CL2021002768A1 - Nuevas cápsulas de avv y composiciones que las contienen. - Google Patents

Nuevas cápsulas de avv y composiciones que las contienen.

Info

Publication number
CL2021002768A1
CL2021002768A1 CL2021002768A CL2021002768A CL2021002768A1 CL 2021002768 A1 CL2021002768 A1 CL 2021002768A1 CL 2021002768 A CL2021002768 A CL 2021002768A CL 2021002768 A CL2021002768 A CL 2021002768A CL 2021002768 A1 CL2021002768 A1 CL 2021002768A1
Authority
CL
Chile
Prior art keywords
avv
capsules
new
compositions containing
aav
Prior art date
Application number
CL2021002768A
Other languages
English (en)
Inventor
James M Wilson
Qiang Wang
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 CL2021002768A1 publication Critical patent/CL2021002768A1/es

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Classifications

    • 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)
  • 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)

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
CL2021002768A 2019-04-29 2021-10-21 Nuevas cápsulas de avv y composiciones que las contienen. CL2021002768A1 (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
CL2021002768A1 true CL2021002768A1 (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 After (2)

Application Number Title Priority Date Filing Date
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.

Country Status (15)

Country Link
US (3) US20220220453A1 (es)
EP (3) EP3976077A4 (es)
JP (3) JP2022530544A (es)
KR (3) KR20220004681A (es)
CN (3) CN113853208A (es)
AU (3) AU2020264975A1 (es)
BR (3) BR112021020545A2 (es)
CA (3) CA3134379A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015928A2 (es)
IL (3) IL287492A (es)
MX (3) MX2021013267A (es)
PE (3) PE20212334A1 (es)
SG (3) SG11202111102SA (es)
WO (3) WO2020223236A1 (es)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
EP4200429A1 (en) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CA3201533A1 (en) 2020-11-11 2022-05-19 European Molecular Biology Laboratory Modified viral particles for gene therapy
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
CA3200192A1 (en) 2020-12-01 2022-06-09 Justin PERCIVAL Compositions and uses thereof for treatment of angelman syndrome
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
IL314434A (en) 2022-01-25 2024-09-01 Univ Pennsylvania AAV capsids for enhanced cardiac transduction and liver targeting neutralization
AU2023228669A1 (en) * 2022-03-01 2024-09-19 Scout Bio, Inc. Viral vector genome encoding an insulin fusion protein
AU2023228691A1 (en) * 2022-03-03 2024-09-19 The Trustees Of The University Of Pennsylvania Viral vectors encoding parathyroid hormone fusions and uses thereof in treating hypoparathyroidism
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
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
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
WO2024130067A2 (en) 2022-12-17 2024-06-20 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

Family Cites Families (18)

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NZ600121A (en) * 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
CA3072423A1 (en) * 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
ES2426245T3 (es) * 2005-04-07 2013-10-22 The Trustees Of The University Of Pennsylvania Método para incrementar la funcionalidad de un vector de AAV
US20090317417A1 (en) * 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
DK2561073T3 (en) * 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
FI3117005T3 (fi) * 2014-03-10 2024-08-29 Uniqure Ip Bv Hyönteissoluissa tuotettuja lisää parannettuja aav-vektoreita
EP3151866B1 (en) * 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
HUE061388T2 (hu) * 2015-07-30 2023-06-28 Massachusetts Eye & Ear Infirmary Õsi vírusszekvenciák és alkalmazásaik
US20180230489A1 (en) * 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3368065A4 (en) * 2015-10-29 2019-03-20 Voyager Therapeutics, Inc. DISTRIBUTION OF POLYNUCLEOTIDES FOR TARGETING THE CENTRAL NERVOUS SYSTEM
EP3448874A4 (en) * 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
SG11201809699XA (en) * 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
EP3528785A4 (en) * 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same
JOP20190269A1 (ar) * 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3071978A1 (en) * 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav

Also Published As

Publication number Publication date
JP2022530633A (ja) 2022-06-30
WO2020223231A1 (en) 2020-11-05
EP3976077A1 (en) 2022-04-06
MX2021013266A (es) 2021-11-17
AU2020266523A1 (en) 2021-11-04
KR20220004681A (ko) 2022-01-11
EP3962507A4 (en) 2023-10-11
US20220249705A1 (en) 2022-08-11
MX2021013267A (es) 2021-11-17
EP3976077A4 (en) 2023-08-30
PE20212334A1 (es) 2021-12-14
JP2022530544A (ja) 2022-06-29
CL2021002824A1 (es) 2022-05-27
SG11202111373YA (en) 2021-11-29
US20220204990A1 (en) 2022-06-30
CN113853207A (zh) 2021-12-28
CA3134379A1 (en) 2020-11-05
WO2020223232A1 (en) 2020-11-05
JP2022530656A (ja) 2022-06-30
WO2020223236A1 (en) 2020-11-05
SG11202111102SA (en) 2021-11-29
EP3976078A1 (en) 2022-04-06
AU2020264976A1 (en) 2021-11-11
CA3134468A1 (en) 2020-11-05
AU2020264975A1 (en) 2021-11-11
CO2021015924A2 (es) 2021-11-30
IL287498A (en) 2021-12-01
PE20220014A1 (es) 2022-01-11
CL2021002811A1 (es) 2022-05-27
CO2021015928A2 (es) 2021-11-30
MX2021013268A (es) 2021-11-17
SG11202111375RA (en) 2021-11-29
IL287492A (en) 2021-12-01
IL287491A (en) 2021-12-01
EP3962507A1 (en) 2022-03-09
KR20220004680A (ko) 2022-01-11
CN113853208A (zh) 2021-12-28
US20220220453A1 (en) 2022-07-14
CN113853209A (zh) 2021-12-28
CO2021015916A2 (es) 2021-11-30
BR112021020545A2 (pt) 2022-01-04
CA3134507A1 (en) 2020-11-05
KR20220004695A (ko) 2022-01-11
BR112021021502A2 (pt) 2022-07-19
PE20212267A1 (es) 2021-11-30
EP3976078A4 (en) 2023-08-30
BR112021020054A2 (pt) 2021-12-07

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