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PE20241995A1 - Metodo para obtener una capside de virus adenoasociado modificada - Google Patents

Metodo para obtener una capside de virus adenoasociado modificada

Info

Publication number
PE20241995A1
PE20241995A1 PE2024000277A PE2024000277A PE20241995A1 PE 20241995 A1 PE20241995 A1 PE 20241995A1 PE 2024000277 A PE2024000277 A PE 2024000277A PE 2024000277 A PE2024000277 A PE 2024000277A PE 20241995 A1 PE20241995 A1 PE 20241995A1
Authority
PE
Peru
Prior art keywords
modified
capsid
adenoasociated
obtaining
virus capsid
Prior art date
Application number
PE2024000277A
Other languages
English (en)
Inventor
Anna Nikolaevna Strelkova
Pavel Andreevich Iakovlev
Dmitry Valentinovich Morozov
Sergei Aleksandrovich Legotskii
Tatiana Evgenievna Shugaeva
Pavel Mikhailovich Gershovich
Alexandr Anatolevich Nadolinskii
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 RU2021124731A external-priority patent/RU2831732C1/ru
Application filed by Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of PE20241995A1 publication Critical patent/PE20241995A1/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
    • C12N15/79—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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/50—Mutagenesis
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00—ICT specially adapted for sequence analysis involving nucleotides or amino acids
    • G16B30/10—Sequence alignment; Homology search
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Theoretical Computer Science (AREA)
  • Evolutionary Biology (AREA)
  • Medical Informatics (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invencion se refiere a un metodo para producir una capside de un virus adenoasociado (AAV) modificada y a una capside de AAV modificada producida por dicho metodo, asi como a un acido nucleico aislado que codifica dicha capside modificada y a un vector basado en un virus adenoasociado recombinante para suministrar una secuencia de acido nucleico heterologa a un sujeto, que incluye dicha capside modificada.
PE2024000277A 2021-08-20 2022-08-18 Metodo para obtener una capside de virus adenoasociado modificada PE20241995A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2021124731A RU2831732C1 (ru) 2021-08-20 Способ получения модифицированного капсида аденоассоциированного вируса
PCT/RU2022/050255 WO2023022631A1 (ru) 2021-08-20 2022-08-18 Способ получения модифицированного капсида аденоассоциированного вируса

Publications (1)

Publication Number Publication Date
PE20241995A1 true PE20241995A1 (es) 2024-09-27

Family

ID=85240917

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2024000277A PE20241995A1 (es) 2021-08-20 2022-08-18 Metodo para obtener una capside de virus adenoasociado modificada

Country Status (17)

Country Link
US (1) US20250154526A1 (es)
EP (1) EP4389880A1 (es)
CN (1) CN118139974A (es)
AR (1) AR126841A1 (es)
AU (1) AU2022328531A1 (es)
CA (1) CA3229580A1 (es)
CL (1) CL2024000508A1 (es)
CO (1) CO2024001768A2 (es)
CR (1) CR20240137A (es)
EC (1) ECSP24013031A (es)
IL (1) IL310949A (es)
MA (1) MA64631B1 (es)
MX (1) MX2024002200A (es)
PE (1) PE20241995A1 (es)
TW (1) TW202317770A (es)
WO (1) WO2023022631A1 (es)
ZA (1) ZA202402156B (es)

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3431140A1 (de) 1984-08-24 1986-03-06 Behringwerke Ag, 3550 Marburg Enhancer fuer eukaryotische expressionssysteme
GB8717430D0 (en) 1987-07-23 1987-08-26 Celltech Ltd Recombinant dna product
CA2720097C (en) * 2007-04-09 2016-07-19 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
JP6042825B2 (ja) * 2011-02-10 2016-12-14 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法
SMT202000386T1 (it) 2011-04-22 2020-09-10 Univ California Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo
BR112014025985A2 (pt) 2012-04-18 2017-07-11 Childrens Hospital Philadelphia composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aav
US20190111107A1 (en) * 2016-01-26 2019-04-18 Defensin Therapeutics Aps Methods for modulating intestinal microbiota
IL312788A (en) * 2017-02-15 2024-07-01 Univ North Carolina Chapel Hill Methods and compositions for gene transfer across the vasculature
WO2019222411A1 (en) * 2018-05-16 2019-11-21 Spark Therapeutics, Inc. Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same

Also Published As

Publication number Publication date
IL310949A (en) 2024-04-01
CN118139974A (zh) 2024-06-04
AR126841A1 (es) 2023-11-22
MA64631B1 (fr) 2024-12-31
CR20240137A (es) 2024-07-29
AU2022328531A1 (en) 2024-03-14
WO2023022631A1 (ru) 2023-02-23
MA64631A1 (fr) 2024-05-31
US20250154526A1 (en) 2025-05-15
MX2024002200A (es) 2024-04-29
CO2024001768A2 (es) 2024-07-08
CL2024000508A1 (es) 2024-08-30
TW202317770A (zh) 2023-05-01
CA3229580A1 (en) 2023-02-23
ZA202402156B (en) 2024-10-30
ECSP24013031A (es) 2024-03-01
EP4389880A1 (en) 2024-06-26

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