IL287491A - New aav capsids and preparations containing them - Google Patents

New aav capsids and preparations containing them

Info

Publication number
IL287491A
IL287491A IL287491A IL28749121A IL287491A IL 287491 A IL287491 A IL 287491A IL 287491 A IL287491 A IL 287491A IL 28749121 A IL28749121 A IL 28749121A IL 287491 A IL287491 A IL 287491A
Authority
IL
Israel
Prior art keywords
compositions containing
containing same
aav capsids
novel aav
novel
Prior art date
Application number
IL287491A
Other languages
English (en)
Hebrew (he)
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 IL287491A publication Critical patent/IL287491A/en

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    • 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
    • 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
    • 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
    • A61K48/0075Medicinal 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
    • 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
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • 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
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2750/14151Methods of production or purification of viral material
    • 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)
  • 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)
IL287491A 2019-04-29 2021-10-21 New aav capsids and preparations containing them IL287491A (en)

Applications Claiming Priority (4)

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
PCT/US2020/030273 WO2020223232A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
IL287491A true IL287491A (en) 2021-12-01

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
IL287491A IL287491A (en) 2019-04-29 2021-10-21 New aav capsids and preparations containing them
IL287492A IL287492A (en) 2019-04-29 2021-10-21 New aav capsids and preparations containing them
IL287498A IL287498A (en) 2019-04-29 2021-10-21 New aav capsids and preparations containing them

Family Applications After (2)

Application Number Title Priority Date Filing Date
IL287492A IL287492A (en) 2019-04-29 2021-10-21 New aav capsids and preparations containing them
IL287498A IL287498A (en) 2019-04-29 2021-10-21 New aav capsids and preparations containing them

Country Status (16)

Country Link
US (3) US12516351B2 (enExample)
EP (3) EP3976078A4 (enExample)
JP (3) JP7644026B2 (enExample)
KR (3) KR20220004680A (enExample)
CN (3) CN113853208A (enExample)
AU (3) AU2020266523A1 (enExample)
BR (3) BR112021021502A2 (enExample)
CA (3) CA3134379A1 (enExample)
CL (3) CL2021002768A1 (enExample)
CO (3) CO2021015928A2 (enExample)
IL (3) IL287491A (enExample)
MX (3) MX2021013266A (enExample)
PE (3) PE20212267A1 (enExample)
PH (3) PH12021552608A1 (enExample)
SG (3) SG11202111373YA (enExample)
WO (3) WO2020223232A1 (enExample)

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MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
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WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
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WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
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Also Published As

Publication number Publication date
JP2022530544A (ja) 2022-06-29
WO2020223236A1 (en) 2020-11-05
JP2022530656A (ja) 2022-06-30
CN113853207A (zh) 2021-12-28
US12516352B2 (en) 2026-01-06
CN113853208A (zh) 2021-12-28
BR112021020545A2 (pt) 2022-01-04
KR20220004681A (ko) 2022-01-11
SG11202111373YA (en) 2021-11-29
US12516351B2 (en) 2026-01-06
EP3976077A1 (en) 2022-04-06
CL2021002768A1 (es) 2022-05-27
CL2021002811A1 (es) 2022-05-27
MX2021013268A (es) 2021-11-17
CO2021015916A2 (es) 2021-11-30
KR20220004695A (ko) 2022-01-11
SG11202111102SA (en) 2021-11-29
EP3976077A4 (en) 2023-08-30
PE20212267A1 (es) 2021-11-30
CA3134379A1 (en) 2020-11-05
PE20220014A1 (es) 2022-01-11
AU2020266523A1 (en) 2021-11-04
CL2021002824A1 (es) 2022-05-27
PH12021552540A1 (en) 2022-06-20
MX2021013266A (es) 2021-11-17
CO2021015928A2 (es) 2021-11-30
JP7644026B2 (ja) 2025-03-11
SG11202111375RA (en) 2021-11-29
WO2020223232A1 (en) 2020-11-05
WO2020223231A1 (en) 2020-11-05
EP3962507A1 (en) 2022-03-09
US20220249705A1 (en) 2022-08-11
CN113853209B (zh) 2024-12-13
IL287498A (en) 2021-12-01
BR112021021502A2 (pt) 2022-07-19
AU2020264976A1 (en) 2021-11-11
BR112021020054A2 (pt) 2021-12-07
JP7690401B2 (ja) 2025-06-10
CA3134507A1 (en) 2020-11-05
CO2021015924A2 (es) 2021-11-30
AU2020264975A1 (en) 2021-11-11
US20220204990A1 (en) 2022-06-30
EP3976078A1 (en) 2022-04-06
PH12021552607A1 (en) 2022-06-27
EP3976078A4 (en) 2023-08-30
US20220220453A1 (en) 2022-07-14
CN113853209A (zh) 2021-12-28
PH12021552608A1 (en) 2022-06-20
JP2022530633A (ja) 2022-06-30
CA3134468A1 (en) 2020-11-05
PE20212334A1 (es) 2021-12-14
KR20220004680A (ko) 2022-01-11
IL287492A (en) 2021-12-01
MX2021013267A (es) 2021-11-17
EP3962507A4 (en) 2023-10-11

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