AR117825A1 - Vectores virales que codifican variantes del fviii recombinantes con mayor expresión para el tratamiento génico de la hemofilia a - Google Patents

Vectores virales que codifican variantes del fviii recombinantes con mayor expresión para el tratamiento génico de la hemofilia a

Info

Publication number
AR117825A1
AR117825A1 ARP200100113A ARP200100113A AR117825A1 AR 117825 A1 AR117825 A1 AR 117825A1 AR P200100113 A ARP200100113 A AR P200100113A AR P200100113 A ARP200100113 A AR P200100113A AR 117825 A1 AR117825 A1 AR 117825A1
Authority
AR
Argentina
Prior art keywords
hemophilia
fviii
viral vectors
gene treatment
greater expression
Prior art date
Application number
ARP200100113A
Other languages
English (en)
Inventor
Friedrich Scheiflinger
Hanspeter Rottensteiner
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 AR117825A1 publication Critical patent/AR117825A1/es

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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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61K48/0066—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
    • 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
    • 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
    • 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

Landscapes

  • 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)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente divulgación proporciona, entre otros aspectos, polinucleótidos modificados con codones que codifican variantes del Factor VIII para expresión en células de mamífero. En algunas realizaciones, la divulgación también proporciona vectores de tratamiento génico de mamífero y métodos para tratar la hemofilia A.
ARP200100113A 2019-01-16 2020-01-16 Vectores virales que codifican variantes del fviii recombinantes con mayor expresión para el tratamiento génico de la hemofilia a AR117825A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962793058P 2019-01-16 2019-01-16

Publications (1)

Publication Number Publication Date
AR117825A1 true AR117825A1 (es) 2021-08-25

Family

ID=69593774

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200100113A AR117825A1 (es) 2019-01-16 2020-01-16 Vectores virales que codifican variantes del fviii recombinantes con mayor expresión para el tratamiento génico de la hemofilia a

Country Status (18)

Country Link
US (2) US11707536B2 (es)
EP (1) EP3911672A1 (es)
JP (1) JP2022517267A (es)
KR (1) KR20210117291A (es)
CN (1) CN113614104A (es)
AR (1) AR117825A1 (es)
AU (1) AU2020208375A1 (es)
BR (1) BR112021013874A2 (es)
CA (1) CA3127065A1 (es)
CL (1) CL2021001880A1 (es)
CO (1) CO2021009338A2 (es)
EA (1) EA202191977A1 (es)
EC (1) ECSP21060605A (es)
IL (2) IL314658A (es)
MX (1) MX2021008550A (es)
PE (1) PE20220429A1 (es)
SG (1) SG11202107421YA (es)
WO (1) WO2020150375A1 (es)

Families Citing this family (5)

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EP4314041A1 (en) * 2021-03-30 2024-02-07 Aavnergene Inc. Modified plasma clotting factor viii and method of use thereof
US11795207B2 (en) 2021-03-30 2023-10-24 AAVnerGene Inc. Modified plasma clotting factor VIII and method of use thereof
US20240254197A1 (en) * 2021-11-25 2024-08-01 Sichuan Real&Best Biotech Co., Ltd. Engineered Human FVIII with Enhanced Secretion Ability and Clotting Activity
AR128760A1 (es) * 2022-03-11 2024-06-12 Homology Medicines Inc Vectores de expresión con promotores dobles bidireccionales y sus usos
CN116715752A (zh) * 2023-06-16 2023-09-08 苏州诺洁贝生物技术有限公司 凝血因子fviii蛋白变体、表达载体和用途

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Also Published As

Publication number Publication date
AU2020208375A1 (en) 2021-07-29
IL314658A (en) 2024-09-01
IL284648B1 (en) 2024-08-01
WO2020150375A1 (en) 2020-07-23
CN113614104A (zh) 2021-11-05
KR20210117291A (ko) 2021-09-28
JP2022517267A (ja) 2022-03-07
US20230211017A1 (en) 2023-07-06
CL2021001880A1 (es) 2022-01-14
CO2021009338A2 (es) 2021-07-30
SG11202107421YA (en) 2021-08-30
EP3911672A1 (en) 2021-11-24
EA202191977A1 (ru) 2021-12-15
IL284648A (en) 2021-08-31
ECSP21060605A (es) 2022-01-31
MX2021008550A (es) 2021-08-19
TW202039546A (zh) 2020-11-01
US20200289672A1 (en) 2020-09-17
US11707536B2 (en) 2023-07-25
BR112021013874A2 (pt) 2021-09-21
CA3127065A1 (en) 2020-07-23
PE20220429A1 (es) 2022-03-29

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