SG11202111373YA - Novel aav capsids and compositions containing same - Google Patents

Novel aav capsids and compositions containing same

Info

Publication number
SG11202111373YA
SG11202111373YA SG11202111373YA SG11202111373YA SG11202111373YA SG 11202111373Y A SG11202111373Y A SG 11202111373YA SG 11202111373Y A SG11202111373Y A SG 11202111373YA SG 11202111373Y A SG11202111373Y A SG 11202111373YA SG 11202111373Y A SG11202111373Y A SG 11202111373YA
Authority
SG
Singapore
Prior art keywords
compositions containing
containing same
aav capsids
novel aav
novel
Prior art date
Application number
SG11202111373YA
Inventor
Kalyani Nambiar
James Wilson
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 SG11202111373YA publication Critical patent/SG11202111373YA/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)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (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)
SG11202111373YA 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same SG11202111373YA (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/US2020/030281 WO2020223236A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
SG11202111373YA true SG11202111373YA (en) 2021-11-29

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
SG11202111375RA SG11202111375RA (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same
SG11202111102SA SG11202111102SA (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same
SG11202111373YA SG11202111373YA (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Family Applications Before (2)

Application Number Title Priority Date Filing Date
SG11202111375RA SG11202111375RA (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same
SG11202111102SA SG11202111102SA (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Country Status (15)

Country Link
US (3) US20220204990A1 (en)
EP (3) EP3976078A4 (en)
JP (3) JP2022530656A (en)
KR (3) KR20220004680A (en)
CN (3) CN113853209A (en)
AU (3) AU2020266523A1 (en)
BR (3) BR112021020545A2 (en)
CA (3) CA3134507A1 (en)
CL (3) CL2021002768A1 (en)
CO (3) CO2021015916A2 (en)
IL (3) IL287498A (en)
MX (3) MX2021013267A (en)
PE (3) PE20212267A1 (en)
SG (3) SG11202111375RA (en)
WO (3) WO2020223232A1 (en)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115968302A (en) 2020-06-17 2023-04-14 宾夕法尼亚州大学信托人 Compositions and methods for treating gene therapy patients
JP2023543125A (en) 2020-08-24 2023-10-13 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 Viral vectors encoding GLP-1 receptor agonist fusions and their use in the treatment of metabolic diseases
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
EP4244209A1 (en) 2020-11-11 2023-09-20 European Molecular Biology Laboratory Modified viral particles for gene therapy
AU2021392642A1 (en) * 2020-12-01 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
IL303236A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
MX2023012513A (en) 2021-04-23 2023-12-15 Univ Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same.
AU2023211652A1 (en) 2022-01-25 2024-08-08 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
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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ532635A (en) * 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2292780T3 (en) * 2003-09-30 2017-12-04 Univ Pennsylvania Clades and sequences of adeno-associated virus (AAV), vectors containing them, and uses thereof
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
JP2009535339A (en) * 2006-04-28 2009-10-01 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 Modified AAV vectors with reduced capsid immunogenicity and uses thereof
EP2826860B1 (en) * 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
IL247729B2 (en) * 2014-03-10 2023-09-01 Uniqure Ip Bv Further improved aav vectors produced in insect cells
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3632923A1 (en) * 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
DK3872085T5 (en) * 2015-07-30 2024-08-12 Massachusetts Eye & Ear Infirmary STOCK VIRUS SEQUENCES AND USES THEREOF
EP3368054A4 (en) * 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3368065A4 (en) * 2015-10-29 2019-03-20 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
KR102392236B1 (en) * 2016-05-18 2022-05-03 보이저 테라퓨틱스, 인크. Regulatory Polynucleotides
EP3528785A4 (en) * 2016-10-19 2020-12-02 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 (en) * 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
KR20200044793A (en) * 2017-08-03 2020-04-29 보이저 테라퓨틱스, 인크. Compositions and methods for delivery of AAV

Also Published As

Publication number Publication date
CA3134379A1 (en) 2020-11-05
US20220220453A1 (en) 2022-07-14
PE20212334A1 (en) 2021-12-14
WO2020223236A1 (en) 2020-11-05
SG11202111102SA (en) 2021-11-29
CO2021015924A2 (en) 2021-11-30
WO2020223231A1 (en) 2020-11-05
EP3976077A1 (en) 2022-04-06
BR112021020054A2 (en) 2021-12-07
IL287491A (en) 2021-12-01
KR20220004680A (en) 2022-01-11
EP3962507A1 (en) 2022-03-09
EP3962507A4 (en) 2023-10-11
SG11202111375RA (en) 2021-11-29
AU2020264976A1 (en) 2021-11-11
CA3134507A1 (en) 2020-11-05
AU2020264975A1 (en) 2021-11-11
CO2021015928A2 (en) 2021-11-30
WO2020223232A1 (en) 2020-11-05
CN113853208A (en) 2021-12-28
CL2021002768A1 (en) 2022-05-27
CN113853207A (en) 2021-12-28
IL287498A (en) 2021-12-01
PE20212267A1 (en) 2021-11-30
CO2021015916A2 (en) 2021-11-30
EP3976078A1 (en) 2022-04-06
EP3976078A4 (en) 2023-08-30
MX2021013267A (en) 2021-11-17
MX2021013266A (en) 2021-11-17
PE20220014A1 (en) 2022-01-11
BR112021020545A2 (en) 2022-01-04
IL287492A (en) 2021-12-01
JP2022530544A (en) 2022-06-29
CA3134468A1 (en) 2020-11-05
KR20220004695A (en) 2022-01-11
CL2021002811A1 (en) 2022-05-27
JP2022530633A (en) 2022-06-30
US20220204990A1 (en) 2022-06-30
JP2022530656A (en) 2022-06-30
US20220249705A1 (en) 2022-08-11
EP3976077A4 (en) 2023-08-30
BR112021021502A2 (en) 2022-07-19
CL2021002824A1 (en) 2022-05-27
AU2020266523A1 (en) 2021-11-04
CN113853209A (en) 2021-12-28
KR20220004681A (en) 2022-01-11
MX2021013268A (en) 2021-11-17

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