AU2016353353B2 - 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 Download PDF

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Publication number
AU2016353353B2
AU2016353353B2 AU2016353353A AU2016353353A AU2016353353B2 AU 2016353353 B2 AU2016353353 B2 AU 2016353353B2 AU 2016353353 A AU2016353353 A AU 2016353353A AU 2016353353 A AU2016353353 A AU 2016353353A AU 2016353353 B2 AU2016353353 B2 AU 2016353353B2
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Prior art keywords
seq
factor viii
sequence
polynucleotide
amino acid
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AU2016353353A
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AU2016353353A1 (en
Inventor
Falko-Günter Falkner
Franziska HORLING
Johannes LENGLER
Hanspeter Rottensteiner
Friedrich Scheiflinger
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Takeda Pharmaceutical Co Ltd
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Takeda Pharmaceutical Co Ltd
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Publication of AU2016353353A1 publication Critical patent/AU2016353353A1/en
Priority to AU2020203761A priority Critical patent/AU2020203761B2/en
Priority to AU2020203760A priority patent/AU2020203760B2/en
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Assigned to TAKEDA PHARMACEUTICAL COMPANY LIMITED reassignment TAKEDA PHARMACEUTICAL COMPANY LIMITED Request for Assignment Assignors: Baxalta GmbH, BAXALTA INCORPORATED
Priority to AU2022201319A priority patent/AU2022201319B2/en
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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
    • 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
    • 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
    • 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
    • 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
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2016353353A 2015-11-13 2016-11-11 Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A Active AU2016353353B2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2020203761A AU2020203761B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AU2020203760A AU2020203760B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AU2022201319A AU2022201319B2 (en) 2015-11-13 2022-02-25 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562255317P 2015-11-13 2015-11-13
US62/255,317 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

Related Child Applications (2)

Application Number Title Priority Date Filing Date
AU2020203760A Division AU2020203760B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AU2020203761A Division AU2020203761B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Publications (2)

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AU2016353353A1 AU2016353353A1 (en) 2018-06-07
AU2016353353B2 true AU2016353353B2 (en) 2020-07-09

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Application Number Title Priority Date Filing Date
AU2016353353A Active AU2016353353B2 (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
AU2020203761A Active AU2020203761B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AU2020203760A Active AU2020203760B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AU2022201319A Active AU2022201319B2 (en) 2015-11-13 2022-02-25 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Family Applications After (3)

Application Number Title Priority Date Filing Date
AU2020203761A Active AU2020203761B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AU2020203760A Active AU2020203760B2 (en) 2015-11-13 2020-06-05 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
AU2022201319A Active AU2022201319B2 (en) 2015-11-13 2022-02-25 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Country Status (18)

Country Link
US (4) US10189888B2 (OSRAM)
EP (1) EP3374387A1 (OSRAM)
JP (4) JP6768798B2 (OSRAM)
KR (2) KR102484553B1 (OSRAM)
CN (3) CN116949052A (OSRAM)
AU (4) AU2016353353B2 (OSRAM)
BR (1) BR112018009717B1 (OSRAM)
CA (1) CA3005334A1 (OSRAM)
CL (1) CL2018001299A1 (OSRAM)
CO (1) CO2018005377A2 (OSRAM)
EA (2) EA038288B1 (OSRAM)
IL (3) IL305526A (OSRAM)
MX (1) MX2018005982A (OSRAM)
MY (2) MY190067A (OSRAM)
SG (2) SG10202106307UA (OSRAM)
TW (3) TWI777175B (OSRAM)
WO (1) WO2017083762A1 (OSRAM)
ZA (3) ZA201803443B (OSRAM)

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AU2016353353B2 (en) 2015-11-13 2020-07-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
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WO2017180861A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsulvania Gene therapy for treating hemophilia b
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US12091675B2 (en) * 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
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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
EP4211153A4 (en) * 2021-11-25 2023-11-01 Sichuan Real&Best Biotech Co., Ltd. ENGINEERED HUMAN FVII1 WITH INCREASED SECRETION ABILITY AND COAGULATION ACTIVITY
CR20240524A (es) * 2022-04-28 2025-05-05 Biocad Joint Stock Co Ácido nucleico de codón optimizado que codifica la proteína del factor viii de coagulación de dominio b suprimido, y uso del mismo
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
TW202435846A (zh) 2023-03-14 2024-09-16 美商希吉隆醫療公司 包含共價交聯多醣及胰島細胞之水膠膠囊
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