[go: up one dir, main page]

PE20242117A1 - Acido nucleico optimizado por codones que codifica la proteina del factor de coagulacion ix, y su uso - Google Patents

Acido nucleico optimizado por codones que codifica la proteina del factor de coagulacion ix, y su uso

Info

Publication number
PE20242117A1
PE20242117A1 PE2024001665A PE2024001665A PE20242117A1 PE 20242117 A1 PE20242117 A1 PE 20242117A1 PE 2024001665 A PE2024001665 A PE 2024001665A PE 2024001665 A PE2024001665 A PE 2024001665A PE 20242117 A1 PE20242117 A1 PE 20242117A1
Authority
PE
Peru
Prior art keywords
nucleic acid
coagulation factor
protein
codon
acid encoding
Prior art date
Application number
PE2024001665A
Other languages
English (en)
Inventor
Alexander Vladimirovich Prokofyev
Pavel Mikhailovich Gershovich
Anna Nikolaevna Strelkova
Natalia Aleksandrovna Spirina
Tatiana Evgenievna Shugaeva
Dmitry Valentinovich Morozov
Original Assignee
Biocad Joint Stock Co
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
Priority claimed from RU2021105703A external-priority patent/RU2831751C2/ru
Application filed by Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of PE20242117A1 publication Critical patent/PE20242117A1/es

Links

Classifications

    • 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
    • C12N15/67—General methods for enhancing the expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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)
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (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)
  • Peptides Or Proteins (AREA)

Abstract

Se refiere a un vector viral de expresion que incluye un acido nucleico aislado con codones optimizados que codifica la proteina FIX (factor IX de coagulacion) que tiene la secuencia de aminoacidos de SEQ ID NO: 1, que incluye una secuencia de nucleotidos que se selecciona del grupo que comprende: SEQ ID NO: 2 o SEQ ID NO: 4; o un casete de expresion que incluye dicho acido nucleico optimizado por estos codones. Tambien se refiere a una composicion farmaceutica que comprende dicho vector viral para administrar el gen FIX a las celulas diana en el tratamiento de hemofilia B.
PE2024001665A 2021-03-05 2022-03-05 Acido nucleico optimizado por codones que codifica la proteina del factor de coagulacion ix, y su uso PE20242117A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2021105703A RU2831751C2 (ru) 2021-03-05 Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови IX, и ее применение
PCT/RU2022/050073 WO2022186734A1 (en) 2021-03-05 2022-03-05 Codon-optimized nucleic acid encoding the fix protein

Publications (1)

Publication Number Publication Date
PE20242117A1 true PE20242117A1 (es) 2024-10-28

Family

ID=83154378

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2024001665A PE20242117A1 (es) 2021-03-05 2022-03-05 Acido nucleico optimizado por codones que codifica la proteina del factor de coagulacion ix, y su uso

Country Status (20)

Country Link
US (1) US20240076691A1 (es)
EP (1) EP4288549A1 (es)
JP (1) JP2024509224A (es)
KR (1) KR20230154067A (es)
CN (1) CN117716038A (es)
AR (1) AR125041A1 (es)
AU (1) AU2022230548A1 (es)
BR (1) BR112023018003A2 (es)
CA (1) CA3212809A1 (es)
CL (1) CL2023002629A1 (es)
CO (1) CO2023011679A2 (es)
CR (1) CR20230429A (es)
EC (1) ECSP23067213A (es)
IL (1) IL305679A (es)
MA (1) MA62371A1 (es)
MX (1) MX2023010350A (es)
PE (1) PE20242117A1 (es)
TW (1) TW202246505A (es)
UY (1) UY39659A (es)
WO (1) WO2022186734A1 (es)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6458563B1 (en) * 1996-06-26 2002-10-01 Emory University Modified factor VIII
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP3630974A1 (en) * 2017-05-22 2020-04-08 Baxalta Incorporated Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b

Also Published As

Publication number Publication date
CN117716038A (zh) 2024-03-15
AU2022230548A1 (en) 2023-09-28
KR20230154067A (ko) 2023-11-07
JP2024509224A (ja) 2024-02-29
EP4288549A1 (en) 2023-12-13
IL305679A (en) 2023-11-01
CR20230429A (es) 2024-03-08
MA62371A1 (fr) 2024-03-29
MX2023010350A (es) 2023-09-22
AR125041A1 (es) 2023-05-31
US20240076691A1 (en) 2024-03-07
CO2023011679A2 (es) 2024-01-15
ECSP23067213A (es) 2023-10-31
CL2023002629A1 (es) 2024-04-01
BR112023018003A2 (pt) 2023-12-26
WO2022186734A1 (en) 2022-09-09
UY39659A (es) 2022-09-30
TW202246505A (zh) 2022-12-01
CA3212809A1 (en) 2022-09-09

Similar Documents

Publication Publication Date Title
EA201991440A1 (ru) ТЕРМОСТАБИЛЬНЫЕ НУКЛЕАЗЫ Cas9
AR108442A1 (es) Proteínas de fusión gdf15 y usos de estas
MX2024015050A (es) L-asparaginasa modificada
JP2017018125A5 (es)
PE20190911A1 (es) Composiciones y anticuerpos anti-lag-3
Molouki et al. The matrix (M) protein of Newcastle disease virus binds to human bax through its BH3 domain
WO2016164797A1 (en) Activatable crispr/cas9 for spatial and temporal control of genome editing
ES2075901T3 (es) Sistemas de expresion de virus recombinantes de arn con cadena negativa y vacunas.
AR087091A1 (es) Polipeptidos quimericos e hibridos del factor viii, y sus metodos de uso
CL2019003409A1 (es) Péptido asociados a tumor (tumap) aislado; ácido nucleico; vector de expresión; célula huésped recombinante que comprendes el péptido; método para producir el péptido; uso para preparar un medicamento útil rara tratar el cáncer; kit farmacéutico. (divisional solicitud 201702407)
AR076349A1 (es) Peptido auxiliar del antigeno del cancer
US20230242627A1 (en) Composition and method for activating latent human immunodeficiency virus (hiv)
Pennington et al. H-IPSE is a pathogen-secreted host nucleus-infiltrating protein (infiltrin) expressed exclusively by the Schistosoma haematobium egg stage
WO2021198706A3 (en) Coronavirus vaccines
AR065075A1 (es) Vectores para la expresion multiple de genes
PE20220429A1 (es) Vectores viricos que codifican variantes del fviii recombinantes con mayor expresion para la genoterapia de la hemofilia a
PE20210918A1 (es) Composiciones y metodos para el tratamiento de la distrofia macular
PE20231439A1 (es) Proteinas f de hmpv estabilizadas por prefusion
CO2024002315A2 (es) Sistemas de edición de gen que comprenden una guía de arn dirigida a estatmina 2 (stmn2) y usos de los mismos
PE20242117A1 (es) Acido nucleico optimizado por codones que codifica la proteina del factor de coagulacion ix, y su uso
BR112019007796A2 (pt) construção de vacina e seus usos contra infecções causadas por staphylococcus
WO2023155901A1 (en) Mutant cytidine deaminases with improved editing precision
AR045000A1 (es) Gen y proteina de eimeria , y su uso
MX2021009161A (es) Acidos nucleicos recombinantes que contienen secuencias promotoras de alfaherpesvirus.
CO2022004535A2 (es) Secuencia de extensión n-terminal para la expresión de péptidos terapéuticos recombinantes