CA3134379A1 - Novel aav capsids and compositions containing same - Google Patents

Novel aav capsids and compositions containing same Download PDF

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Publication number
CA3134379A1
CA3134379A1 CA3134379A CA3134379A CA3134379A1 CA 3134379 A1 CA3134379 A1 CA 3134379A1 CA 3134379 A CA3134379 A CA 3134379A CA 3134379 A CA3134379 A CA 3134379A CA 3134379 A1 CA3134379 A1 CA 3134379A1
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CA
Canada
Prior art keywords
seq
acid sequence
aav
nucleic acid
raav
Prior art date
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Pending
Application number
CA3134379A
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English (en)
French (fr)
Inventor
Kalyani NAMBIAR
James M. Wilson
Qiang Wang
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University of Pennsylvania Penn
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Individual
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Publication of CA3134379A1 publication Critical patent/CA3134379A1/en
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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

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  • 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)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (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)
CA3134379A 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same Pending CA3134379A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962840184P 2019-04-29 2019-04-29
US62/840,184 2019-04-29
US201962913314P 2019-10-10 2019-10-10
US62/913,314 2019-10-10
US201962924095P 2019-10-21 2019-10-21
US62/924,095 2019-10-21
PCT/US2020/030266 WO2020223231A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
CA3134379A1 true CA3134379A1 (en) 2020-11-05

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
CA3134379A Pending CA3134379A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same
CA3134468A Pending CA3134468A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same
CA3134507A Pending CA3134507A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Family Applications After (2)

Application Number Title Priority Date Filing Date
CA3134468A Pending CA3134468A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same
CA3134507A Pending CA3134507A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Country Status (16)

Country Link
US (3) US12516352B2 (https=)
EP (3) EP3976077A4 (https=)
JP (3) JP7690401B2 (https=)
KR (3) KR20220004695A (https=)
CN (3) CN113853207A (https=)
AU (3) AU2020264976A1 (https=)
BR (3) BR112021020054A2 (https=)
CA (3) CA3134379A1 (https=)
CL (3) CL2021002768A1 (https=)
CO (3) CO2021015928A2 (https=)
IL (3) IL287492A (https=)
MX (3) MX2021013267A (https=)
PE (3) PE20220014A1 (https=)
PH (3) PH12021552607A1 (https=)
SG (3) SG11202111102SA (https=)
WO (3) WO2020223231A1 (https=)

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Publication number Publication date
CL2021002811A1 (es) 2022-05-27
EP3976077A1 (en) 2022-04-06
CN113853207A (zh) 2021-12-28
EP3976077A4 (en) 2023-08-30
CA3134468A1 (en) 2020-11-05
US20220220453A1 (en) 2022-07-14
EP3976078A1 (en) 2022-04-06
WO2020223232A1 (en) 2020-11-05
IL287492A (en) 2021-12-01
US12516352B2 (en) 2026-01-06
CA3134507A1 (en) 2020-11-05
WO2020223236A1 (en) 2020-11-05
WO2020223231A1 (en) 2020-11-05
PE20220014A1 (es) 2022-01-11
JP7644026B2 (ja) 2025-03-11
BR112021020054A2 (pt) 2021-12-07
PE20212267A1 (es) 2021-11-30
KR20220004680A (ko) 2022-01-11
MX2021013267A (es) 2021-11-17
CO2021015928A2 (es) 2021-11-30
CN113853209A (zh) 2021-12-28
AU2020264975A1 (en) 2021-11-11
JP7690401B2 (ja) 2025-06-10
EP3976078A4 (en) 2023-08-30
CL2021002768A1 (es) 2022-05-27
US20220204990A1 (en) 2022-06-30
IL287498A (en) 2021-12-01
PH12021552540A1 (en) 2022-06-20
SG11202111373YA (en) 2021-11-29
BR112021020545A2 (pt) 2022-01-04
CO2021015924A2 (es) 2021-11-30
US12516351B2 (en) 2026-01-06
JP2022530633A (ja) 2022-06-30
US20220249705A1 (en) 2022-08-11
EP3962507A1 (en) 2022-03-09
JP2022530656A (ja) 2022-06-30
JP2022530544A (ja) 2022-06-29
SG11202111375RA (en) 2021-11-29
CL2021002824A1 (es) 2022-05-27
PH12021552607A1 (en) 2022-06-27
MX2021013268A (es) 2021-11-17
SG11202111102SA (en) 2021-11-29
AU2020266523A1 (en) 2021-11-04
PH12021552608A1 (en) 2022-06-20
AU2020264976A1 (en) 2021-11-11
CO2021015916A2 (es) 2021-11-30
PE20212334A1 (es) 2021-12-14
CN113853209B (zh) 2024-12-13
KR20220004695A (ko) 2022-01-11
KR20220004681A (ko) 2022-01-11
BR112021021502A2 (pt) 2022-07-19
CN113853208A (zh) 2021-12-28
MX2021013266A (es) 2021-11-17
EP3962507A4 (en) 2023-10-11
IL287491A (en) 2021-12-01

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