ECSP23067213A - Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX y su uso - Google Patents

Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX y su uso

Info

Publication number
ECSP23067213A
ECSP23067213A ECSENADI202367213A ECDI202367213A ECSP23067213A EC SP23067213 A ECSP23067213 A EC SP23067213A EC SENADI202367213 A ECSENADI202367213 A EC SENADI202367213A EC DI202367213 A ECDI202367213 A EC DI202367213A EC SP23067213 A ECSP23067213 A EC SP23067213A
Authority
EC
Ecuador
Prior art keywords
codon
protein
nucleic acid
acid encoding
coagulation factor
Prior art date
Application number
ECSENADI202367213A
Other languages
English (en)
Inventor
Tatiana Evgenievna Shugaeva
Alexander Vladimirovich Prokofyev
Anna Nikolaevna Strelkova
Dmitry Valentinovich Morozov
Natalia Aleksandrovna Spirina
Pavel Mikhailovich Gershovich
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/RU2021105703A/ru
Application filed by Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of ECSP23067213A publication Critical patent/ECSP23067213A/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
    • 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
    • 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
    • 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
    • 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)
    • 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

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)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente solicitud está relacionada con los campos de la genética, la terapia génica y la biología molecular. Más específicamente, la presente invención se refiere a un ácido nucleico aislado optimizado en codones que codifica la proteína FIX (factor de coagulación IX), un casete de expresión y un vector basado en el mismo, así como un virus recombinante basado en el AAV5 (virus adenoasociado de serotipo 5) para aumentar la expresión del gen FIX en las células diana, y su uso.
ECSENADI202367213A 2021-03-05 2023-09-05 Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX y su uso ECSP23067213A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
RU2021105703A RU2021105703A (ru) 2021-03-05 Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови IX, и ее применение

Publications (1)

Publication Number Publication Date
ECSP23067213A true ECSP23067213A (es) 2023-10-31

Family

ID=83154378

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202367213A ECSP23067213A (es) 2021-03-05 2023-09-05 Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX y su uso

Country Status (19)

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)
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
CN110945127B (zh) * 2017-05-22 2024-07-12 武田药品工业株式会社 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体

Also Published As

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

Similar Documents

Publication Publication Date Title
CO2022017334A2 (es) Ácido nucleico codón-optimizado que codifica la proteína smn1 y su uso
CO2021002965A2 (es) Moléculas de ácido nucleico y sus usos para la terapia génica no viral
BR112021021720A2 (pt) Composições úteis para tratamento de doença de pompe
BR112022021459A2 (pt) Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
AR119869A1 (es) Proteína vp1 modificada aislada de la cápside del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en la misma
MX2022010388A (es) Polipéptidos de la cápside y vectores de virus adenoasociado.
Gad et al. A viral histone H4 encoded by Cotesia plutellae bracovirus inhibits haemocyte-spreading behaviour of the diamondback moth, Plutella xylostella
ECSP23067213A (es) Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX y su uso
BR112023015177A2 (pt) Ácido nucleico compreendendo smn1 e mir-23a para tratamento da atrofia muscular espinhal, cassete de expressão, vetor de expressão, vírus recombinante, composição farmacêutica, uso e método relacionados
BR112021026356A2 (pt) Sistema de expressão de baculovirus
CL2022003099A1 (es) Terapia génica con vectores duales de disferlina
BR112021019032A2 (pt) Vetores de fator h e usos dos mesmos
CO2022008784A2 (es) Proteínas gpcr de opsinas quiméricas
CO2021015432A2 (es) Secuencia promotora y productos y usos relacionados de la misma
ECSP24013002A (es) Proteína vp1 de la cápsida aav9 modificada aislada
Zhang et al. Identification of differentially expressed proteins in Ostrinia furnacalis adults after exposure to ultraviolet A
AR129552A1 (es) Ácido nucleico con actividad promotora y uso del mismo
CO2024001768A2 (es) Método para obtener una cápside de virus adenoasociado modificada
CL2024000503A1 (es) Proteína vp1 de cápside modificada y aislada de aav5
Vafina et al. A one-step protocol for chromatographic purification of non-recombinant exogenous bacterial enzyme: nuclease of Serratia marcescens
AR129164A1 (es) Ácido nucleico aislado que codifica una proteína de fusión basada en fviii-bdd y en un péptido señal heterólogo, y uso de la misma
Ghendon et al. Replication of two influenza virus strains and a recombinant in HEF and LEP cells
BR112023026961A2 (pt) Composições e métodos para modular a expressão de genes
BR112022018193A2 (pt) Vetor de expressão do fator 2 relacionado ao fator nuclear e2 humano e aplicação do vetor de expressão
CO2024001455A2 (es) Sistema de expresión de baculovirus