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AR124119A2 - Partículas virales modificadas y usos de estas - Google Patents

Partículas virales modificadas y usos de estas

Info

Publication number
AR124119A2
AR124119A2 ARP210103220A ARP210103220A AR124119A2 AR 124119 A2 AR124119 A2 AR 124119A2 AR P210103220 A ARP210103220 A AR P210103220A AR P210103220 A ARP210103220 A AR P210103220A AR 124119 A2 AR124119 A2 AR 124119A2
Authority
AR
Argentina
Prior art keywords
aav
capsid
capsid protein
particles
protein
Prior art date
Application number
ARP210103220A
Other languages
English (en)
Inventor
Leah Sabin
Christos Kyratsous
Sven Moller-Tank
Original Assignee
Regeneron Pharma
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
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of AR124119A2 publication Critical patent/AR124119A2/es

Links

Classifications

    • 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
    • A61K48/005Medicinal 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
    • 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
    • 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
    • C07K14/195Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/315Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • 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
    • C12N2750/14123Virus like particles [VLP]
    • 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
    • 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
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

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

Abstract

En la presente se proporcionan composiciones y métodos para adaptar partículas de virus adenoasociado (AAV) que comprenden cápsides de AAV de animales no primates, AAV remoto, o una combinación de estos. El AAV adaptado, en consecuencia, puede ser una plataforma de terapia génica viable para el tratamiento de un paciente que lo necesita, y puede ser particularmente útil en pacientes excluidos de modalidades de tratamiento actuales que implican partículas de AAV terapéuticas actuales debido a su alto título de anticuerpos contra las partículas de AAV terapéuticas actuales. Las partículas virales de AAV recombinante comprenden (i) una cápside de AAV que comprende proteínas de la cápside de AAV VP1, VP2 y VP3, y (ii) empaquetada dentro de la cápside de AAV, una secuencia de ácido nucleico que comprende una secuencia de Repetición Terminal Invertida (ITR) de AAV, en donde al menos una de la proteína de la cápside VP1 de AAV, cualquier porción de la proteína de la cápside VP1 de AAV, la proteína de la cápside VP2 de AAV, cualquier porción de la proteína de la cápside VP2 de AAV, la proteína de la cápside VP3 de AAV, y cualquier porción de la proteína de la cápside VP3 de AAV comprende una secuencia de aminoácidos.
ARP210103220A 2019-05-24 2021-11-19 Partículas virales modificadas y usos de estas AR124119A2 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962852791P 2019-05-24 2019-05-24

Publications (1)

Publication Number Publication Date
AR124119A2 true AR124119A2 (es) 2023-02-15

Family

ID=71094822

Family Applications (2)

Application Number Title Priority Date Filing Date
ARP200101465A AR118997A1 (es) 2019-05-24 2020-05-22 Partículas virales modificadas y usos de estas
ARP210103220A AR124119A2 (es) 2019-05-24 2021-11-19 Partículas virales modificadas y usos de estas

Family Applications Before (1)

Application Number Title Priority Date Filing Date
ARP200101465A AR118997A1 (es) 2019-05-24 2020-05-22 Partículas virales modificadas y usos de estas

Country Status (19)

Country Link
US (1) US20220241430A1 (es)
EP (1) EP3976631A1 (es)
JP (3) JP2022533438A (es)
KR (1) KR20220011664A (es)
CN (2) CN114989267A (es)
AR (2) AR118997A1 (es)
AU (1) AU2020283537A1 (es)
BR (1) BR112021023692A2 (es)
CA (1) CA3140386A1 (es)
CL (2) CL2021003096A1 (es)
CO (1) CO2021017692A2 (es)
IL (1) IL288233A (es)
MA (1) MA56035A (es)
MX (1) MX2021014338A (es)
PE (1) PE20212357A1 (es)
PH (1) PH12021552887A1 (es)
SG (1) SG11202112917PA (es)
TW (1) TW202110869A (es)
WO (1) WO2020242984A1 (es)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200128112A (ko) 2018-02-28 2020-11-11 유니버시티 오브 피츠버그-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Aav에 기반한 유전자 및 단백질 전달을 위한 모듈식 시스템
JP2022533438A (ja) * 2019-05-24 2022-07-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 改変ウイルス粒子およびその使用
WO2022187377A1 (en) * 2021-03-02 2022-09-09 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated viruses and methods and materials for making and using adeno-associated viruses
EP4314021A4 (en) * 2021-04-30 2025-12-17 Univ Duke COMPOSITIONS WITH CHIMERATIVE CAPSIBILITIES FROM ADENO-ASSOCIATED VIRUS AND METHOD FOR USE THEM
CA3222914A1 (en) * 2021-06-23 2022-12-29 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
CA3256953A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. VECTORS AND METHODS FOR IN VIVO ANTIBODY PRODUCTION
WO2023240124A1 (en) 2022-06-07 2023-12-14 Regeneron Pharmaceuticals, Inc. Pseudotyped viral particles for targeting tcr-expressing cells
WO2025042462A2 (en) * 2023-08-24 2025-02-27 Ohio State Innovation Foundation Assays and methods for quality control of genome packaging for gene therapy
WO2025054526A1 (en) * 2023-09-07 2025-03-13 Regeneron Pharmaceuticals, Inc. Production and purification of covalently surface modified adeno-associated virus
US20250163468A1 (en) * 2023-11-21 2025-05-22 Regeneron Pharmaceuticals, Inc. Covalently surface modified adeno-associated virus production by in vitro conjugation, and purification of covalently surface modified adeno-associated virus

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BR112019027854A2 (pt) 2017-06-27 2020-07-07 Regeneron Pharmaceuticals, Inc. proteínas de capsídeo viral recombinante, ácido nucleico isolado, vetor viral recombinante, composição, métodos de direcionamento do vetor viral recombinante de interesse a uma célula alvo, de distribuição de um nucleotídeo de interesse a uma célula alvo, de inativação de uma proteína do capsídeo viral e de produção de um vetor viral, célula empacotadora para produzir um vetor viral, e, molécula e proteína de ligação.
JP2022533438A (ja) * 2019-05-24 2022-07-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 改変ウイルス粒子およびその使用

Also Published As

Publication number Publication date
EP3976631A1 (en) 2022-04-06
US20220241430A1 (en) 2022-08-04
CN113874386A (zh) 2021-12-31
PE20212357A1 (es) 2021-12-17
JP2025032110A (ja) 2025-03-11
JP2025119015A (ja) 2025-08-13
WO2020242984A1 (en) 2020-12-03
TW202110869A (zh) 2021-03-16
JP2022533438A (ja) 2022-07-22
IL288233A (en) 2022-01-01
CL2023000629A1 (es) 2023-10-20
SG11202112917PA (en) 2021-12-30
CO2021017692A2 (es) 2022-01-17
CN114989267A (zh) 2022-09-02
CL2021003096A1 (es) 2022-09-20
CA3140386A1 (en) 2020-12-03
KR20220011664A (ko) 2022-01-28
MA56035A (fr) 2022-04-06
WO2020242984A9 (en) 2021-01-14
MX2021014338A (es) 2022-01-06
AR118997A1 (es) 2021-11-17
PH12021552887A1 (en) 2022-09-05
BR112021023692A2 (pt) 2022-01-04
AU2020283537A1 (en) 2021-12-16

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