WO2014193716A3 - 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 Download PDF

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Publication number
WO2014193716A3
WO2014193716A3 PCT/US2014/039015 US2014039015W WO2014193716A3 WO 2014193716 A3 WO2014193716 A3 WO 2014193716A3 US 2014039015 W US2014039015 W US 2014039015W WO 2014193716 A3 WO2014193716 A3 WO 2014193716A3
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WO
WIPO (PCT)
Prior art keywords
disclosed
capsid
gene therapy
modified
compositions
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Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US2014/039015
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French (fr)
Other versions
WO2014193716A2 (en
Inventor
Arun Srivastava
Li Zhong
Sergei Zolotukhin
George V. Aslanidi
Mavis Agbandje-Mckenna
Kim M. VAN VLIET
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Florida
University of Florida Research Foundation Inc
Original Assignee
University of Florida
University of Florida Research Foundation Inc
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
Priority to KR1020217008950A priority Critical patent/KR102298579B1/en
Priority to EP18202680.7A priority patent/EP3492597A3/en
Priority to EP14733803.2A priority patent/EP2999791B1/en
Priority to CA2917018A priority patent/CA2917018A1/en
Priority to JP2016515056A priority patent/JP6628286B2/en
Priority to DK14733803.2T priority patent/DK2999791T3/en
Priority to ES14733803T priority patent/ES2714292T3/en
Priority to LTEP14733803.2T priority patent/LT2999791T/en
Priority to HK16105737.9A priority patent/HK1217730B/en
Priority to US14/891,241 priority patent/US20160333372A1/en
Priority to KR1020157036164A priority patent/KR102234672B1/en
Application filed by University of Florida, University of Florida Research Foundation Inc filed Critical University of Florida
Priority to KR1020227033180A priority patent/KR102649543B1/en
Priority to SG11201510757PA priority patent/SG11201510757PA/en
Priority to CN201480041247.6A priority patent/CN105408486B/en
Priority to KR1020217027678A priority patent/KR20210111878A/en
Priority to AU2014274457A priority patent/AU2014274457B2/en
Publication of WO2014193716A2 publication Critical patent/WO2014193716A2/en
Publication of WO2014193716A3 publication Critical patent/WO2014193716A3/en
Anticipated expiration legal-status Critical
Priority to US15/824,023 priority patent/US10934327B2/en
Priority to AU2020200041A priority patent/AU2020200041B2/en
Priority to US17/132,607 priority patent/US12570699B2/en
Priority to AU2022204246A priority patent/AU2022204246A1/en
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/19—Dendritic cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/24—Antigen-presenting cells [APC]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/34—Antigenic peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4244—Enzymes
    • A61K40/4246—Telomerase or [telomerase reverse transcriptase [TERT]
    • 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
    • A61K48/0008—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 '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
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • 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
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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Abstract

Disclosed are next-generation multi-mutated capsid protein-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using these high transduction efficiency vector constructs in a variety of therapeutic applications including, inter alia, as delivery agents for the treatment or amelioration of one or more diseases or abnormal conditions in an affected mammal using in vivo and/or ex situ viral vector-based gene therapy protocols. Also disclosed are large-scale production methods for the multi-mutated, capsid-modified rAAV expression vectors, viral particles, and infectious virions, as well as use of the disclosed compositions in the manufacture of medicaments for use in a variety of in vitro and/or in vivo therapeutic methodologies.
PCT/US2014/039015 2007-04-09 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer Ceased WO2014193716A2 (en)

Priority Applications (20)

Application Number Priority Date Filing Date Title
AU2014274457A AU2014274457B2 (en) 2013-05-21 2014-05-21 Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
KR1020227033180A KR102649543B1 (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
EP14733803.2A EP2999791B1 (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
CA2917018A CA2917018A1 (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
JP2016515056A JP6628286B2 (en) 2013-05-21 2014-05-21 Capsid-modified rAAV3 vector composition and method for use in gene therapy of human liver cancer
DK14733803.2T DK2999791T3 (en) 2013-05-21 2014-05-21 CAPSID MODIFIED RAAV3 VECTOR COMPOSITIONS AND METHODS FOR USE IN GENTERAPY AGAINST HUMAN CANCER
ES14733803T ES2714292T3 (en) 2007-04-09 2014-05-21 Raav3 vector compositions with modified capsid and methods of use in gene therapy of human liver cancer
LTEP14733803.2T LT2999791T (en) 2007-04-09 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
HK16105737.9A HK1217730B (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
EP18202680.7A EP3492597A3 (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
KR1020157036164A KR102234672B1 (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
KR1020217008950A KR102298579B1 (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
CN201480041247.6A CN105408486B (en) 2013-05-21 2014-05-21 Capsid-modified RAAV3 vector compositions and uses in gene therapy of human liver cancer
US14/891,241 US20160333372A1 (en) 2007-04-09 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
KR1020217027678A KR20210111878A (en) 2013-05-21 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
US15/824,023 US10934327B2 (en) 2007-04-09 2017-11-28 Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer
AU2020200041A AU2020200041B2 (en) 2013-05-21 2020-01-03 Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
US17/132,607 US12570699B2 (en) 2007-04-09 2020-12-23 Capsid-modified, rAAV3 vector compositions and methods of use in gene therapy of human liver cancer
AU2022204246A AU2022204246A1 (en) 2013-05-21 2022-06-17 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer

Applications Claiming Priority (2)

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
US13/899,481 2013-05-21

Related Parent Applications (1)

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

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US14/891,241 A-371-Of-International US20160333372A1 (en) 2007-04-09 2014-05-21 Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
US15/824,023 Continuation US10934327B2 (en) 2007-04-09 2017-11-28 Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer

Publications (2)

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WO2014193716A2 WO2014193716A2 (en) 2014-12-04
WO2014193716A3 true WO2014193716A3 (en) 2015-01-22

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PCT/US2014/039015 Ceased WO2014193716A2 (en) 2007-04-09 2014-05-21 Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer

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EP (2) EP3492597A3 (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)
SG (2) SG11201510757PA (en)
WO (1) WO2014193716A2 (en)

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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
ES2714007T3 (en) 2007-04-09 2019-05-24 Univ Florida Compositions of rAAV vectors that have tyrosine modified capsid proteins and methods for their use
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
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