SG11202007942YA - Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor - Google Patents

Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Info

Publication number
SG11202007942YA
SG11202007942YA SG11202007942YA SG11202007942YA SG11202007942YA SG 11202007942Y A SG11202007942Y A SG 11202007942YA SG 11202007942Y A SG11202007942Y A SG 11202007942YA SG 11202007942Y A SG11202007942Y A SG 11202007942YA SG 11202007942Y A SG11202007942Y A SG 11202007942YA
Authority
SG
Singapore
Prior art keywords
aav
vectors
associated virus
uses therefor
novel adeno
Prior art date
Application number
SG11202007942YA
Inventor
James Wilson
April Tepe
Kevin Turner
Joshua Sims
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 SG11202007942YA publication Critical patent/SG11202007942YA/en

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Classifications

    • 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
    • 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
    • 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
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SG11202007942YA 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor SG11202007942YA (en)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201862635968P 2018-02-27 2018-02-27
US201862635964P 2018-02-27 2018-02-27
US201862663797P 2018-04-27 2018-04-27
US201862663788P 2018-04-27 2018-04-27
US201862667587P 2018-05-06 2018-05-06
US201862667881P 2018-05-07 2018-05-07
US201862667888P 2018-05-07 2018-05-07
US201862667585P 2018-05-29 2018-05-29
US201862677471P 2018-05-29 2018-05-29
US201862677474P 2018-05-29 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862703673P 2018-07-26 2018-07-26
US201862722382P 2018-08-24 2018-08-24
US201862722388P 2018-08-24 2018-08-24
PCT/US2019/019861 WO2019169004A1 (en) 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Publications (1)

Publication Number Publication Date
SG11202007942YA true SG11202007942YA (en) 2020-09-29

Family

ID=67809202

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202007942YA SG11202007942YA (en) 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Country Status (14)

Country Link
US (1) US20210123073A1 (en)
EP (1) EP3768695A4 (en)
JP (1) JP7498665B2 (en)
KR (1) KR20210006327A (en)
CN (1) CN112236443A (en)
AU (1) AU2019228504A1 (en)
BR (1) BR112020017278A2 (en)
CA (1) CA3091795A1 (en)
CL (1) CL2020002201A1 (en)
IL (1) IL276860A (en)
MA (1) MA52087A (en)
MX (1) MX2020008932A (en)
SG (1) SG11202007942YA (en)
WO (1) WO2019169004A1 (en)

Families Citing this family (17)

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Publication number Priority date Publication date Assignee Title
PT3356390T (en) 2015-09-28 2021-04-21 Univ Florida Methods and compositions for antibody-evading virus vectors
BR112020020266A2 (en) 2018-04-03 2021-01-19 Stridebio, Inc. VIRUSES WITH ANTIBODY EVASION
EP3884057A4 (en) * 2018-12-21 2022-09-07 The University of North Carolina at Chapel Hill Optimized galc genes and expression cassettes and their use
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
BR112022006530A2 (en) 2019-10-17 2022-07-05 Stridebio Inc ADENO-ASSOCIATED VIRAL VECTORS FOR THE TREATMENT OF NIEMANN-PICK TYPE C DISEASE
WO2021080991A1 (en) * 2019-10-21 2021-04-29 The Trustees Of The University Of Pennsylvania Aav3b variants with improved production yield and liver tropism
US20230204595A1 (en) * 2020-01-03 2023-06-29 Sarepta Therapeutics, Inc. Methods for analyzing aav capsid proteins
US20230151359A1 (en) * 2020-03-09 2023-05-18 University Of Massachusetts Gene replacement therapy for foxg1 syndrome
JP2023531451A (en) 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 Compositions and methods for treatment of gene therapy patients
EP4179097A1 (en) 2020-07-13 2023-05-17 The Trustees of The University of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
IL302127A (en) * 2020-10-18 2023-06-01 Univ Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4323520A1 (en) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
WO2023081807A1 (en) * 2021-11-04 2023-05-11 The Trustees of the University of Pennsylvania Penn Center for Innovation Compositions and methods for reducing pcsk9 levels in a subject
TW202338086A (en) 2022-01-10 2023-10-01 賓州大學委員會 Compositions useful in treatment of metachromatic leukodystrophy
TW202340467A (en) 2022-01-10 2023-10-16 賓州大學委員會 Compositions and methods useful for treatment of c9orf72-mediated disorders
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004083441A2 (en) * 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Random peptide library displayed on aav vectors
JP5054975B2 (en) 2003-09-30 2012-10-24 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 Syngeneic strains of adeno-associated virus (AAV), sequences, vectors containing them and uses thereof
JP5702519B2 (en) * 2005-04-07 2015-04-15 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 How to increase the function of an AAV vector
PT3198018T (en) * 2014-09-24 2021-02-24 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
CA2969145A1 (en) * 2014-11-26 2016-06-02 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
CN109661470A (en) 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 New A AV8 mutant capsids and composition containing it
IL305149A (en) * 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
MX2019001938A (en) * 2016-08-15 2019-07-04 Genzyme Corp Methods for detecting aav.
JOP20190200A1 (en) * 2017-02-28 2019-08-27 Univ Pennsylvania Compositions useful in treatment of spinal muscular atrophy
SG11201907714UA (en) * 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor

Also Published As

Publication number Publication date
AU2019228504A1 (en) 2020-09-10
US20210123073A1 (en) 2021-04-29
EP3768695A4 (en) 2022-04-06
CL2020002201A1 (en) 2020-12-18
IL276860A (en) 2020-10-29
JP7498665B2 (en) 2024-06-12
EP3768695A1 (en) 2021-01-27
CN112236443A (en) 2021-01-15
WO2019169004A9 (en) 2019-10-31
MA52087A (en) 2021-06-02
MX2020008932A (en) 2020-10-01
BR112020017278A2 (en) 2020-12-22
JP2021515548A (en) 2021-06-24
WO2019169004A8 (en) 2019-12-05
WO2019169004A1 (en) 2019-09-06
CA3091795A1 (en) 2019-09-06
KR20210006327A (en) 2021-01-18

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