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
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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)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (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 (4) US20220249705A1 (enExample)
EP (3) EP3976077A4 (enExample)
JP (3) JP7690401B2 (enExample)
KR (3) KR20220004695A (enExample)
CN (3) CN113853207A (enExample)
AU (3) AU2020264975A1 (enExample)
BR (3) BR112021020054A2 (enExample)
CA (3) CA3134379A1 (enExample)
CL (3) CL2021002768A1 (enExample)
CO (3) CO2021015924A2 (enExample)
IL (3) IL287498A (enExample)
MX (3) MX2021013268A (enExample)
PE (3) PE20212334A1 (enExample)
PH (3) PH12021552608A1 (enExample)
SG (3) SG11202111375RA (enExample)
WO (3) WO2020223232A1 (enExample)

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AU2022262771A1 (en) 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
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CL2021002811A1 (es) 2022-05-27
MX2021013266A (es) 2021-11-17
IL287491A (en) 2021-12-01
KR20220004680A (ko) 2022-01-11
US20220204990A1 (en) 2022-06-30
PE20212267A1 (es) 2021-11-30
US20260201416A1 (en) 2026-07-16
JP2022530633A (ja) 2022-06-30
IL287498A (en) 2021-12-01
MX2021013268A (es) 2021-11-17
CO2021015916A2 (es) 2021-11-30
PH12021552607A1 (en) 2022-06-27
EP3976078A1 (en) 2022-04-06
SG11202111373YA (en) 2021-11-29
JP2022530544A (ja) 2022-06-29
EP3962507A1 (en) 2022-03-09
AU2020266523A1 (en) 2021-11-04
WO2020223232A1 (en) 2020-11-05
CA3134507A1 (en) 2020-11-05
EP3976078A4 (en) 2023-08-30
SG11202111375RA (en) 2021-11-29
KR20220004681A (ko) 2022-01-11
JP2022530656A (ja) 2022-06-30
EP3962507A4 (en) 2023-10-11
BR112021020054A2 (pt) 2021-12-07
CN113853207A (zh) 2021-12-28
PH12021552608A1 (en) 2022-06-20
MX2021013267A (es) 2021-11-17
WO2020223231A1 (en) 2020-11-05
IL287492A (en) 2021-12-01
CO2021015924A2 (es) 2021-11-30
US20220220453A1 (en) 2022-07-14
US12516351B2 (en) 2026-01-06
CN113853209A (zh) 2021-12-28
PE20212334A1 (es) 2021-12-14
PH12021552540A1 (en) 2022-06-20
CL2021002768A1 (es) 2022-05-27
PE20220014A1 (es) 2022-01-11
US12516352B2 (en) 2026-01-06
CA3134468A1 (en) 2020-11-05
WO2020223236A1 (en) 2020-11-05
JP7690401B2 (ja) 2025-06-10
EP3976077A1 (en) 2022-04-06
CO2021015928A2 (es) 2021-11-30
EP3976077A4 (en) 2023-08-30
CL2021002824A1 (es) 2022-05-27
US20220249705A1 (en) 2022-08-11
SG11202111102SA (en) 2021-11-29
AU2020264976A1 (en) 2021-11-11
AU2020264975A1 (en) 2021-11-11
BR112021021502A2 (pt) 2022-07-19
CN113853208A (zh) 2021-12-28
BR112021020545A2 (pt) 2022-01-04
KR20220004695A (ko) 2022-01-11
CN113853209B (zh) 2024-12-13
JP7644026B2 (ja) 2025-03-11

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