MX2021013267A - Nuevas capsides de aav y composiciones que las contienen. - Google Patents

Nuevas capsides de aav y composiciones que las contienen.

Info

Publication number
MX2021013267A
MX2021013267A MX2021013267A MX2021013267A MX2021013267A MX 2021013267 A MX2021013267 A MX 2021013267A MX 2021013267 A MX2021013267 A MX 2021013267A MX 2021013267 A MX2021013267 A MX 2021013267A MX 2021013267 A MX2021013267 A MX 2021013267A
Authority
MX
Mexico
Prior art keywords
novel aav
compositions containing
containing same
aav capsids
novel
Prior art date
Application number
MX2021013267A
Other languages
English (en)
Inventor
James M Wilson
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 MX2021013267A publication Critical patent/MX2021013267A/es

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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

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 la presente se proporcionan nuevas cápsides de AAV y rAAV que las comprenden; en una forma de realización, los vectores que utilizan una nueva cápside de AAV demuestran una transducción mayor de un tejido diana seleccionado en comparación con un AAV del arte previo.
MX2021013267A 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen. MX2021013267A (es)

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/US2020/030281 WO2020223236A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
MX2021013267A true MX2021013267A (es) 2021-11-17

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
MX2021013266A MX2021013266A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.
MX2021013268A MX2021013268A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.
MX2021013267A MX2021013267A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.

Family Applications Before (2)

Application Number Title Priority Date Filing Date
MX2021013266A MX2021013266A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.
MX2021013268A MX2021013268A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.

Country Status (15)

Country Link
US (3) US20220220453A1 (es)
EP (3) EP3962507A4 (es)
JP (3) JP2022530656A (es)
KR (3) KR20220004680A (es)
CN (3) CN113853208A (es)
AU (3) AU2020264976A1 (es)
BR (3) BR112021020545A2 (es)
CA (3) CA3134468A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015924A2 (es)
IL (3) IL287498A (es)
MX (3) MX2021013266A (es)
PE (3) PE20212267A1 (es)
SG (3) SG11202111102SA (es)
WO (3) WO2020223236A1 (es)

Families Citing this family (13)

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JP2023531451A (ja) 2020-06-17 2023-07-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 遺伝子療法患者の治療のための組成物及び方法
US20230372539A1 (en) 2020-08-24 2023-11-23 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
IL302741A (en) 2020-11-11 2023-07-01 European Molecular Biology Laboratory Viral particles adapted for gene therapy
JP2023551903A (ja) 2020-12-01 2023-12-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異的標的化モチーフを有する新規組成物及びそれを含有する組成物
EP4255500A1 (en) * 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023168293A2 (en) * 2022-03-01 2023-09-07 The Trustees Of The University Of Pennsylvania Viral vector genome encoding an insulin fusion protein
WO2023168403A2 (en) * 2022-03-03 2023-09-07 The Trustees Of The University Of Pennsylvania Viral vectors encoding parathyroid hormone fusions and uses thereof in treating hypoparathyroidism
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
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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL220644B1 (pl) * 2001-11-13 2015-11-30 Univ Pennsylvania Wirus stowarzyszony z adenowirusem (AAV), kompozycja, wyizolowane białko kapsydowe, wyizolowane lub syntetyczne cząsteczki kwasu nukleinowego, sposób wytwarzania zrekombinowanego wirusa, komórka gospodarza
HUE033158T2 (en) * 2003-09-30 2017-11-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors containing them, and their use
EP1866422B1 (en) * 2005-04-07 2016-04-06 The Trustees of The University of Pennsylvania Method of increasing the function of an aav vector
WO2008027084A2 (en) * 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
CA2833908C (en) * 2010-04-23 2021-02-09 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
CA2942289A1 (en) * 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US10738087B2 (en) * 2015-07-30 2020-08-11 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
MX2018004755A (es) * 2015-10-29 2018-12-19 Voyager Therapeutics Inc Entrega de polinucleotidos dirigidos al sistema nervioso central.
US11326182B2 (en) * 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CA3024448A1 (en) * 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Modulatory polynucleotides
AU2017345470B2 (en) * 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids 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
US20220220453A1 (en) 2022-07-14
CA3134468A1 (en) 2020-11-05
AU2020264976A1 (en) 2021-11-11
BR112021020545A2 (pt) 2022-01-04
IL287491A (en) 2021-12-01
CA3134507A1 (en) 2020-11-05
PE20212334A1 (es) 2021-12-14
CN113853207A (zh) 2021-12-28
MX2021013268A (es) 2021-11-17
KR20220004680A (ko) 2022-01-11
US20220204990A1 (en) 2022-06-30
JP2022530544A (ja) 2022-06-29
CO2021015916A2 (es) 2021-11-30
EP3962507A4 (en) 2023-10-11
WO2020223236A1 (en) 2020-11-05
WO2020223232A1 (en) 2020-11-05
EP3962507A1 (en) 2022-03-09
SG11202111102SA (en) 2021-11-29
BR112021020054A2 (pt) 2021-12-07
CL2021002824A1 (es) 2022-05-27
IL287492A (en) 2021-12-01
WO2020223231A1 (en) 2020-11-05
EP3976078A1 (en) 2022-04-06
CL2021002768A1 (es) 2022-05-27
CO2021015924A2 (es) 2021-11-30
EP3976077A4 (en) 2023-08-30
AU2020266523A1 (en) 2021-11-04
CO2021015928A2 (es) 2021-11-30
MX2021013266A (es) 2021-11-17
JP2022530656A (ja) 2022-06-30
CN113853209A (zh) 2021-12-28
IL287498A (en) 2021-12-01
CA3134379A1 (en) 2020-11-05
SG11202111373YA (en) 2021-11-29
KR20220004695A (ko) 2022-01-11
AU2020264975A1 (en) 2021-11-11
JP2022530633A (ja) 2022-06-30
EP3976077A1 (en) 2022-04-06
BR112021021502A2 (pt) 2022-07-19
US20220249705A1 (en) 2022-08-11
PE20212267A1 (es) 2021-11-30
KR20220004681A (ko) 2022-01-11
CL2021002811A1 (es) 2022-05-27
SG11202111375RA (en) 2021-11-29
CN113853208A (zh) 2021-12-28
PE20220014A1 (es) 2022-01-11
EP3976078A4 (en) 2023-08-30

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