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MA64631A1 - Procédé de production de capside modifié de virus adéno-associé - Google Patents

Procédé de production de capside modifié de virus adéno-associé

Info

Publication number
MA64631A1
MA64631A1 MA64631A MA64631A MA64631A1 MA 64631 A1 MA64631 A1 MA 64631A1 MA 64631 A MA64631 A MA 64631A MA 64631 A MA64631 A MA 64631A MA 64631 A1 MA64631 A1 MA 64631A1
Authority
MA
Morocco
Prior art keywords
associated virus
capsid
modified
producing modified
virus capsid
Prior art date
Application number
MA64631A
Other languages
English (en)
Other versions
MA64631B1 (fr
Inventor
Pavel Andreevich Iakovlev
Dmitry Valentinovich Morozov
Anna Nikolaevna Strelkova
Pavel Mikhailovich Gershovich
Tatiana Evgenievna Shugaeva
Sergei Aleksandrovich Legotskii
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 MA64631A1 publication Critical patent/MA64631A1/fr
Publication of MA64631B1 publication Critical patent/MA64631B1/fr

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0041Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01DNA viruses
    • C07K14/015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/50Mutagenesis
    • GPHYSICS
    • G16INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16BBIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B30/00ICT specially adapted for sequence analysis involving nucleotides or amino acids
    • G16B30/10Sequence alignment; Homology search
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special 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 présente invention se rapporte au domaine de la thérapie génique et de la biologie moléculaire. La présente invention concerne plus particulièrement un procédé de production de capside modifié de virus adéno-associé (aav) et un capside modifié de aav obtenu selon ce procédé, ainsi qu'un acide nucléique séparé codant ledit capside modifié, et un vecteur sur la base d'un virus adéno-associé recombinant pour administrer à un sujet une séquence hétérologue d'acide nucléique comprenant ledit capside modifié.
MA64631A 2021-08-20 2022-08-18 Procédé de production de capside modifié de virus adéno-associé MA64631B1 (fr)

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 (fr) 2021-08-20 2022-08-18 Procédé de production de capside modifié de virus adéno-associé

Publications (2)

Publication Number Publication Date
MA64631A1 true MA64631A1 (fr) 2024-05-31
MA64631B1 MA64631B1 (fr) 2024-12-31

Family

ID=85240917

Family Applications (1)

Application Number Title Priority Date Filing Date
MA64631A MA64631B1 (fr) 2021-08-20 2022-08-18 Procédé de production de capside modifié de virus adéno-associé

Country Status (17)

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

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 (fr) * 2007-04-09 2016-07-19 University Of Florida Research Foundation, Inc. Compositions a base de vecteurs raav comprenant des proteines de capside modifiees par la tyrosine et procedes d'utilisation correspondants
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
SI3693025T1 (sl) 2011-04-22 2022-04-29 The Regents Of The University Of California Virioni adeno-povezanega virusa z varianto kapsida in postopki za njihovo uporabo
HUE054087T2 (hu) 2012-04-18 2021-09-28 Childrens Hospital Philadelphia Készítmények és eljárások nagy hatékonyságú géntranszferre AAV kapszidvariánsok alkalmazásával
MX2018009100A (es) * 2016-01-26 2019-02-28 Defensin Therapeutics Aps Metodos para modular la flora intestinal.
CA3052829A1 (fr) * 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Methodes et compositions pour le transfert de genes a travers le systeme vasculaire
SG11202011191WA (en) * 2018-05-16 2020-12-30 Spark Therapeutics Inc Codon-optimized acid αlpha-glucosidase expression cassettes and methods of using same

Also Published As

Publication number Publication date
MX2024002200A (es) 2024-04-29
CA3229580A1 (fr) 2023-02-23
EP4389880A1 (fr) 2024-06-26
CR20240137A (es) 2024-07-29
CN118139974A (zh) 2024-06-04
ZA202402156B (en) 2024-10-30
IL310949A (en) 2024-04-01
CL2024000508A1 (es) 2024-08-30
AR126841A1 (es) 2023-11-22
US20250154526A1 (en) 2025-05-15
WO2023022631A1 (fr) 2023-02-23
TW202317770A (zh) 2023-05-01
PE20241995A1 (es) 2024-09-27
AU2022328531A1 (en) 2024-03-14
MA64631B1 (fr) 2024-12-31
CO2024001768A2 (es) 2024-07-08
ECSP24013031A (es) 2024-03-01

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