SG10201709552XA - Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer - Google Patents

Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer

Info

Publication number
SG10201709552XA
SG10201709552XA SG10201709552XA SG10201709552XA SG10201709552XA SG 10201709552X A SG10201709552X A SG 10201709552XA SG 10201709552X A SG10201709552X A SG 10201709552XA SG 10201709552X A SG10201709552X A SG 10201709552XA SG 10201709552X A SG10201709552X A SG 10201709552XA
Authority
SG
Singapore
Prior art keywords
capsid
modified
gene therapy
liver cancer
human liver
Prior art date
Application number
SG10201709552XA
Inventor
Arun Srivastava
Li Zhong
Sergei Zolotukhin
George V Aslanidi
Mavis Agbandje-Mckenna
Vliet Kim M Van
Original Assignee
Univ Florida
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
Priority claimed from US13/899,481 external-priority patent/US9920097B2/en
Application filed by Univ Florida filed Critical Univ Florida
Publication of SG10201709552XA publication Critical patent/SG10201709552XA/en

Links

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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/461Cellular immunotherapy characterised by the cell type used
    • A61K39/4615Dendritic cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/462Cellular immunotherapy characterized by the effect or the function of the cells
    • A61K39/4622Antigen presenting cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/463Cellular immunotherapy characterised by recombinant expression
    • A61K39/4634Antigenic peptides; polypeptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/464454Enzymes
    • A61K39/464457Telomerase or [telomerase reverse transcriptase [TERT]
    • 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
    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/005Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • 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
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/515Animal cells
    • A61K2039/5158Antigen-pulsed cells, e.g. T-cells
    • 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
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • 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
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • 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
    • C12N2750/14171Demonstrated in vivo effect
    • 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
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Manufacturing & Machinery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG10201709552XA 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer SG10201709552XA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US13/899,481 US9920097B2 (en) 2007-04-09 2013-05-21 Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use

Publications (1)

Publication Number Publication Date
SG10201709552XA true SG10201709552XA (en) 2018-01-30

Family

ID=51023068

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201709552XA SG10201709552XA (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
SG11201510757PA SG11201510757PA (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201510757PA SG11201510757PA (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer

Country Status (10)

Country Link
EP (2) EP2999791B1 (en)
JP (3) JP6628286B2 (en)
KR (4) KR102649543B1 (en)
CN (3) CN111763690A (en)
AU (3) AU2014274457B2 (en)
CA (1) CA2917018A1 (en)
DK (1) DK2999791T3 (en)
HK (1) HK1217730A1 (en)
SG (2) SG10201709552XA (en)
WO (1) WO2014193716A2 (en)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5410017A (en) * 1993-05-21 1995-04-25 Rohm And Haas Company Continuous thermal polycondensation process for preparing polypeptide polymers
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
WO2016126857A1 (en) 2015-02-03 2016-08-11 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
JP7064214B2 (en) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Methods and Compositions for Antibodies Avoidant Viral Vectors
US20200181644A1 (en) * 2015-10-22 2020-06-11 University Of Florida Research Foundation, Incorporated Synthetic combinatorial aav3 capsid library
MX2018007237A (en) * 2015-12-14 2018-12-11 Univ North Carolina Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors.
CA3033125A1 (en) * 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2018035503A1 (en) * 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
MX2020000244A (en) * 2017-06-27 2020-09-28 Regeneron Pharma Tropism-modified recombinant viral vectors and uses thereof for the targeted introduction of genetic material into human cells.
CA3075180A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Lipid nanoparticle formulations of non-viral, capsid-free dna vectors
JP2021510539A (en) * 2018-01-16 2021-04-30 シーエルエス セラピューティクス リミテッドCLS Therapeutics Limited Treatment of diseases by liver expression of enzymes with deoxyribonuclease (DNase) activity
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
WO2019191418A1 (en) * 2018-03-29 2019-10-03 Asklepios Biopharmaceutical, Inc. Liver tropic recombinant aav6 vectors that evade neutralization
EP3774854A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
EP3784697A4 (en) * 2018-04-27 2022-07-06 Spark Therapeutics, Inc. Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
JP7142815B2 (en) * 2018-06-21 2022-09-28 株式会社遺伝子治療研究所 Adeno-associated virus virions for gene transfer into human liver
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
WO2020079256A1 (en) * 2018-10-19 2020-04-23 Genethon Modulation, monitoring and prediction of the immune response directed against aav gene therapy vectors
SI3911354T1 (en) * 2019-01-18 2023-12-29 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
EA202192501A1 (en) 2019-03-21 2021-12-16 Страйдбайо, Инк. RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS
EP3736330A1 (en) * 2019-05-08 2020-11-11 European Molecular Biology Laboratory Modified adeno-associated virus (aav) particles for gene therapy
EP3999120A4 (en) * 2019-07-04 2024-02-21 Childrens Medical Res Institute Methods and aav vectors for in vivo transduction
AU2020311618A1 (en) * 2019-07-11 2022-03-03 Centre Hospitalier Universitaire De Nantes Chemically-modified adeno-associated virus
WO2021076925A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
US20230340526A1 (en) * 2019-11-25 2023-10-26 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target hepatocytes and evade the humoral immune response
WO2021108468A1 (en) * 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target human hepatocytes in the liver of humanized mice
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
AU2021267856A1 (en) * 2020-05-05 2022-12-01 The University Of North Carolina At Chapel Hill Modified adeno-associated virus 5 capsids and uses thereof
JP2023548432A (en) 2020-11-11 2023-11-16 ヨーロピアン モレキュラー バイオロジー ラボラトリー Modified virus particles for gene therapy
CN113647359A (en) * 2021-08-15 2021-11-16 芜湖职业技术学院 Construction method of tunicamycin injection induced acute liver injury mouse model
WO2023072181A1 (en) * 2021-10-28 2023-05-04 Shanghai Vitalgen Biopharma Co., Ltd. Adeno-associated virus capsid
WO2023201368A2 (en) * 2022-04-15 2023-10-19 Dyno Therapeutics, Inc. Capsid variants and methods of using the same

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
EP2357010B1 (en) * 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
EP2623605B1 (en) * 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. RAAV vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
CA2762118A1 (en) * 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
WO2012109570A1 (en) * 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CN103060331B (en) * 2012-12-05 2014-10-08 南京医科大学第一附属医院 Preparation method and application of capsid-protein-mutant double-stranded recombinant adeno-associated virus containing mediated-membrane-stabile CD40L gene

Also Published As

Publication number Publication date
AU2014274457A1 (en) 2016-01-21
HK1217730A1 (en) 2017-01-20
WO2014193716A2 (en) 2014-12-04
DK2999791T3 (en) 2019-03-18
CN105408486A (en) 2016-03-16
EP3492597A3 (en) 2019-08-28
JP7344595B2 (en) 2023-09-14
KR102649543B1 (en) 2024-03-21
JP2016520311A (en) 2016-07-14
EP2999791B1 (en) 2018-12-05
AU2020200041B2 (en) 2022-03-17
KR102234672B1 (en) 2021-04-06
WO2014193716A3 (en) 2015-01-22
KR102298579B1 (en) 2021-09-07
AU2014274457B2 (en) 2019-10-24
KR20220136477A (en) 2022-10-07
JP6628286B2 (en) 2020-01-08
JP2022068181A (en) 2022-05-09
EP2999791A2 (en) 2016-03-30
EP3492597A2 (en) 2019-06-05
JP2020048571A (en) 2020-04-02
KR20160010617A (en) 2016-01-27
JP7021786B2 (en) 2022-02-17
CA2917018A1 (en) 2014-12-04
SG11201510757PA (en) 2016-01-28
KR20210111878A (en) 2021-09-13
CN111763690A (en) 2020-10-13
CN105408486B (en) 2020-07-14
KR20210037001A (en) 2021-04-05
AU2020200041A1 (en) 2020-02-06
CN111411125A (en) 2020-07-14
AU2022204246A1 (en) 2022-07-21

Similar Documents

Publication Publication Date Title
HK1217730A1 (en) Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer raav3
IL273090B (en) Methods and compositions for the treatment of cancer
HK1226301A1 (en) Modified therapeutic agents and compositions thereof
IL286759A (en) Therapeutic methods and compositions
SG10201801433XA (en) Composition and vaccine for treating prostate cancer
EP2943565A4 (en) Compositions and methods for delivery of immune cells to treat un-resectable or non-resected tumor cells and tumor relapse
HK1211832A1 (en) Novel compositions and uses of anti-hypertension agents for cancer therapy
IL238177A0 (en) Methods and compositions for the treatment of cancer
EP3193914A4 (en) Organ protection in psma-targeted radionuclide therapy of prostate cancer
EP2968474A4 (en) Skin compositions and uses
EP2884984A4 (en) Therapeutic compositions and methods
EP2961419A4 (en) Phf20 and jmjd3 compositions and methods of use in cancer immunotherapy
SG11201508167PA (en) Therapeutic compositions and uses thereof
EP3049536A4 (en) Methods and compositions relating to cancer therapy with dna damaging agents
EP3004396A4 (en) Methods and compositions for the treatment of cancer
ZA201604370B (en) Prostate cancer gene profiles and methods of using the same
HK1252038A1 (en) Gene expression profiles and uses thereof in breast cancer
EP3044590A4 (en) Methods and compositions for tumor vasculature imaging and targeted therapy
EP2958591A4 (en) Methods and compositions relating to the treatment of cancer
EP3010504A4 (en) Methods and compositions to treat cancer
EP2852370A4 (en) Radiance compositions and methods of use
EP2928456A4 (en) Methods and compositions for treatment of cancer
EP2895186A4 (en) Therapeutic compositions and related methods
EP2744786A4 (en) Substituted 2-imidazolidinones and 2-imidazolones and their use in the treatment of cancer
HK1207328A1 (en) Application of icaritin in preparing medicament for treating primary liver cancer