CL2021002811A1 - Nuevas cápsides de aav y composiciones que las contienen. - Google Patents

Nuevas cápsides de aav y composiciones que las contienen.

Info

Publication number
CL2021002811A1
CL2021002811A1 CL2021002811A CL2021002811A CL2021002811A1 CL 2021002811 A1 CL2021002811 A1 CL 2021002811A1 CL 2021002811 A CL2021002811 A CL 2021002811A CL 2021002811 A CL2021002811 A CL 2021002811A CL 2021002811 A1 CL2021002811 A1 CL 2021002811A1
Authority
CL
Chile
Prior art keywords
novel aav
compositions containing
aav capsids
novel
aav
Prior art date
Application number
CL2021002811A
Other languages
English (en)
Inventor
James M Wilson
Kalyani Nambiar
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of CL2021002811A1 publication Critical patent/CL2021002811A1/es

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    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Materials For Medical Uses (AREA)

Abstract

En el presente documento se proporcionan nuevas cápsides de AAV y rAAV que las comprenden. En una forma de realización, los vectores que emplean una nueva cápside de AAV muestran una mayor transducción de un tejido diana seleccionado en comparación con un AAV de la técnica anterior.
CL2021002811A 2019-04-29 2021-10-26 Nuevas cápsides de aav y composiciones que las contienen. CL2021002811A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962840184P 2019-04-29 2019-04-29
US201962913314P 2019-10-10 2019-10-10
US201962924095P 2019-10-21 2019-10-21

Publications (1)

Publication Number Publication Date
CL2021002811A1 true CL2021002811A1 (es) 2022-05-27

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
CL2021002768A CL2021002768A1 (es) 2019-04-29 2021-10-21 Nuevas cápsulas de avv y composiciones que las contienen.
CL2021002811A CL2021002811A1 (es) 2019-04-29 2021-10-26 Nuevas cápsides de aav y composiciones que las contienen.
CL2021002824A CL2021002824A1 (es) 2019-04-29 2021-10-27 Nuevas cápsulas de aav y composiciones que las contienen.

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CL2021002768A CL2021002768A1 (es) 2019-04-29 2021-10-21 Nuevas cápsulas de avv y composiciones que las contienen.

Family Applications After (1)

Application Number Title Priority Date Filing Date
CL2021002824A CL2021002824A1 (es) 2019-04-29 2021-10-27 Nuevas cápsulas de aav y composiciones que las contienen.

Country Status (16)

Country Link
US (3) US12516351B2 (es)
EP (3) EP3976078A4 (es)
JP (3) JP7644026B2 (es)
KR (3) KR20220004681A (es)
CN (3) CN113853207A (es)
AU (3) AU2020264975A1 (es)
BR (3) BR112021020545A2 (es)
CA (3) CA3134379A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015916A2 (es)
IL (3) IL287492A (es)
MX (3) MX2021013266A (es)
PE (3) PE20212267A1 (es)
PH (3) PH12021552608A1 (es)
SG (3) SG11202111375RA (es)
WO (3) WO2020223236A1 (es)

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IL287492A (en) 2021-12-01
JP2022530544A (ja) 2022-06-29
EP3962507A4 (en) 2023-10-11
PH12021552607A1 (en) 2022-06-27
US12516351B2 (en) 2026-01-06
CL2021002768A1 (es) 2022-05-27
PH12021552540A1 (en) 2022-06-20
JP2022530656A (ja) 2022-06-30
US20220204990A1 (en) 2022-06-30
CO2021015916A2 (es) 2021-11-30
SG11202111375RA (en) 2021-11-29
AU2020264975A1 (en) 2021-11-11
PE20212267A1 (es) 2021-11-30
WO2020223232A1 (en) 2020-11-05
CN113853209B (zh) 2024-12-13
PE20212334A1 (es) 2021-12-14
JP7644026B2 (ja) 2025-03-11
CO2021015928A2 (es) 2021-11-30
MX2021013267A (es) 2021-11-17
US20220249705A1 (en) 2022-08-11
WO2020223231A1 (en) 2020-11-05
CA3134468A1 (en) 2020-11-05
US12516352B2 (en) 2026-01-06
SG11202111102SA (en) 2021-11-29
AU2020266523A1 (en) 2021-11-04
CL2021002824A1 (es) 2022-05-27
BR112021020054A2 (pt) 2021-12-07
PH12021552608A1 (en) 2022-06-20
CO2021015924A2 (es) 2021-11-30
IL287491A (en) 2021-12-01
IL287498A (en) 2021-12-01
CN113853207A (zh) 2021-12-28
CN113853208A (zh) 2021-12-28
KR20220004680A (ko) 2022-01-11
MX2021013268A (es) 2021-11-17
US20220220453A1 (en) 2022-07-14
BR112021021502A2 (pt) 2022-07-19
CN113853209A (zh) 2021-12-28
SG11202111373YA (en) 2021-11-29
KR20220004681A (ko) 2022-01-11
EP3976077A1 (en) 2022-04-06
EP3976077A4 (en) 2023-08-30
JP2022530633A (ja) 2022-06-30
EP3976078A1 (en) 2022-04-06
MX2021013266A (es) 2021-11-17
AU2020264976A1 (en) 2021-11-11
KR20220004695A (ko) 2022-01-11
EP3976078A4 (en) 2023-08-30
BR112021020545A2 (pt) 2022-01-04
EP3962507A1 (en) 2022-03-09
JP7690401B2 (ja) 2025-06-10
WO2020223236A1 (en) 2020-11-05
PE20220014A1 (es) 2022-01-11
CA3134379A1 (en) 2020-11-05
CA3134507A1 (en) 2020-11-05

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