CL2021002824A1 - Nuevas cápsulas de aav y composiciones que las contienen. - Google Patents

Nuevas cápsulas de aav y composiciones que las contienen.

Info

Publication number
CL2021002824A1
CL2021002824A1 CL2021002824A CL2021002824A CL2021002824A1 CL 2021002824 A1 CL2021002824 A1 CL 2021002824A1 CL 2021002824 A CL2021002824 A CL 2021002824A CL 2021002824 A CL2021002824 A CL 2021002824A CL 2021002824 A1 CL2021002824 A1 CL 2021002824A1
Authority
CL
Chile
Prior art keywords
capsules
compositions containing
new aav
aav
new
Prior art date
Application number
CL2021002824A
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 CL2021002824A1 publication Critical patent/CL2021002824A1/es

Links

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 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.
CL2021002824A 2019-04-29 2021-10-27 Nuevas cápsulas de aav y composiciones que las contienen. CL2021002824A1 (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
CL2021002824A1 true CL2021002824A1 (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 Before (2)

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.

Country Status (15)

Country Link
US (3) US20220249705A1 (es)
EP (3) EP3976078A4 (es)
JP (3) JP2022530544A (es)
KR (3) KR20220004681A (es)
CN (3) CN113853209A (es)
AU (3) AU2020264975A1 (es)
BR (3) BR112021020054A2 (es)
CA (3) CA3134507A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015928A2 (es)
IL (3) IL287492A (es)
MX (3) MX2021013266A (es)
PE (3) PE20220014A1 (es)
SG (3) SG11202111373YA (es)
WO (3) WO2020223236A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER 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
CA3200192A1 (en) * 2020-12-01 2022-06-09 Justin PERCIVAL Compositions and uses thereof for treatment of angelman syndrome
KR20230117157A (ko) 2020-12-01 2023-08-07 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 조직 특이적 표적화 모티프를 갖는 신규 조성물 및 이를 포함하는 조성물
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
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

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2406745C (en) * 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
AU2004278684B2 (en) * 2003-09-30 2011-05-12 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
CN112029737A (zh) * 2005-04-07 2020-12-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
CN101495624A (zh) * 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
CA3066596A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2015137802A1 (en) * 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
AU2016206478A1 (en) * 2015-01-16 2017-06-15 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
ES2945427T3 (es) * 2015-07-30 2023-07-03 Massachusetts Eye & Ear Infirmary Secuencias de AAV ancestrales y usos de las mismas
WO2017075335A1 (en) * 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
AU2016343979A1 (en) * 2015-10-29 2018-05-10 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
US11326182B2 (en) * 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR20240056729A (ko) * 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11192925B2 (en) * 2016-10-19 2021-12-07 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
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Also Published As

Publication number Publication date
KR20220004680A (ko) 2022-01-11
PE20212334A1 (es) 2021-12-14
MX2021013266A (es) 2021-11-17
JP2022530633A (ja) 2022-06-30
EP3962507A4 (en) 2023-10-11
IL287498A (en) 2021-12-01
US20220220453A1 (en) 2022-07-14
MX2021013268A (es) 2021-11-17
IL287492A (en) 2021-12-01
CA3134507A1 (en) 2020-11-05
BR112021021502A2 (pt) 2022-07-19
SG11202111373YA (en) 2021-11-29
EP3976077A4 (en) 2023-08-30
AU2020266523A1 (en) 2021-11-04
EP3962507A1 (en) 2022-03-09
IL287491A (en) 2021-12-01
BR112021020054A2 (pt) 2021-12-07
WO2020223231A1 (en) 2020-11-05
WO2020223232A1 (en) 2020-11-05
PE20212267A1 (es) 2021-11-30
CL2021002811A1 (es) 2022-05-27
AU2020264975A1 (en) 2021-11-11
EP3976077A1 (en) 2022-04-06
EP3976078A4 (en) 2023-08-30
MX2021013267A (es) 2021-11-17
KR20220004681A (ko) 2022-01-11
CN113853207A (zh) 2021-12-28
US20220249705A1 (en) 2022-08-11
WO2020223236A1 (en) 2020-11-05
CO2021015928A2 (es) 2021-11-30
CN113853209A (zh) 2021-12-28
BR112021020545A2 (pt) 2022-01-04
CA3134379A1 (en) 2020-11-05
US20220204990A1 (en) 2022-06-30
SG11202111102SA (en) 2021-11-29
CO2021015916A2 (es) 2021-11-30
CA3134468A1 (en) 2020-11-05
JP2022530656A (ja) 2022-06-30
JP2022530544A (ja) 2022-06-29
CO2021015924A2 (es) 2021-11-30
PE20220014A1 (es) 2022-01-11
CL2021002768A1 (es) 2022-05-27
SG11202111375RA (en) 2021-11-29
KR20220004695A (ko) 2022-01-11
EP3976078A1 (en) 2022-04-06
AU2020264976A1 (en) 2021-11-11
CN113853208A (zh) 2021-12-28

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