Title:
IMMUNO-EVASIVE VECTORS AND USE FOR GENE THERAPY
Document Type and Number:
WIPO Patent Application WO/2019/140311
Kind Code:
A1
Abstract:
Provided is an enveloped viral vector comprising a viral particle surrounded by an envelope, wherein the viral particle comprises a heterologous transgene, and the envelope comprises a lipid bilayer and one or more immunosuppressive molecules, and methods for preparing and using same.
Inventors:
WINSLOW GENINE (US)
Application Number:
PCT/US2019/013361
Publication Date:
July 18, 2019
Filing Date:
January 11, 2019
Export Citation:
Assignee:
CHAMELEON BIOSCIENCES INC (US)
International Classes:
A61K35/76; C12N15/86; C07K14/705
Domestic Patent References:
| WO2006007539A1 | 2006-01-19 | |||
| WO2017136764A1 | 2017-08-10 | |||
| WO2011106376A2 | 2011-09-01 |
Foreign References:
| US20160067228A1 | 2016-03-10 | |||
| US20130020559A1 | 2013-01-24 |
Other References:
AMINE MELIANI ET AL: "Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors", BLOOD ADVANCES, vol. 1, no. 23, 24 October 2017 (2017-10-24), pages 2019 - 2031, XP055576464, ISSN: 2473-9529, DOI: 10.1182/bloodadvances.2017010181
SAHIL ADRIOUCH ET AL: "Improved Immunological Tolerance Following Combination Therapy with CTLA-4/Ig and AAV-Mediated PD-L1/2 Muscle Gene Transfer", FRONTIERS IN MICROBIOLOGY, vol. 2, 1 January 2011 (2011-01-01), XP055576248, DOI: 10.3389/fmicb.2011.00199
SELOT RUCHITA S ET AL: "Developing Immunologically Inert Adeno-Associated Virus (AAV) Vectors for Gene Therapy: Possibilities and Limitations", CURRENT PHARMACEUTICAL BIOTECHNOLOGY, vol. 14, no. 12, 2013, pages 1072 - 1082, XP009512398
CASEY A MAGUIRE ET AL: "Microvesicle-associated AAV Vector as a Novel Gene Delivery System", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 20, no. 5, 1 May 2012 (2012-05-01), US, pages 960 - 971, XP055405355, ISSN: 1525-0016, DOI: 10.1038/mt.2011.303
DIESENDRUCK YAEL ET AL: "Novel immune check point inhibiting antibodies in cancer therapy-Opportunities and challenges", DRUG RESISTANCE UPDATES, CHURCHILL LIVINGSTONE, EDINBURGH, GB, vol. 30, 4 February 2017 (2017-02-04), pages 39 - 47, XP029960699, ISSN: 1368-7646, DOI: 10.1016/J.DRUP.2017.02.001
JOSE LUIS PEREZ-GRACIA ET AL: "Orchestrating immune check-point blockade for cancer immunotherapy in combinations", CURRENT OPINION IN IMMUNOLOGY, vol. 27, 1 April 2014 (2014-04-01), pages 89 - 97, XP055200525, ISSN: 0952-7915, DOI: 10.1016/j.coi.2014.01.002
MELIANI ET AL., BLOOD ADVANCES, vol. 1, no. 23, 2017, pages 2019 - 31
NATHWANI ET AL., N ENGL J MED, vol. 365, 2011, pages 2357 - 65
SIMONELLI ET AL., MOLECULAR THERAPY, vol. 18, no. 3, 2010, pages 643 - 650
CHILDERS ET AL., SCI TRANSL MED, vol. 6, 2014, pages 220ral0
MINGOZZI ET AL., BLOOD, vol. 122, no. 1, 2013, pages 23 - 36
CHERMULE ET AL., GENE THERAPY, vol. 6, 1999, pages 1574 - 1583
MASAT ET AL., DISCOV MED, vol. 15, no. 85, 2013, pages 379 - 389
NATHWANI ET AL., N ENGL J MED, vol. 371, 2014, pages 1994 - 2004
CHIERMULE ET AL., GENE THERAPY, vol. 6, 1999, pages 1574 - 1583
ASOKAN ET AL., MOLECULAR THERAPY, vol. 20, no. 4, 2012, pages 699 - 708
GYORGY ET AL., BIOMATERIALS, vol. 35, no. 26, 2014, pages 7598 - 7609
HUDRY ET AL., GENE THER, vol. 23, no. 4, April 2016 (2016-04-01), pages 380 - 92
ADRIOUCH ET AL., FRONT MICROBIOL, vol. 2, 2011, pages 199
SAMBROOK ET AL.: "Molecular Cloning: A Laboratory Manual", 2012, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", 2003
"Methods in Enzymology", ACADEMIC PRESS, INC.
"PCR 2: A Practical Approach", 1995
"Antibodies, A Laboratory Manual", 1988
R.I. FRESHNEY: "Culture of Animal Cells: A Manual of Basic Technique and Specialized Applications", 2010, J. WILEY AND SONS
"Oligonucleotide Synthesis", 1984
"Methods in Molecular Biology", HUMANA PRESS
"Cell Biology: A Laboratory Notebook", 1998, ACADEMIC PRESS
J.P. MATHER; P.E. ROBERTS: "Introduction to Cell and Tissue Culture", 1998, PLENUM PRESS
"Cell and Tissue Culture: Laboratory Procedures", August 1993, J. WILEY AND SONS
"Handbook of Experimental Immunology", 1996
"Gene Transfer Vectors for Mammalian Cells", 1987
"PCR: The Polymerase Chain Reaction", 1994
"Current Protocols in Immunology", 1991
"Short Protocols in Molecular Biology", 2002, J. WILEY AND SONS
C.A. JANEWAY ET AL., IMMUNOBIOLOGY, 2004
P. FINCH, ANTIBODIES, 1997
"Antibodies: A Practical Approach", 1988, IRL PRESS
"Monoclonal Antibodies: A Practical Approach", 2000, OXFORD UNIVERSITY PRESS
E. HARLOW; D. LANE: "Using Antibodies: A Laboratory Manual", 1999, COLD SPRING HARBOR LABORATORY PRESS
"The Antibodies", 1995, HARWOOD ACADEMIC PUBLISHERS
"Cancer: Principles and Practice of Oncology", 2011, J.B. LIPPINCOTT COMPANY
"UniProtKB", Database accession no. Q2VF45
LIND ET AL., EUR J BIOCHEM., vol. 232, no. 1, 15 August 1995 (1995-08-15), pages 19 - 27
"UniProtKB", Database accession no. P00740
MONAHAN ET AL., HUM GENE THER., vol. 26, no. 2, 2015, pages 69 - 81
"NCBI", Database accession no. NP_005205.2
"NCBI", Database accession no. NP_054862.1
SHILLER ET AL., MOL THER METHODS CLIN DEV, vol. 9, 2018, pages 278 - 287
THROM ET AL., BLOOD, vol. 113, no. 21, 2009, pages 5104 - 5110
BOOTH ET AL., GENE THER, vol. 11, no. 10, 2004, pages 829 - 837
AYUSO ET AL., HUM GENE THER, vol. 25, 2014, pages 977 - 987
XIAO ET AL., J VIROL, vol. 72, no. 3, 1998, pages 2224 - 2232
RYU ET AL., MOL THER, vol. 21.B, 2013
RYU ET AL., MOL THER 2013, vol. 21.B, 2013
MELIANI ET AL., HUM GENE THER METHODS, vol. 26, 2015, pages 45 - 53
"REMINGTON'S PHARMACEUTICAL SCIENCES", 1991, MACK PUB. CO.
MELAINI ET AL., BLOOD ADVANCES, vol. 1, no. 23, 2017, pages 2019 - 31
SAHIL ADRIOUCH ET AL: "Improved Immunological Tolerance Following Combination Therapy with CTLA-4/Ig and AAV-Mediated PD-L1/2 Muscle Gene Transfer", FRONTIERS IN MICROBIOLOGY, vol. 2, 1 January 2011 (2011-01-01), XP055576248, DOI: 10.3389/fmicb.2011.00199
SELOT RUCHITA S ET AL: "Developing Immunologically Inert Adeno-Associated Virus (AAV) Vectors for Gene Therapy: Possibilities and Limitations", CURRENT PHARMACEUTICAL BIOTECHNOLOGY, vol. 14, no. 12, 2013, pages 1072 - 1082, XP009512398
CASEY A MAGUIRE ET AL: "Microvesicle-associated AAV Vector as a Novel Gene Delivery System", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 20, no. 5, 1 May 2012 (2012-05-01), US, pages 960 - 971, XP055405355, ISSN: 1525-0016, DOI: 10.1038/mt.2011.303
DIESENDRUCK YAEL ET AL: "Novel immune check point inhibiting antibodies in cancer therapy-Opportunities and challenges", DRUG RESISTANCE UPDATES, CHURCHILL LIVINGSTONE, EDINBURGH, GB, vol. 30, 4 February 2017 (2017-02-04), pages 39 - 47, XP029960699, ISSN: 1368-7646, DOI: 10.1016/J.DRUP.2017.02.001
JOSE LUIS PEREZ-GRACIA ET AL: "Orchestrating immune check-point blockade for cancer immunotherapy in combinations", CURRENT OPINION IN IMMUNOLOGY, vol. 27, 1 April 2014 (2014-04-01), pages 89 - 97, XP055200525, ISSN: 0952-7915, DOI: 10.1016/j.coi.2014.01.002
MELIANI ET AL., BLOOD ADVANCES, vol. 1, no. 23, 2017, pages 2019 - 31
NATHWANI ET AL., N ENGL J MED, vol. 365, 2011, pages 2357 - 65
SIMONELLI ET AL., MOLECULAR THERAPY, vol. 18, no. 3, 2010, pages 643 - 650
CHILDERS ET AL., SCI TRANSL MED, vol. 6, 2014, pages 220ral0
MINGOZZI ET AL., BLOOD, vol. 122, no. 1, 2013, pages 23 - 36
CHERMULE ET AL., GENE THERAPY, vol. 6, 1999, pages 1574 - 1583
MASAT ET AL., DISCOV MED, vol. 15, no. 85, 2013, pages 379 - 389
NATHWANI ET AL., N ENGL J MED, vol. 371, 2014, pages 1994 - 2004
CHIERMULE ET AL., GENE THERAPY, vol. 6, 1999, pages 1574 - 1583
ASOKAN ET AL., MOLECULAR THERAPY, vol. 20, no. 4, 2012, pages 699 - 708
GYORGY ET AL., BIOMATERIALS, vol. 35, no. 26, 2014, pages 7598 - 7609
HUDRY ET AL., GENE THER, vol. 23, no. 4, April 2016 (2016-04-01), pages 380 - 92
ADRIOUCH ET AL., FRONT MICROBIOL, vol. 2, 2011, pages 199
SAMBROOK ET AL.: "Molecular Cloning: A Laboratory Manual", 2012, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", 2003
"Methods in Enzymology", ACADEMIC PRESS, INC.
"PCR 2: A Practical Approach", 1995
"Antibodies, A Laboratory Manual", 1988
R.I. FRESHNEY: "Culture of Animal Cells: A Manual of Basic Technique and Specialized Applications", 2010, J. WILEY AND SONS
"Oligonucleotide Synthesis", 1984
"Methods in Molecular Biology", HUMANA PRESS
"Cell Biology: A Laboratory Notebook", 1998, ACADEMIC PRESS
J.P. MATHER; P.E. ROBERTS: "Introduction to Cell and Tissue Culture", 1998, PLENUM PRESS
"Cell and Tissue Culture: Laboratory Procedures", August 1993, J. WILEY AND SONS
"Handbook of Experimental Immunology", 1996
"Gene Transfer Vectors for Mammalian Cells", 1987
"PCR: The Polymerase Chain Reaction", 1994
"Current Protocols in Immunology", 1991
"Short Protocols in Molecular Biology", 2002, J. WILEY AND SONS
C.A. JANEWAY ET AL., IMMUNOBIOLOGY, 2004
P. FINCH, ANTIBODIES, 1997
"Antibodies: A Practical Approach", 1988, IRL PRESS
"Monoclonal Antibodies: A Practical Approach", 2000, OXFORD UNIVERSITY PRESS
E. HARLOW; D. LANE: "Using Antibodies: A Laboratory Manual", 1999, COLD SPRING HARBOR LABORATORY PRESS
"The Antibodies", 1995, HARWOOD ACADEMIC PUBLISHERS
"Cancer: Principles and Practice of Oncology", 2011, J.B. LIPPINCOTT COMPANY
"UniProtKB", Database accession no. Q2VF45
LIND ET AL., EUR J BIOCHEM., vol. 232, no. 1, 15 August 1995 (1995-08-15), pages 19 - 27
"UniProtKB", Database accession no. P00740
MONAHAN ET AL., HUM GENE THER., vol. 26, no. 2, 2015, pages 69 - 81
"NCBI", Database accession no. NP_005205.2
"NCBI", Database accession no. NP_054862.1
SHILLER ET AL., MOL THER METHODS CLIN DEV, vol. 9, 2018, pages 278 - 287
THROM ET AL., BLOOD, vol. 113, no. 21, 2009, pages 5104 - 5110
BOOTH ET AL., GENE THER, vol. 11, no. 10, 2004, pages 829 - 837
AYUSO ET AL., HUM GENE THER, vol. 25, 2014, pages 977 - 987
XIAO ET AL., J VIROL, vol. 72, no. 3, 1998, pages 2224 - 2232
RYU ET AL., MOL THER, vol. 21.B, 2013
RYU ET AL., MOL THER 2013, vol. 21.B, 2013
MELIANI ET AL., HUM GENE THER METHODS, vol. 26, 2015, pages 45 - 53
"REMINGTON'S PHARMACEUTICAL SCIENCES", 1991, MACK PUB. CO.
MELAINI ET AL., BLOOD ADVANCES, vol. 1, no. 23, 2017, pages 2019 - 31
Attorney, Agent or Firm:
DONAHUE, Brian A. et al. (US)
Download PDF: