BR112023018003A2 - Ácido nucleico códon-otimizado codificante para uma proteína do fator ix de coagulação e cassetes de expressão, vetores de expressão, vírus recombinantes, composições farmacêuticas, usos e métodos associados - Google Patents

Ácido nucleico códon-otimizado codificante para uma proteína do fator ix de coagulação e cassetes de expressão, vetores de expressão, vírus recombinantes, composições farmacêuticas, usos e métodos associados

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Publication number
BR112023018003A2
BR112023018003A2 BR112023018003A BR112023018003A BR112023018003A2 BR 112023018003 A2 BR112023018003 A2 BR 112023018003A2 BR 112023018003 A BR112023018003 A BR 112023018003A BR 112023018003 A BR112023018003 A BR 112023018003A BR 112023018003 A2 BR112023018003 A2 BR 112023018003A2
Authority
BR
Brazil
Prior art keywords
nucleic acid
codon
protein
expression
optimized nucleic
Prior art date
Application number
BR112023018003A
Other languages
English (en)
Inventor
Pavel Mikhailovich Gershovich
Dmitry Valentinovich Morozov
Alexander Vladimirovich Prokofyev
Tatiana Evgenievna Shugaeva
Natalia Aleksandrovna Spirina
Anna Nikolaevna Strelkova
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 BR112023018003A2 publication Critical patent/BR112023018003A2/pt

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    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • 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)
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    • 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
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Biomedical Technology (AREA)
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  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
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  • 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

ácido nucleico códon-otimizado codificante para uma proteína do fator ix de coagulação e cassetes de expressão, vetores de expressão, vírus recombinantes, composições farmacêuticas, usos e métodos associados. o presente pedido refere-se às áreas de genética, terapia genética e biologia molecular. mais especificamente, a presente invenção refere-se a um ácido nucleico isolado códon-otimizado que codifica a proteína fix (fator de coagulação ix), um cassete de expressão e um vetor baseado nele, bem como um ácido nucleico baseado em aav5 (sorotipo 5 do vírus adeno-associado). vírus recombinante para aumentar a expressão do gene fix em células alvo e utilização do mesmo.
BR112023018003A 2021-03-05 2022-03-05 Ácido nucleico códon-otimizado codificante para uma proteína do fator ix de coagulação e cassetes de expressão, vetores de expressão, vírus recombinantes, composições farmacêuticas, usos e métodos associados BR112023018003A2 (pt)

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
BR112023018003A2 true BR112023018003A2 (pt) 2023-12-26

Family

ID=83154378

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023018003A BR112023018003A2 (pt) 2021-03-05 2022-03-05 Ácido nucleico códon-otimizado codificante para uma proteína do fator ix de coagulação e cassetes de expressão, vetores de expressão, vírus recombinantes, composições farmacêuticas, usos e métodos associados

Country Status (19)

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

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
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
MX2023010350A (es) 2023-09-22
US20240076691A1 (en) 2024-03-07
CN117716038A (zh) 2024-03-15
AU2022230548A1 (en) 2023-09-28

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