Title:
COMPOSITIONS AND METHODS FOR DELIVERY OF AAV
Document Type and Number:
WIPO Patent Application WO/2019/028306
Kind Code:
A3
Abstract:
The invention provides compositions and methods for the preparation, manufacture, formulation and therapeutic use of adeno-associated virus (AAV) particles for the prevention and/or treatment of diseases.
Inventors:
SAH DINAH WEN-YEE (US)
PATZKE HOLGER (US)
HOU JINZHAO (US)
NONNENMACHER MATHIEU E (US)
GOULET MARTIN (US)
CARTER TODD (US)
PATZKE HOLGER (US)
HOU JINZHAO (US)
NONNENMACHER MATHIEU E (US)
GOULET MARTIN (US)
CARTER TODD (US)
Application Number:
PCT/US2018/045088
Publication Date:
March 07, 2019
Filing Date:
August 03, 2018
Export Citation:
Assignee:
VOYAGER THERAPEUTICS INC (US)
International Classes:
C12N7/00; A61K39/12; A61K48/00; C12N15/86
Domestic Patent References:
| WO2017100671A1 | 2017-06-15 | |||
| WO2015038958A1 | 2015-03-19 |
Other References:
KEN Y CHAN ET AL: "Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems", NATURE NEUROSCIENCE, vol. 20, no. 8, 26 June 2017 (2017-06-26), US, pages 1172 - 1179, XP055527909, ISSN: 1097-6256, DOI: 10.1038/nn.4593
BENJAMIN E DEVERMAN ET AL: "Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain", NATURE BIOTECHNOLOGY, vol. 34, no. 2, 1 February 2016 (2016-02-01), pages 204 - 209, XP055328659, ISSN: 1087-0156, DOI: 10.1038/nbt.3440
CATHERINE GÉRARD ET AL: "An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models", MOLECULAR THERAPY - METHODS & CLINICAL DEVELOPMENT, vol. 1, 1 January 2014 (2014-01-01), pages 14044, XP055210120, DOI: 10.1038/mtm.2014.44
KOU JINGHONG ET AL: "Catalytic Immunoglobulin Gene Delivery in a Mouse Model of Alzheimer's Disease: Prophylactic and Therapeutic Applicat", MOLECULAR NEUROBIOLOGY, HUMANA PRESS, US, vol. 51, no. 1, 15 April 2014 (2014-04-15), pages 43 - 56, XP035437376, ISSN: 0893-7648, [retrieved on 20140415], DOI: 10.1007/S12035-014-8691-Z
KEVIN D FOUST ET AL: "Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 21, no. 12, 6 September 2013 (2013-09-06), US, pages 2148 - 2159, XP055358429, ISSN: 1525-0016, DOI: 10.1038/mt.2013.211
MORGANE PERDOMINI ET AL: "Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia", NATURE MEDICINE, vol. 20, no. 5, 6 April 2014 (2014-04-06), pages 542 - 547, XP055210141, ISSN: 1078-8956, DOI: 10.1038/nm.3510
PLEGER SVEN T ET AL: "Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model", SCIENCE TRANSLATIONAL MEDICINE,, vol. 3, no. 92 ra 64, 20 July 2011 (2011-07-20), pages 117 - 126, XP008168700, ISSN: 1946-6242, DOI: 10.1126/SCITRANSLMED.3002097
BENJAMIN E DEVERMAN ET AL: "Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain", NATURE BIOTECHNOLOGY, vol. 34, no. 2, 1 February 2016 (2016-02-01), pages 204 - 209, XP055328659, ISSN: 1087-0156, DOI: 10.1038/nbt.3440
CATHERINE GÉRARD ET AL: "An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models", MOLECULAR THERAPY - METHODS & CLINICAL DEVELOPMENT, vol. 1, 1 January 2014 (2014-01-01), pages 14044, XP055210120, DOI: 10.1038/mtm.2014.44
KOU JINGHONG ET AL: "Catalytic Immunoglobulin Gene Delivery in a Mouse Model of Alzheimer's Disease: Prophylactic and Therapeutic Applicat", MOLECULAR NEUROBIOLOGY, HUMANA PRESS, US, vol. 51, no. 1, 15 April 2014 (2014-04-15), pages 43 - 56, XP035437376, ISSN: 0893-7648, [retrieved on 20140415], DOI: 10.1007/S12035-014-8691-Z
KEVIN D FOUST ET AL: "Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 21, no. 12, 6 September 2013 (2013-09-06), US, pages 2148 - 2159, XP055358429, ISSN: 1525-0016, DOI: 10.1038/mt.2013.211
MORGANE PERDOMINI ET AL: "Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia", NATURE MEDICINE, vol. 20, no. 5, 6 April 2014 (2014-04-06), pages 542 - 547, XP055210141, ISSN: 1078-8956, DOI: 10.1038/nm.3510
PLEGER SVEN T ET AL: "Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model", SCIENCE TRANSLATIONAL MEDICINE,, vol. 3, no. 92 ra 64, 20 July 2011 (2011-07-20), pages 117 - 126, XP008168700, ISSN: 1946-6242, DOI: 10.1126/SCITRANSLMED.3002097
Attorney, Agent or Firm:
WARD, Donna T. (US)
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