BR112021020054A2 - Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos - Google Patents

Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos

Info

Publication number
BR112021020054A2
BR112021020054A2 BR112021020054A BR112021020054A BR112021020054A2 BR 112021020054 A2 BR112021020054 A2 BR 112021020054A2 BR 112021020054 A BR112021020054 A BR 112021020054A BR 112021020054 A BR112021020054 A BR 112021020054A BR 112021020054 A2 BR112021020054 A2 BR 112021020054A2
Authority
BR
Brazil
Prior art keywords
aav
capsids
same
adeno
compositions containing
Prior art date
Application number
BR112021020054A
Other languages
English (en)
Portuguese (pt)
Inventor
M Wilson James
Kalyani Nambiar
Qiang Wang
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 BR112021020054A2 publication Critical patent/BR112021020054A2/pt

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    • CCHEMISTRY; METALLURGY
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    • 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
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    • 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

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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)
  • 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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Materials For Medical Uses (AREA)
BR112021020054A 2019-04-29 2020-04-28 Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos BR112021020054A2 (pt)

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/US2020/030266 WO2020223231A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
BR112021020054A2 true BR112021020054A2 (pt) 2021-12-07

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
BR112021020054A BR112021020054A2 (pt) 2019-04-29 2020-04-28 Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos
BR112021021502A BR112021021502A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos
BR112021020545A BR112021020545A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos

Family Applications After (2)

Application Number Title Priority Date Filing Date
BR112021021502A BR112021021502A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos
BR112021020545A BR112021020545A2 (pt) 2019-04-29 2020-04-28 Capsídeos de aav e composições contendo os mesmos

Country Status (16)

Country Link
US (3) US20220249705A1 (enExample)
EP (3) EP3962507A4 (enExample)
JP (3) JP7690401B2 (enExample)
KR (3) KR20220004681A (enExample)
CN (3) CN113853207A (enExample)
AU (3) AU2020264975A1 (enExample)
BR (3) BR112021020054A2 (enExample)
CA (3) CA3134468A1 (enExample)
CL (3) CL2021002768A1 (enExample)
CO (3) CO2021015916A2 (enExample)
IL (3) IL287492A (enExample)
MX (3) MX2021013266A (enExample)
PE (3) PE20220014A1 (enExample)
PH (3) PH12021552608A1 (enExample)
SG (3) SG11202111102SA (enExample)
WO (3) WO2020223236A1 (enExample)

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WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CN116648448A (zh) 2020-11-11 2023-08-25 欧洲分子生物学实验室 用于基因治疗的修饰病毒粒子
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
MX2023006444A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
EP4469584A1 (en) 2022-01-25 2024-12-04 The Trustees of The University of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
IL315248A (en) * 2022-03-01 2024-10-01 Univ Pennsylvania Viral vector genome encoding an insulin fusion protein
IL315251A (en) * 2022-03-03 2024-10-01 Univ Pennsylvania Viral vectors encoding parathyroid hormone fusions and their uses for the treatment of hypoparathyroidism
KR20250007064A (ko) 2022-04-06 2025-01-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Her2 양성 전이성 유방암 및 기타 암을 치료하기 위한 조성물 및 방법
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2024015966A2 (en) * 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
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
GB202319008D0 (en) * 2023-12-13 2024-01-24 Ucl Business Ltd AAV-mediated gene therapy of diseases caused by impaired function of GLDC
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease

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Publication number Publication date
CL2021002768A1 (es) 2022-05-27
US20220220453A1 (en) 2022-07-14
CN113853207A (zh) 2021-12-28
CA3134507A1 (en) 2020-11-05
WO2020223236A1 (en) 2020-11-05
KR20220004681A (ko) 2022-01-11
CA3134379A1 (en) 2020-11-05
JP7690401B2 (ja) 2025-06-10
BR112021020545A2 (pt) 2022-01-04
SG11202111373YA (en) 2021-11-29
CA3134468A1 (en) 2020-11-05
MX2021013268A (es) 2021-11-17
AU2020266523A1 (en) 2021-11-04
AU2020264976A1 (en) 2021-11-11
MX2021013267A (es) 2021-11-17
SG11202111102SA (en) 2021-11-29
EP3976077A1 (en) 2022-04-06
PE20212267A1 (es) 2021-11-30
CL2021002824A1 (es) 2022-05-27
EP3976078A4 (en) 2023-08-30
PE20212334A1 (es) 2021-12-14
PH12021552540A1 (en) 2022-06-20
IL287498A (en) 2021-12-01
PE20220014A1 (es) 2022-01-11
KR20220004695A (ko) 2022-01-11
EP3976077A4 (en) 2023-08-30
US20220249705A1 (en) 2022-08-11
CO2021015916A2 (es) 2021-11-30
MX2021013266A (es) 2021-11-17
CN113853209A (zh) 2021-12-28
EP3962507A1 (en) 2022-03-09
JP2022530633A (ja) 2022-06-30
EP3976078A1 (en) 2022-04-06
IL287491A (en) 2021-12-01
IL287492A (en) 2021-12-01
SG11202111375RA (en) 2021-11-29
WO2020223231A1 (en) 2020-11-05
CN113853208A (zh) 2021-12-28
JP2022530656A (ja) 2022-06-30
JP2022530544A (ja) 2022-06-29
JP7644026B2 (ja) 2025-03-11
PH12021552607A1 (en) 2022-06-27
EP3962507A4 (en) 2023-10-11
BR112021021502A2 (pt) 2022-07-19
CN113853209B (zh) 2024-12-13
CO2021015924A2 (es) 2021-11-30
AU2020264975A1 (en) 2021-11-11
WO2020223232A1 (en) 2020-11-05
KR20220004680A (ko) 2022-01-11
US20220204990A1 (en) 2022-06-30
CL2021002811A1 (es) 2022-05-27
PH12021552608A1 (en) 2022-06-20
CO2021015928A2 (es) 2021-11-30

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