасьютикал Инк. Векторы экспрессии фактора viii на основе аденоассоциированного вируса
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
KR102698387B1 (ko) * 2015-02-06 2024-08-22 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
EP3270944B1 (en) 2015-03-17 2019-10-23 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
CN116949052A (zh) 2015-11-13 2023-10-27 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体

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