MA64631B1 - 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
MA64631B1
MA64631B1 MA64631A MA64631A MA64631B1 MA 64631 B1 MA64631 B1 MA 64631B1 MA 64631 A MA64631 A MA 64631A MA 64631 A MA64631 A MA 64631A MA 64631 B1 MA64631 B1 MA 64631B1
Authority
MA
Morocco
Prior art keywords
associated virus
adeno
capsid
modified
modified capsid
Prior art date
Application number
MA64631A
Other languages
English (en)
Other versions
MA64631A1 (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
Joint Stock Company "Biocad"
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 Joint Stock Company "Biocad" filed Critical Joint Stock Company "Biocad"
Publication of MA64631A1 publication Critical patent/MA64631A1/fr
Publication of MA64631B1 publication Critical patent/MA64631B1/fr

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 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 MA64631A1 (fr) 2024-05-31
MA64631B1 true 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 (19)

Country Link
US (1) US20250154526A1 (fr)
EP (1) EP4389880A4 (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)
JO (1) JOP20240035A1 (fr)
MA (1) MA64631B1 (fr)
MX (1) MX2024002200A (fr)
PE (1) PE20241995A1 (fr)
PY (1) PY2272100A (fr)
TW (1) TW202317770A (fr)
WO (1) WO2023022631A1 (fr)
ZA (1) ZA202402156B (fr)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP2623605B1 (fr) * 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. Compositions de vecteur RAAV comprenant des protéines de capside à tyrosine modifié et procédés d'utilisation
US11905312B2 (en) * 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature

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
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
KR20180121489A (ko) * 2016-01-26 2018-11-07 디펜신 테라퓨틱스 에이피에스 장내 미생물을 조절하는 방법
FI3445773T3 (fi) * 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
WO2019210267A2 (fr) * 2018-04-27 2019-10-31 Spark Therapeutics, Inc. Capsides modifiées de vaa à tropisme accru et vecteurs de vaa comprenant les capsides modifiées et leurs procédés de préparation et leurs méthodes d'utilisation
CA3100213A1 (fr) * 2018-05-16 2019-11-21 Spark Therapeutics, Inc. Cassettes d'expression d'alpha-glucosidase acide optimisees par des codons et leurs methodes d'utilisation
RU2751592C2 (ru) * 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2623605B1 (fr) * 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. Compositions de vecteur RAAV comprenant des protéines de capside à tyrosine modifié et procédés d'utilisation
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
US11905312B2 (en) * 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature

Also Published As

Publication number Publication date
TW202317770A (zh) 2023-05-01
IL310949A (en) 2024-04-01
CN118139974A (zh) 2024-06-04
PE20241995A1 (es) 2024-09-27
ECSP24013031A (es) 2024-03-01
CL2024000508A1 (es) 2024-08-30
US20250154526A1 (en) 2025-05-15
CA3229580A1 (fr) 2023-02-23
MA64631A1 (fr) 2024-05-31
EP4389880A1 (fr) 2024-06-26
WO2023022631A1 (fr) 2023-02-23
JOP20240035A1 (ar) 2024-02-20
ZA202402156B (en) 2024-10-30
CR20240137A (es) 2024-07-29
CO2024001768A2 (es) 2024-07-08
PY2272100A (es) 2023-05-04
EP4389880A4 (fr) 2026-04-08
AR126841A1 (es) 2023-11-22
MX2024002200A (es) 2024-04-29
AU2022328531A1 (en) 2024-03-14

Similar Documents

Publication Publication Date Title
Mrksich et al. Influence of ionizable lipid tail length on lipid nanoparticle delivery of mRNA of varying length
WO2023024500A1 (fr) Constructions et procédés de préparation d'arn circulaire
MA62178A1 (fr) Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale
MX2023007204A (es) Lipidos ionizables.
MA49599B1 (fr) Anticorps spécifiques à cd47 et pd-l1
US20190119198A1 (en) Biodegradable amino-ester nanomaterials for nucleic acid delivery
MA56142A1 (fr) Protéine modifiée séparée vp1 de capside de aav5
MA46230A (fr) Mutations de protéine d'enveloppe du vih stabilisant la forme trimère
WO2023031392A3 (fr) Nouvelles nanoparticules lipidiques pour l'administration d'acides nucléiques comprenant de la phosphatidylsérine
CA2604532A1 (fr) Molecules arn a faible activation
PE20240086A1 (es) Anticuerpos anti-cd122 y usos de estos
FR3104976B1 (fr) Composition à base de rétinol
MA64631B1 (fr) Procédé de production de capside modifié de virus adéno-associé
JP2009062372A (ja) 高分子質量レクチンの生産
WO2023069967A3 (fr) Compositions utiles dans le traitement d'un trouble du déficit en cdkl5 (cdd)
MX2025012376A (es) Métodos y composiciones farmacéuticas de apoe para el tratamiento y la prevención de enfermedad de alzheimer
CA2470579A1 (fr) Production efficace de fragments f(ab')2 dans des cellules de mammiferes
MX2022013504A (es) Composiciones útiles en el tratamiento del trastorno de deficiencia de cdkl5 (cdd).
MA64618B2 (fr) Protéine modifiée séparée vp1 de capside aav9
CN120513298A (zh) 具有用于提高mRNA胞内稳定性和生物发生的套索帽结构的RNA及其用途
Kumar et al. Unveiling Fundamentals and Research Opportunities of mRNA Technology
CO2025012267A2 (es) Anticuerpo biespecífico aislado que se une específicamente al cd3 y al antígeno tumoral, y uso del mismo.
MX2025000200A (es) Metodos y composiciones farmaceuticas para el tratamiento y la prevencion de la enfermedad de alzheimer
MA62371A1 (fr) Acide nucléique à codons optimisés codant pour la protéine du facteur fix
AR126727A1 (es) Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4