MA64618B2 - Protéine modifiée séparée vp1 de capside aav9 - Google Patents

Protéine modifiée séparée vp1 de capside aav9

Info

Publication number
MA64618B2
MA64618B2 MA64618A MA64618A MA64618B2 MA 64618 B2 MA64618 B2 MA 64618B2 MA 64618 A MA64618 A MA 64618A MA 64618 A MA64618 A MA 64618A MA 64618 B2 MA64618 B2 MA 64618B2
Authority
MA
Morocco
Prior art keywords
aav9
modified protein
separate modified
aav9 capsid
capsid
Prior art date
Application number
MA64618A
Other languages
English (en)
Other versions
MA64618A1 (fr
Inventor
Pavel Andreevich IAKOVLEV
Dmitry Valentinovich MOROZOV
Anna Nikolaevna STRELKOVA
Pavel Mikhailovich GERSHOVICH
Tatiana Evgenievna SHUGAEVA
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 RU2021124726A external-priority patent/RU2825667C2/ru
Application filed by Joint Stock Company "Biocad" filed Critical Joint Stock Company "Biocad"
Publication of MA64618A1 publication Critical patent/MA64618A1/fr
Publication of MA64618B2 publication Critical patent/MA64618B2/fr

Links

Classifications

    • 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
    • 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
    • C07K14/075—Adenoviridae
    • 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
    • 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
    • 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
    • C12N15/861—Adenoviral vectors
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (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. L'invention concerne plus précisément une protéine modifiée séparée VP1 de capside de virus adéno-associé de sérotype 9 (AAV9), qui comprend une ou plusieurs substitutions d'acide aminé par rapport à la protéine VP1 de capside AAV9 de type sauvage qui permettent d'augmenter l'efficacité de la production (construction) d'un vecteur sur la d'un virus adéno-associé de sérotype 9 recombinant (r(AAV9), ainsi qu'un capside et un vecteur à base de celui-ci.
MA64618A 2021-08-20 2022-08-21 Protéine modifiée séparée vp1 de capside aav9 MA64618B2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
RU2021124726A RU2825667C2 (ru) 2021-08-20 Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе
PCT/RU2022/050258 WO2023022634A1 (fr) 2021-08-20 2022-08-21 Protéine modifiée séparée vp1 de capside aav9

Publications (2)

Publication Number Publication Date
MA64618A1 MA64618A1 (fr) 2024-04-30
MA64618B2 true MA64618B2 (fr) 2025-04-30

Family

ID=85240922

Family Applications (1)

Application Number Title Priority Date Filing Date
MA64618A MA64618B2 (fr) 2021-08-20 2022-08-21 Protéine modifiée séparée vp1 de capside aav9

Country Status (18)

Country Link
US (1) US20240350663A1 (fr)
EP (1) EP4389760A4 (fr)
CN (1) CN117836312A (fr)
AR (1) AR126839A1 (fr)
AU (1) AU2022329857A1 (fr)
CA (1) CA3229573A1 (fr)
CL (1) CL2024000481A1 (fr)
CO (1) CO2024001457A2 (fr)
CR (1) CR20240089A (fr)
EC (1) ECSP24013002A (fr)
IL (1) IL310947A (fr)
JO (1) JOP20240034A1 (fr)
MA (1) MA64618B2 (fr)
MX (1) MX2024002086A (fr)
PE (1) PE20241336A1 (fr)
PY (1) PY2272096A (fr)
TW (1) TW202317769A (fr)
WO (1) WO2023022634A1 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120842333A (zh) * 2025-06-20 2025-10-28 上海朗昇生物科技有限公司 工程化的腺相关病毒衣壳蛋白和载体及其用途

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016115382A1 (fr) * 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Procédés et compositions destinés au transfert de gènes ciblé
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
WO2017058892A2 (fr) * 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Procédés et compositions pour vecteurs viraux évitant les anticorps
WO2017070516A1 (fr) * 2015-10-22 2017-04-27 University Of Massachusetts Vecteurs du sérotype de virus adéno-associés ciblant la prostate
WO2018152333A1 (fr) * 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Méthodes et compositions pour le transfert de gènes à travers le système vasculaire
WO2020210713A1 (fr) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomaux

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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
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
DK2359869T3 (en) * 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP1668143B1 (fr) * 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Variantes des virus associes aux adenovirus (aav), sequences, vecteurs les contenant, et leur utilisation
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
JP6342886B2 (ja) 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
NZ716102A (en) * 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2019146745A1 (fr) * 2018-01-26 2019-08-01 国立大学法人徳島大学 Nouveau virion de virus adéno-associé pour traiter la maladie de tay-sachs et la maladie de sandhoff
EP3941530A4 (fr) * 2019-03-21 2022-12-14 PTC Therapeutics, Inc. Vecteur et procédé pour traiter le syndrome d'angelman
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
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение

Patent Citations (6)

* 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
WO2016115382A1 (fr) * 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Procédés et compositions destinés au transfert de gènes ciblé
WO2017058892A2 (fr) * 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Procédés et compositions pour vecteurs viraux évitant les anticorps
WO2017070516A1 (fr) * 2015-10-22 2017-04-27 University Of Massachusetts Vecteurs du sérotype de virus adéno-associés ciblant la prostate
WO2018152333A1 (fr) * 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Méthodes et compositions pour le transfert de gènes à travers le système vasculaire
WO2020210713A1 (fr) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomaux

Also Published As

Publication number Publication date
EP4389760A4 (fr) 2025-11-26
CR20240089A (es) 2024-07-29
CN117836312A (zh) 2024-04-05
MA64618A1 (fr) 2024-04-30
CL2024000481A1 (es) 2024-07-12
MX2024002086A (es) 2024-04-29
PE20241336A1 (es) 2024-07-03
EP4389760A1 (fr) 2024-06-26
AR126839A1 (es) 2023-11-22
ECSP24013002A (es) 2024-03-01
US20240350663A1 (en) 2024-10-24
WO2023022634A1 (fr) 2023-02-23
IL310947A (en) 2024-04-01
JOP20240034A1 (ar) 2024-02-20
CO2024001457A2 (es) 2024-06-17
CA3229573A1 (fr) 2023-02-23
AU2022329857A1 (en) 2024-04-04
PY2272096A (es) 2023-05-18
TW202317769A (zh) 2023-05-01

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