MX2016003262A - Vectores del factor viii del virus adeno-asociado. - Google Patents

Vectores del factor viii del virus adeno-asociado.

Info

Publication number
MX2016003262A
MX2016003262A MX2016003262A MX2016003262A MX2016003262A MX 2016003262 A MX2016003262 A MX 2016003262A MX 2016003262 A MX2016003262 A MX 2016003262A MX 2016003262 A MX2016003262 A MX 2016003262A MX 2016003262 A MX2016003262 A MX 2016003262A
Authority
MX
Mexico
Prior art keywords
factor viii
adeno
associated virus
vectors
virus factor
Prior art date
Application number
MX2016003262A
Other languages
English (en)
Inventor
Cameron Colosi Peter
Nathwani Amit
Mcintosh Jenny
Tuddenham Edward
Original Assignee
Biomarin Pharm Inc
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.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=51585244&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=MX2016003262(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of MX2016003262A publication Critical patent/MX2016003262A/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36Blood coagulation or fibrinolysis factors
    • A61K38/37Factors VIII
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente invención se refiere a vectores mejorados del Factor VIII (FVIII) del virus adeno-asociado (AAV), que incluyen vectores del FVIII del AAV que producen un polipéptido del Factor VIII funcional y vectores del FVIII del AAV con alta actividad de expresión.
MX2016003262A 2013-09-12 2014-09-10 Vectores del factor viii del virus adeno-asociado. MX2016003262A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361877042P 2013-09-12 2013-09-12
PCT/US2014/054960 WO2015038625A1 (en) 2013-09-12 2014-09-10 Adeno-associated virus factor viii vectors

Publications (1)

Publication Number Publication Date
MX2016003262A true MX2016003262A (es) 2016-09-29

Family

ID=51585244

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2016003262A MX2016003262A (es) 2013-09-12 2014-09-10 Vectores del factor viii del virus adeno-asociado.
MX2021012014A MX2021012014A (es) 2013-09-12 2016-03-11 Vectores del factor viii del virus adeno-asociado.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2021012014A MX2021012014A (es) 2013-09-12 2016-03-11 Vectores del factor viii del virus adeno-asociado.

Country Status (33)

Country Link
US (5) US9504762B2 (es)
EP (2) EP3044231B1 (es)
JP (4) JP6735672B2 (es)
KR (2) KR20230006058A (es)
CN (2) CN112538501A (es)
AR (1) AR097657A1 (es)
AU (1) AU2014318890B2 (es)
CA (3) CA3178384A1 (es)
CL (1) CL2016000502A1 (es)
CY (2) CY1124213T1 (es)
DK (1) DK3044231T3 (es)
ES (1) ES2813698T3 (es)
FI (1) FIC20230007I1 (es)
FR (1) FR23C1007I2 (es)
HK (1) HK1226416A1 (es)
HR (1) HRP20201324T1 (es)
HU (2) HUE050484T2 (es)
IL (2) IL244404B (es)
LT (2) LT3044231T (es)
MX (2) MX2016003262A (es)
NL (1) NL301222I2 (es)
NO (1) NO2023003I1 (es)
NZ (1) NZ716549A (es)
PE (1) PE20160769A1 (es)
PH (1) PH12016500328A1 (es)
PL (1) PL3044231T3 (es)
PT (1) PT3044231T (es)
RS (1) RS61039B1 (es)
RU (1) RU2710307C2 (es)
SG (1) SG11201601932UA (es)
SI (1) SI3044231T1 (es)
TW (1) TWI632239B (es)
WO (1) WO2015038625A1 (es)

Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
AU2016232146B2 (en) * 2015-03-17 2021-11-04 Vrije Universiteit Brussel Optimized liver-specific expression systems for FVIII and FIX
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
WO2016168728A2 (en) * 2015-04-16 2016-10-20 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
PE20181338A1 (es) 2015-09-24 2018-08-21 Biomarin Pharm Inc Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
PE20181206A1 (es) 2015-10-28 2018-07-23 Sangamo Therapeutics Inc Construcciones especificas del higado, casetes de expresion del factor viii y metodos de uso de estos
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
JP6695426B2 (ja) * 2015-11-13 2020-05-20 バクスアルタ インコーポレイテッド 組換えfviii変異型をコードする血友病a遺伝子治療用高発現ウイルスベクター
BR112018009717B1 (pt) * 2015-11-13 2021-12-14 Takeda Pharmaceutical Company Limited Polinucleotídeo, vetor de vírus adeno-associado, partícula de um vírus adeno-associado, métodos para produzir uma partícula de vírus adeno-associado e para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado
CN109562191A (zh) * 2016-04-15 2019-04-02 宾夕法尼亚州大学信托人 用于治疗血友病a的基因疗法
CA3023473A1 (en) * 2016-05-25 2017-11-30 Lonza Houston Inc. Methods for isolating adeno-associated virus using a polydiallyldialkylammonium salt
EP3491008A2 (en) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018312565A1 (en) * 2017-08-01 2020-02-27 Spark Therapeutics, Inc. Factor VIII (FVIII) gene therapy methods
KR20210008501A (ko) 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법
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
JP2021523198A (ja) 2018-05-14 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド 幼若対象におけるaavベクターの安定的発現
WO2020018419A1 (en) 2018-07-16 2020-01-23 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
AU2018434176A1 (en) * 2018-07-27 2021-01-28 Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh Improved concepts for the treatment of genetic disorders with high-capacity plal-generated gold nanoparticles
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
US20210348135A1 (en) * 2018-10-12 2021-11-11 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
WO2020150375A1 (en) 2019-01-16 2020-07-23 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11202108357PA (en) 2019-02-15 2021-08-30 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
AU2020234713A1 (en) * 2019-03-13 2021-11-04 Generation Bio Co. Non-viral DNA vectors and uses thereof for expressing FVIII therapeutics
JP6831943B1 (ja) 2019-10-24 2021-02-17 三ツ星ベルト株式会社 摩擦伝動ベルトおよびその製造方法
EP4051704A2 (en) * 2019-11-01 2022-09-07 Freeline Therapeutics Limited Factor viii construct
CN114981299A (zh) 2019-12-12 2022-08-30 武田药品工业株式会社 使用编码具有增加的表达的重组fviii变体的病毒载体的a型血友病的基因疗法
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
JP2023548067A (ja) 2020-11-02 2023-11-15 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルスを濃縮するためのプロセス
WO2022264040A1 (en) 2021-06-14 2022-12-22 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
EP4359547A1 (en) * 2021-06-22 2024-05-01 Pfizer Inc. Production of adeno-associated virus vector in insect cells
KR20240021951A (ko) * 2021-07-28 2024-02-19 바이오마린 파머수티컬 인크. 대규모 아데노-관련 바이러스 생산 시스템
JP2024532262A (ja) * 2021-08-23 2024-09-05 バイオベラティブ セラピューティクス インコーポレイテッド 最適化第viii因子遺伝子
WO2023036054A2 (en) * 2021-09-08 2023-03-16 Inspirar Limited Composition and method for treating hemophilia
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
AR128760A1 (es) * 2022-03-11 2024-06-12 Homology Medicines Inc Vectores de expresión con promotores dobles bidireccionales y sus usos
WO2023205300A2 (en) * 2022-04-20 2023-10-26 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
WO2024064856A1 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of cardiomyopathy with aav gene therapy vectors
WO2024064863A2 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
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
CN118715025A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于增强基因表达的构建体
CN116555266A (zh) * 2023-06-09 2023-08-08 呈诺再生医学科技(北京)有限公司 肝脏特异性表达的重组启动子和载体及其在肝脏相关疾病治疗中的应用
CN118064436B (zh) * 2024-04-18 2024-07-26 上海凌医生物科技有限公司 一种增强基因表达的嵌合启动子

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0127839B1 (en) 1983-05-27 1992-07-15 THE TEXAS A&M UNIVERSITY SYSTEM Method for producing a recombinant baculovirus expression vector
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
ZA848495B (en) 1984-01-31 1985-09-25 Idaho Res Found Production of polypeptides in insect cells
RU2219241C2 (ru) * 1993-07-13 2003-12-20 Рон-Пуленк Роре С.А. Дефектный рекомбинантный аденовирусный вектор (варианты)
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
US6383794B1 (en) * 1998-08-24 2002-05-07 Uab Research Foundation Methods of producing high titer recombinant adeno-associated virus
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
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
DK1804839T3 (da) 2004-09-22 2012-04-10 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
CN1286981C (zh) * 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
EP1739179A1 (en) * 2005-06-30 2007-01-03 Octapharma AG Serum-free stable transfection and production of recombinant human proteins in human cell lines
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
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2768521B1 (en) * 2011-10-18 2016-07-13 CSL Behring GmbH Combined use of a sulfated glycosaminoglycan and a hyaluronidase for improving the bioavailability of factor viii
CN103215308B (zh) * 2013-02-01 2015-12-23 中国科学院苏州生物医学工程技术研究所 表达重组人fviii的整合质粒、细胞株及其构建方法和应用
EP3044231B1 (en) 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii

Also Published As

Publication number Publication date
IL244404B (en) 2021-10-31
LTPA2023508I1 (es) 2023-04-11
CA3178379A1 (en) 2015-03-19
TW201546285A (zh) 2015-12-16
JP2023133597A (ja) 2023-09-22
BR112016005576A2 (pt) 2017-09-12
PL3044231T3 (pl) 2021-01-11
US20180161403A1 (en) 2018-06-14
HUE050484T2 (hu) 2020-12-28
CY2023006I2 (el) 2023-11-15
US20220339262A1 (en) 2022-10-27
RS61039B1 (sr) 2020-12-31
CN105636981A (zh) 2016-06-01
PH12016500328A1 (en) 2016-05-02
SG11201601932UA (en) 2016-04-28
CN112538501A (zh) 2021-03-23
JP2021072871A (ja) 2021-05-13
PE20160769A1 (es) 2016-08-12
TWI632239B (zh) 2018-08-11
RU2019141647A (ru) 2020-01-27
FIC20230007I1 (fi) 2023-02-08
EP3044231B1 (en) 2020-08-05
JP7523211B2 (ja) 2024-07-26
US9504762B2 (en) 2016-11-29
EP3044231A1 (en) 2016-07-20
CY1124213T1 (el) 2022-03-24
US20170095538A1 (en) 2017-04-06
US10463718B2 (en) 2019-11-05
AU2014318890B2 (en) 2019-03-07
CA2921232A1 (en) 2015-03-19
CA2921232C (en) 2023-03-14
SI3044231T1 (sl) 2020-12-31
IL244404A0 (en) 2016-04-21
NZ716549A (en) 2022-10-28
RU2016113702A (ru) 2017-10-17
CA3178384A1 (en) 2015-03-19
JP6735672B2 (ja) 2020-08-05
HRP20201324T1 (hr) 2020-11-27
LT3044231T (lt) 2020-11-10
US11406690B2 (en) 2022-08-09
EP3770169A1 (en) 2021-01-27
NL301222I2 (nl) 2023-10-19
HUS2300003I1 (hu) 2023-01-28
BR112016005576A8 (pt) 2022-10-18
ES2813698T3 (es) 2021-03-24
FR23C1007I2 (fr) 2024-06-14
NO2023003I1 (no) 2023-01-12
ES2813698T8 (es) 2022-01-26
RU2710307C2 (ru) 2019-12-26
JP2019193675A (ja) 2019-11-07
RU2019141647A3 (es) 2020-05-18
KR102484396B1 (ko) 2023-01-04
IL286859A (en) 2021-10-31
CN105636981B (zh) 2020-11-06
WO2015038625A1 (en) 2015-03-19
HK1226416A1 (zh) 2017-09-29
CY2023006I1 (el) 2023-06-09
US20150071883A1 (en) 2015-03-12
DK3044231T3 (da) 2020-09-07
KR20230006058A (ko) 2023-01-10
KR20160049015A (ko) 2016-05-04
AR097657A1 (es) 2016-04-06
AU2014318890A1 (en) 2016-02-25
US20200101140A1 (en) 2020-04-02
NL301222I1 (es) 2023-03-01
FR23C1007I1 (fr) 2023-03-24
CL2016000502A1 (es) 2016-10-21
JP2016534739A (ja) 2016-11-10
MX2021012014A (es) 2021-10-22
PT3044231T (pt) 2020-08-27

Similar Documents

Publication Publication Date Title
MX2021012014A (es) Vectores del factor viii del virus adeno-asociado.
PH12018502664A1 (en) Variant adeno-associated viruses and methods of using
ZA201801657B (en) Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
TR201901582T4 (tr) Kapsid
MX357202B (es) Composicion de particula tipo virus.
EP3221456A4 (en) Genome-modified recombinant adeno-associated virus vectors
FR3021319B1 (fr) Peptides, compositions les comprenant et utilisations notamment cosmetiques
SG10201909716RA (en) Modified j-chain
MX2016001094A (es) Nanoemulsion de aminosilicona.
EP3166959A4 (en) Targeting peptides that bind s. mutans, constructs comprising such peptides and uses thereof
ZA201803551B (en) Personal care composition comprising taurine, arginine, glycine
IL266526A (en) Recombinant virus, preparations containing it and its uses
SG11201509743WA (en) Lentiviral vectors containing an mhc class i, mhc class ii, or beta-2 microglobulin upstream promoter sequence
IL272655A (en) Recombinant adeno-associated vectors
MX2015012905A (es) Formulaciones de factor viii recombinantes.
Naimisharay Apne Apne Pinjare-2
NZ756883A (en) Adeno-associated virus factor viii vectors
BR112016007816A2 (pt) composições peptídicas inovadoras
Joshi Jahaj Ka Panchhi
എം രാമചന്ദ്രൻ പിള്ള പരിണയം-ഉരൽപുരയിൽ നിന്ന് ഒരുറച്ച ശബ്ദം
若梅 We are family
Haryanto Cover AJTM Vol 8 No 2 2105
ލިޔުންތެރިން ފަލަޞްޠީން-ޤުރުއާނާއި ސުންނަތުން
Win The Yati (Vol 10 No 108) 2013/03
EA201991576A1 (ru) Частичные агонисты инсулинового рецептора