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
Other languages
English (en)
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 Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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
US201862635964P 2018-02-27 2018-02-27
US201862635968P 2018-02-27 2018-02-27
US201862663788P 2018-04-27 2018-04-27
US201862663797P 2018-04-27 2018-04-27
US201862667587P 2018-05-06 2018-05-06
US201862667888P 2018-05-07 2018-05-07
US201862667881P 2018-05-07 2018-05-07
US201862667585P 2018-05-29 2018-05-29
US201862677474P 2018-05-29 2018-05-29
US201862677471P 2018-05-29 2018-05-29
US201862703673P 2018-07-26 2018-07-26
US201862703670P 2018-07-26 2018-07-26
US201862722382P 2018-08-24 2018-08-24
US201862722388P 2018-08-24 2018-08-24
PCT/US2019/019861 WO2019169004A1 (fr) 2018-02-27 2019-02-27 Nouveaux vecteurs de virus adéno-associés (aav), vecteurs aav ayant une déamidation de capside réduite et leurs utilisations

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

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10202107733QA (en) 2015-09-28 2021-09-29 Univ North Carolina Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2019195449A1 (fr) 2018-04-03 2019-10-10 Stridebio, Inc. Vecteurs de virus évitant les anticorps
EP3884057A4 (fr) * 2018-12-21 2022-09-07 The University of North Carolina at Chapel Hill Gènes galc optimisés et cassettes d'expression et leur utilisation
MX2021011468A (es) 2019-03-21 2021-12-15 Vectores de virus adenoasociados recombinantes.
WO2021076925A1 (fr) 2019-10-17 2021-04-22 Stridebio, Inc. Vecteurs de virus adéno-associés pour le traitement de la maladie de niemann-pick de type c
TW202130653A (zh) * 2019-10-21 2021-08-16 賓州大學委員會 具改進生產產率及肝臟趨性之aav3b變異體
MX2022008003A (es) * 2020-01-03 2022-10-07 Sarepta Therapeutics Inc Métodos para analizar proteínas de la cápside de aav.
WO2021183433A1 (fr) * 2020-03-09 2021-09-16 University Of Massachusetts Thérapie de remplacement de gène pour le syndrome de foxg1
WO2021257668A1 (fr) 2020-06-17 2021-12-23 The Trustees Of The University Of Pennsylvania Compositions et méthodes pour le traitement de patients de thérapie génique
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease
CA3195553A1 (fr) * 2020-10-18 2022-04-21 Qiang Wang Vecteur de virus adeno-associe(aav) ameliore et ses utilisations
CA3209779A1 (fr) 2021-02-01 2022-08-04 Regenxbio Inc. Therapie genique de ceroides-lipofuscinoses neuronales
JP2024515612A (ja) 2021-04-12 2024-04-10 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 球脊髄性筋萎縮症(sbma)の治療に有用な組成物
WO2023081807A1 (fr) * 2021-11-04 2023-05-11 The Trustees of the University of Pennsylvania Penn Center for Innovation Compositions et procédés pour réduire les taux de pcsk9 chez un sujet
WO2023133584A1 (fr) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions utiles dans le traitement d'une leucodystrophie métachromatique
WO2023133574A1 (fr) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions et méthodes utiles pour le traitement de troubles médiés par c9orf72
WO2023196892A1 (fr) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Immunisation passive avec des anticorps neutralisants anti-aav pour empêcher la transduction hors cible de vecteurs aav administrés par voie intrathécale

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070172460A1 (en) * 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
DK3211085T3 (da) * 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
DK2359867T3 (en) 2005-04-07 2015-01-05 Univ Pennsylvania A method for increasing an AAV vector function
WO2016049230A1 (fr) * 2014-09-24 2016-03-31 City Of Hope Variants de vecteur de virus adénoassocié pour une édition de haute efficacité du génome et procédés correspondants
EP3224362A4 (fr) * 2014-11-26 2018-06-06 The Regents of The University of California Compositions thérapeutiques comprenant des facteurs de transcription et leurs procédés de préparation et d'utilisation
IL298604A (en) * 2016-04-15 2023-01-01 Univ Pennsylvania Novel aav8 mutant capsids and preparations containing them
MX2019000962A (es) * 2016-07-26 2019-08-01 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado.
TN2019000047A1 (en) * 2016-08-15 2020-07-15 Genzyme Corp Methods for detecting aav
HRP20240257T1 (hr) * 2017-02-28 2024-05-24 The Trustees Of The University Of Pennsylvania Adeno-povezani virusni (aav) clade f vektor i njegova uporaba
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي

Also Published As

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

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