CO2021008877A2 - Uso de vectores lentivirales que expresan el factor ix - Google Patents

Uso de vectores lentivirales que expresan el factor ix

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Publication number
CO2021008877A2
CO2021008877A2 CONC2021/0008877A CO2021008877A CO2021008877A2 CO 2021008877 A2 CO2021008877 A2 CO 2021008877A2 CO 2021008877 A CO2021008877 A CO 2021008877A CO 2021008877 A2 CO2021008877 A2 CO 2021008877A2
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Colombia
Prior art keywords
lentiviral
lentiviral vectors
fix
hemophilia
polypeptide
Prior art date
Application number
CONC2021/0008877A
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English (en)
Inventor
Tongyao Liu
Alessio Cantore
Luigi Naldini
Susannah Patarroyo-White
Douglas Drager
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Bioverativ Therapeutics Inc
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Publication date
Application filed by Bioverativ Therapeutics Inc filed Critical Bioverativ Therapeutics Inc
Publication of CO2021008877A2 publication Critical patent/CO2021008877A2/es

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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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • A61K48/005—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
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    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16051—Methods of production or purification of viral material
    • C12N2740/16052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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

Abstract

La presente descripción proporciona vectores lentivirales que comprenden una secuencia de ácido nucleico que codifica un polipéptido con actividad de factor IX (FIX), y métodos para usar dichos vectores lentivirales. Los vectores lentivirales dirigidos al hígado descritos en la presente memoria pueden usarse para terapia génica, en donde la administración génica lentiviral permite la integración estable del casete de expresión del transgén en el genoma de las células diana (p. ej., hepatocitos) de sujetos pediátricos (p. ej., neonatales) o adultos, consiguiendo una mejoría en la expresión de FIX a dosis bajas del vector lentiviral. La presente descripción también proporciona métodos para tratar trastornos hemorrágicos tales como la hemofilia (p. ej., hemofilia B) que comprenden administrar a un sujeto que lo necesita un vector lentiviral dirigido al hígado que comprende una secuencia de ácido nucleico que codifica un polipéptido con actividad de secuencia de FIX a dosificaciones bajas.
CONC2021/0008877A 2018-12-06 2021-07-05 Uso de vectores lentivirales que expresan el factor ix CO2021008877A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862776393P 2018-12-06 2018-12-06
PCT/US2019/064711 WO2020118069A2 (en) 2018-12-06 2019-12-05 Use of lentiviral vectors expressing factor ix

Publications (1)

Publication Number Publication Date
CO2021008877A2 true CO2021008877A2 (es) 2021-07-30

Family

ID=69024682

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0008877A CO2021008877A2 (es) 2018-12-06 2021-07-05 Uso de vectores lentivirales que expresan el factor ix

Country Status (14)

Country Link
US (1) US20200199626A1 (es)
EP (1) EP3891289A2 (es)
JP (1) JP2022514465A (es)
KR (1) KR20210100661A (es)
CN (1) CN113396223A (es)
AU (1) AU2019393880A1 (es)
BR (1) BR112021010047A2 (es)
CA (1) CA3121786A1 (es)
CO (1) CO2021008877A2 (es)
IL (1) IL283547A (es)
MX (1) MX2021006648A (es)
SG (1) SG11202105880TA (es)
TW (1) TW202039855A (es)
WO (1) WO2020118069A2 (es)

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GB202205615D0 (en) * 2022-04-14 2022-06-01 Genespire S R L Lentiviral vector
WO2023198930A1 (en) * 2022-04-14 2023-10-19 Genespire S.R.L. Lentiviral vector

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Publication number Publication date
SG11202105880TA (en) 2021-07-29
IL283547A (en) 2021-07-29
CA3121786A1 (en) 2020-06-11
WO2020118069A3 (en) 2020-07-16
BR112021010047A2 (pt) 2021-08-24
EP3891289A2 (en) 2021-10-13
CN113396223A (zh) 2021-09-14
MX2021006648A (es) 2021-07-07
KR20210100661A (ko) 2021-08-17
TW202039855A (zh) 2020-11-01
US20200199626A1 (en) 2020-06-25
JP2022514465A (ja) 2022-02-14
AU2019393880A1 (en) 2021-07-15
WO2020118069A2 (en) 2020-06-11

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