SG11201804064WA - Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a - Google Patents

Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Info

Publication number
SG11201804064WA
SG11201804064WA SG11201804064WA SG11201804064WA SG11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA SG 11201804064W A SG11201804064W A SG 11201804064WA
Authority
SG
Singapore
Prior art keywords
international
rule
gene therapy
hemophilia
vienna
Prior art date
Application number
SG11201804064WA
Other languages
English (en)
Inventor
Falko-Günter Falkner
Franziska Horling
Johannes Lengler
Hanspeter Rottensteiner
Friedrich Scheiflinger
Original Assignee
Baxalta Inc
Baxalta GmbH
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
Application filed by Baxalta Inc, Baxalta GmbH filed Critical Baxalta Inc
Publication of SG11201804064WA publication Critical patent/SG11201804064WA/en

Links

Classifications

    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0058—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
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
SG11201804064WA 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a SG11201804064WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562255323P 2015-11-13 2015-11-13
PCT/US2016/061688 WO2017083764A1 (fr) 2015-11-13 2016-11-11 Vecteurs viraux codant pour des variants du facteur viii de recombinaison avec une expression augmentée pour la thérapie génique de l'hémophilie a

Publications (1)

Publication Number Publication Date
SG11201804064WA true SG11201804064WA (en) 2018-06-28

Family

ID=57539606

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201804064WA SG11201804064WA (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Country Status (18)

Country Link
US (1) US10189889B2 (fr)
EP (1) EP3374388A1 (fr)
JP (1) JP6695426B2 (fr)
KR (1) KR102404550B1 (fr)
CN (1) CN108602876B (fr)
AU (1) AU2016354550B2 (fr)
BR (1) BR112018009732A8 (fr)
CA (1) CA3005565A1 (fr)
CL (2) CL2018001301A1 (fr)
CO (1) CO2018005380A2 (fr)
EA (2) EA036944B1 (fr)
IL (1) IL259295A (fr)
MX (1) MX2018005969A (fr)
NZ (1) NZ742555A (fr)
SG (1) SG11201804064WA (fr)
TW (2) TWI823830B (fr)
WO (1) WO2017083764A1 (fr)
ZA (1) ZA201803442B (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
EP3923992A1 (fr) 2019-02-15 2021-12-22 CRISPR Therapeutics AG Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
CN109735547B (zh) * 2019-03-19 2022-05-31 青岛蔚蓝生物集团有限公司 一种提高毕赤酵母外源蛋白表达量的启动子及其应用
US20200405883A1 (en) * 2019-06-20 2020-12-31 Baxalta Incorporated Method of treatment with viral-based gene therapy
CN112575034B (zh) * 2019-09-29 2023-04-25 济南赛尔生物科技股份有限公司 一种治疗a型血友病的产品及应用

Family Cites Families (40)

* 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
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
ATE322547T1 (de) 1993-06-10 2006-04-15 Genetic Therapy Inc Adenovirale vektoren für die behandlung der hämophilie
SE504074C2 (sv) 1993-07-05 1996-11-04 Pharmacia Ab Proteinberedning för subkutan, intramuskulär eller intradermal administrering
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
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target 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.
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
US7041635B2 (en) 2003-01-28 2006-05-09 In2Gen Co., Ltd. Factor VIII polypeptide
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
KR100959454B1 (ko) 2007-12-10 2010-05-25 주식회사 동부하이텍 반도체 소자 및 그 제조 방법
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US20130017997A1 (en) * 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
CA2863329C (fr) * 2012-01-12 2023-01-03 Biogen Idec Ma Inc. Procedes de reduction de l'immunogenicite contre le facteur viii chez les individus soumis a un traitement par facteur viii
RS63870B1 (sr) * 2012-02-15 2023-01-31 Bioverativ Therapeutics Inc Sastavi faktora viii i postupci za pravljenje i upotrebu istih
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
CA2868391A1 (fr) * 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprenant du n1-methyl-pseudouracils et methodes pour les preparer
GB201210357D0 (en) * 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
WO2014008172A2 (fr) 2012-07-03 2014-01-09 Expression Therapeutics, Llc Lignée cellulaire en suspension à haut rendement, son système et son procédé de production
EP3513802B1 (fr) 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vecteur pour thérapie génique de l'hémophilie ciblée sur le foie et ses procédés et son utilisation
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
HRP20231183T1 (hr) 2013-02-15 2024-01-05 Bioverativ Therapeutics Inc. Optimizirani gen faktora viii
RU2710307C2 (ru) * 2013-09-12 2019-12-26 Байомарин Фармасьютикал Инк. Векторы экспрессии фактора viii на основе аденоассоциированного вируса
BR112017002781A2 (pt) 2014-08-13 2017-12-19 Sabatino Denise cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasia
DK3270944T3 (da) 2015-03-17 2020-01-27 Univ Brussel Vrije Optimerede leverspecifikke ekspressionssystemer til FVIII og FIX
EA038288B1 (ru) 2015-11-13 2021-08-05 Такеда Фармасьютикал Компани Лимитед Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a

Also Published As

Publication number Publication date
CL2020001611A1 (es) 2020-10-30
CA3005565A1 (fr) 2017-05-18
TW202229555A (zh) 2022-08-01
BR112018009732A8 (pt) 2019-02-26
JP2018537089A (ja) 2018-12-20
KR102404550B1 (ko) 2022-05-31
EP3374388A1 (fr) 2018-09-19
WO2017083764A1 (fr) 2017-05-18
TW201723180A (zh) 2017-07-01
US20170226188A1 (en) 2017-08-10
NZ742555A (en) 2023-12-22
CL2018001301A1 (es) 2018-12-28
CO2018005380A2 (es) 2018-05-31
KR20180070700A (ko) 2018-06-26
BR112018009732A2 (pt) 2018-11-21
AU2016354550B2 (en) 2020-04-16
EA201891138A1 (ru) 2018-12-28
EA036944B1 (ru) 2021-01-19
JP6695426B2 (ja) 2020-05-20
TWI823830B (zh) 2023-12-01
AU2016354550A1 (en) 2018-06-07
IL259295A (en) 2018-07-31
MX2018005969A (es) 2018-11-29
EA202092049A1 (ru) 2020-11-10
ZA201803442B (en) 2019-07-31
CN108602876B (zh) 2022-07-05
US10189889B2 (en) 2019-01-29
CN108602876A (zh) 2018-09-28

Similar Documents

Publication Publication Date Title
SG11201804070XA (en) Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11201804064WA (en) Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11201909203WA (en) Tissue selective transgene expression
SG11201809700YA (en) Gdf15 fusion proteins and uses thereof
SG11201810003UA (en) Using programmable dna binding proteins to enhance targeted genome modification
SG11201807134RA (en) Transposon system and methods of use
SG11201809344QA (en) Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde
SG11201909432SA (en) Compounds for increasing genome editing efficiency
SG11201901563UA (en) De novo synthesized nucleic acid libraries
SG11201808990QA (en) Compositions for topical application of compounds
SG11201808552XA (en) T-cell modulatory multimeric polypeptides and methods of use thereof
SG11201804400SA (en) Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use
SG11201808622SA (en) Chimeric receptors to flt3 and methods of use thereof
SG11201806398YA (en) Optimized factor viii genes
SG11201807912SA (en) Vaccine against rsv
SG11201804373VA (en) Compositions and methods for immunooncology
SG11201407548UA (en) Treatment of amd using aav sflt-1
SG11202000312UA (en) Encapsulated polynucleotides and methods of use
SG11201805162VA (en) Plasmid constructs for heterologous protein expression and methods of use
SG11201811232XA (en) Treatment of amd using aav2 variant with aflibercept
SG11201407866XA (en) Method for expression of heterologous proteins using a recombinant negative-strand rna virus vector
SG11201803642WA (en) Bacteria-based protein delivery
SG11201805579SA (en) Recombinant igg fc multimers
SG11201807593TA (en) Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
SG11201809374VA (en) Cd40l-fc fusion polypeptides and methods of use thereof