PH12021552887A1 - Modified viral particles and uses thereof - Google Patents

Modified viral particles and uses thereof

Info

Publication number
PH12021552887A1
PH12021552887A1 PH1/2021/552887A PH12021552887A PH12021552887A1 PH 12021552887 A1 PH12021552887 A1 PH 12021552887A1 PH 12021552887 A PH12021552887 A PH 12021552887A PH 12021552887 A1 PH12021552887 A1 PH 12021552887A1
Authority
PH
Philippines
Prior art keywords
aav
particles
viral particles
modified viral
current
Prior art date
Application number
PH1/2021/552887A
Other languages
English (en)
Inventor
Christos Kyratsous
Sven Moller-Tank
Leah Sabin
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 PH12021552887A1 publication Critical patent/PH12021552887A1/en

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
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/315—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • 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

  • 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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PH1/2021/552887A 2019-05-24 2020-05-22 Modified viral particles and uses thereof PH12021552887A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962852791P 2019-05-24 2019-05-24
PCT/US2020/034328 WO2020242984A1 (en) 2019-05-24 2020-05-22 Modified viral particles and uses thereof

Publications (1)

Publication Number Publication Date
PH12021552887A1 true PH12021552887A1 (en) 2022-09-05

Family

ID=71094822

Family Applications (1)

Application Number Title Priority Date Filing Date
PH1/2021/552887A PH12021552887A1 (en) 2019-05-24 2020-05-22 Modified viral particles and uses thereof

Country Status (19)

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

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019227910B2 (en) 2018-02-28 2025-07-17 The Regents Of The University Of California A modular system for gene and protein delivery based on AAV
KR20220011664A (ko) * 2019-05-24 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 변형된 바이러스 입자 및 이의 용도
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
WO2022232575A1 (en) * 2021-04-30 2022-11-03 Duke University Compositions comprising adeno-associated virus chimera capsid proteins and methods of using the same
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
EP4536836A1 (en) 2022-06-07 2025-04-16 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
WO2025111473A1 (en) * 2023-11-21 2025-05-30 Regeneron Pharmaceuticals, Inc. Covalently surface modified adeno-associated virus production by in vitro conjugation, and purification of covalently surface modified adeno-associated virus

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997005266A1 (en) 1995-07-25 1997-02-13 Introgene B.V. Methods and means for targeted gene delivery
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
DK1127150T3 (da) 1998-11-05 2007-09-24 Univ Pennsylvania Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse
SG168422A1 (en) * 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
MY172472A (en) 2009-12-10 2019-11-26 Regeneron Pharma Mice that make heavy chain antibodies
GB201002362D0 (en) 2010-02-11 2010-03-31 Isis Innovation Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds
IL293294B2 (en) * 2013-03-15 2023-04-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired aav glycan vectors
ES2990174T3 (es) * 2014-03-10 2024-11-29 Uniqure Ip Bv Vectores de AAV mejorados producidos en células de insecto
BR112016021572A2 (pt) 2014-03-21 2017-10-03 Regeneron Pharma Métodos para preparar um animal não humano geneticamente modificado e para produzir proteínas de ligação, hibridoma, ácido nucleico, célula, e, proteína de ligação
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
LT3645553T (lt) * 2017-06-27 2023-04-25 Regeneron Pharmaceuticals, Inc. Modifikuoto tropizmo rekombinantinės viruso dalelės ir jų panaudojimas genetinės medžiagos tiksliniam įvedimui į žmogaus ląsteles
SI3645551T1 (sl) 2017-06-27 2024-06-28 Regeneron Pharmaceuticals, Inc. S tropizmi spremenjeni rekombinantni virusni vektorji in njihova uporaba za tarčni vnos genetskega materiala v človeške celice
KR20220011664A (ko) * 2019-05-24 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 변형된 바이러스 입자 및 이의 용도

Also Published As

Publication number Publication date
TW202110869A (zh) 2021-03-16
AR118997A1 (es) 2021-11-17
BR112021023692A2 (pt) 2022-01-04
CL2023000629A1 (es) 2023-10-20
CL2021003096A1 (es) 2022-09-20
EP3976631A1 (en) 2022-04-06
KR20220011664A (ko) 2022-01-28
US20220241430A1 (en) 2022-08-04
CN114989267A (zh) 2022-09-02
WO2020242984A9 (en) 2021-01-14
JP2022533438A (ja) 2022-07-22
CN113874386A (zh) 2021-12-31
CA3140386A1 (en) 2020-12-03
MX2021014338A (es) 2022-01-06
CO2021017692A2 (es) 2022-01-17
SG11202112917PA (en) 2021-12-30
WO2020242984A1 (en) 2020-12-03
IL288233A (en) 2022-01-01
MA56035A (fr) 2022-04-06
AR124119A2 (es) 2023-02-15
JP2025032110A (ja) 2025-03-11
PE20212357A1 (es) 2021-12-17
JP2025119015A (ja) 2025-08-13
AU2020283537A1 (en) 2021-12-16

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