MX2023010350A - 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
MX2023010350A
MX2023010350A MX2023010350A MX2023010350A MX2023010350A MX 2023010350 A MX2023010350 A MX 2023010350A MX 2023010350 A MX2023010350 A MX 2023010350A MX 2023010350 A MX2023010350 A MX 2023010350A MX 2023010350 A MX2023010350 A MX 2023010350A
Authority
MX
Mexico
Prior art keywords
nucleic acid
codon
optimized nucleic
acid encoding
fix
Prior art date
Application number
MX2023010350A
Other languages
English (en)
Inventor
Dmitry Valentinovich Morozov
Anna Nikolaevna Strelkova
Pavel Mikhailovich Gershovich
Tatiana Evgenievna Shugaeva
Alexander Vladimirovich Prokofyev
Natalia Aleksandrovna Spirina
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 MX2023010350A publication Critical patent/MX2023010350A/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.
MX2023010350A 2021-03-05 2022-03-05 Acido nucleico optimizado por codones que codifica la proteina del factor de coagulacion ix, y su uso. MX2023010350A (es)

Applications Claiming Priority (2)

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
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
MX2023010350A true MX2023010350A (es) 2023-09-22

Family

ID=83154378

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023010350A MX2023010350A (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 (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
ECSP23067213A (es) 2023-10-31
AR125041A1 (es) 2023-05-31
EP4288549A1 (en) 2023-12-13
US20240076691A1 (en) 2024-03-07
CN117716038A (zh) 2024-03-15
AU2022230548A1 (en) 2023-09-28

Similar Documents

Publication Publication Date Title
JP2020508685A5 (es)
ZA202213092B (en) Codon-optimized nucleic acid that encodes smn1 protein, and use thereof
MX2021001599A (es) Moleculas de acido nucleico y sus usos para la terapia genica no viral.
MX2022003116A (es) Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
Wang et al. Recoding of the vesicular stomatitis virus L gene by computer-aided design provides a live, attenuated vaccine candidate
MX2020001593A (es) Moleculas de acido nucleico y usos de las mismas.
Molouki et al. The matrix (M) protein of Newcastle disease virus binds to human bax through its BH3 domain
ATE469216T1 (de) Modifizierte vitamin-k-abhängige polypeptide
GB201201178D0 (en) Novel enzymes
MX2022002184A (es) Proteina vp1 modificada aislada de la capside del virus adeno-asociado de serotipo 5 (aav5), capside y vector basado en la misma.
MX2022010388A (es) Polipéptidos de la cápside y vectores de virus adenoasociado.
BR112021021792A2 (pt) Composições úteis para tratamento de doença de pompe
MX2023008825A (es) Efecto sinergico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal.
MX2023010350A (es) Acido nucleico optimizado por codones que codifica la proteina del factor de coagulacion ix, y su uso.
Yadav et al. Species selectivity in poxviral complement regulators is dictated by the charge reversal in the central complement control protein modules
MX2024002379A (es) Cápsides y vectores de aav modificados.
CR20240089A (es) Proteína de la cápside VP1 modificada aislada del virus adenoasociado de serotipo 9 (AAV9), cápside y vector basado en la misma
MX2021007379A (es) Metodos y composiciones para el tratamiento de enfermedades de almacenamiento de glucogeno.
CR20240137A (es) Método para obtener una cápside de virus adenoasociado modificada
CR20240088A (es) Proteína VP1 modificada y aislada de la cápside del virus adenoasociado del serotipo 5 (AAV5), cápside y vector basados en esta
WO2023178338A3 (en) Methods and compositions for treating tmem43 related cardiomyopathy with a viral vector
Karimi et al. Cloning of human factor 9 cDNA in AAV vector and assessment of its expression in HEK293T cells
WO2024044689A3 (en) Small molecule-inducible gene expression switches
EA202191148A1 (ru) Рнк, кодирующая белок
MY190213A (en) Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues