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/RU2831751C2/ru
Application filed by Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of ECSP23067213A publication Critical patent/ECSP23067213A/es

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    • 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
    • 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
    • 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)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (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)
  • Peptides Or Proteins (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 RU2831751C2 (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 (22)

Country Link
US (1) US20240076691A1 (es)
EP (1) EP4288549A4 (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)
JO (1) JOP20230208A1 (es)
MA (1) MA62371B1 (es)
MX (1) MX2023010350A (es)
PE (1) PE20242117A1 (es)
PY (1) PY2215552A (es)
TW (1) TW202246505A (es)
UY (1) UY39659A (es)
WO (1) WO2022186734A1 (es)

Family Cites Families (9)

* 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
US11008561B2 (en) * 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
ES2764453T3 (es) * 2015-03-17 2020-06-03 Univ Brussel Vrije Sistemas de expresión específica de hígado optimizados para FVIII y FIX
PE20180675A1 (es) * 2015-06-23 2018-04-19 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
CN118581121A (zh) * 2017-05-22 2024-09-03 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
CN113164560A (zh) * 2018-10-26 2021-07-23 Vrije布鲁塞尔大学 改善基因疗法的新工具及其用途
SG11202105880TA (en) * 2018-12-06 2021-07-29 Bioverativ Therapeutics Inc Use of lentiviral vectors expressing factor ix

Also Published As

Publication number Publication date
PY2215552A (es) 2022-09-06
EP4288549A1 (en) 2023-12-13
CN117716038A (zh) 2024-03-15
MX2023010350A (es) 2023-09-22
CO2023011679A2 (es) 2024-01-15
TW202246505A (zh) 2022-12-01
JP2024509224A (ja) 2024-02-29
KR20230154067A (ko) 2023-11-07
BR112023018003A2 (pt) 2023-12-26
MA62371B1 (fr) 2025-12-31
UY39659A (es) 2022-09-30
US20240076691A1 (en) 2024-03-07
WO2022186734A1 (en) 2022-09-09
AU2022230548A1 (en) 2023-09-28
EP4288549A4 (en) 2025-06-25
CA3212809A1 (en) 2022-09-09
JOP20230208A1 (ar) 2023-09-05
IL305679A (en) 2023-11-01
AR125041A1 (es) 2023-05-31
CR20230429A (es) 2024-03-08
PE20242117A1 (es) 2024-10-28
CL2023002629A1 (es) 2024-04-01
MA62371A1 (fr) 2024-03-29

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