CL2023002629A1 - Ácido nucleico optimizado por codones que codifica la proteína del factor - Google Patents

Ácido nucleico optimizado por codones que codifica la proteína del factor

Info

Publication number
CL2023002629A1
CL2023002629A1 CL2023002629A CL2023002629A CL2023002629A1 CL 2023002629 A1 CL2023002629 A1 CL 2023002629A1 CL 2023002629 A CL2023002629 A CL 2023002629A CL 2023002629 A CL2023002629 A CL 2023002629A CL 2023002629 A1 CL2023002629 A1 CL 2023002629A1
Authority
CL
Chile
Prior art keywords
codon
nucleic acid
acid encoding
factor protein
optimized nucleic
Prior art date
Application number
CL2023002629A
Other languages
English (en)
Inventor
Vladimirovich Prokofyev Alexander
Mikhailovich Gershovich Pavel
Nikolaevna Strelkova Anna
Aleksandrovna Spirina Natalia
Evgenievna Shugaeva Tatiana
Valentinovich MOROZOV Dmitry
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 CL2023002629A1 publication Critical patent/CL2023002629A1/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)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (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.
CL2023002629A 2021-03-05 2023-09-04 Ácido nucleico optimizado por codones que codifica la proteína del factor CL2023002629A1 (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
CL2023002629A1 true CL2023002629A1 (es) 2024-04-01

Family

ID=83154378

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2023002629A CL2023002629A1 (es) 2021-03-05 2023-09-04 Ácido nucleico optimizado por codones que codifica la proteína del factor

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
US10842853B2 (en) * 2017-05-22 2020-11-24 Baxalta Incorporated Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B

Also Published As

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

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
CO2022001833A2 (es) Proteína vp1 modificada aislada de la cápside del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en la misma.
CL2021002755A1 (es) Composiciones útiles para el tratamiento de la enfermedad de pompe.
CO2023009633A2 (es) Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal
BR112022021459A2 (pt) Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
Han et al. Quantitative proteomic analysis of duck ovarian follicles infected with duck Tembusu virus by label-free LC-MS
CO2021008877A2 (es) Uso de vectores lentivirales que expresan el factor ix
Oda et al. Hypothermia produces rat liver proteomic changes as in hibernating mammals but decreases endoplasmic reticulum chaperones
BR112022016965A2 (pt) Polipeptídeos e vetores de capsídeo do vírus adenoassociado
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
BR112021026356A2 (pt) Sistema de expressão de baculovirus
BR112022019304A2 (pt) Variantes de capsídeo de aav e usos das mesmas
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
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
Dabbaghizadeh Structure and function of mitochondrial small heat shock protein 22 in Drosophila melanogaster
AR123041A1 (es) Transactivadores del dominio de unión al adn y usos de los mismos
CO2023011603A2 (es) Aplicación del inhibidor de apoptosis 5 (api5) para la restitución epitelial