Title:
AAV VARIANTS WITH ENHANCED TROPISM
Document Type and Number:
WIPO Patent Application WO/2020/028751
Kind Code:
A3
Abstract:
The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.
Inventors:
SAH DINAH WEN-YEE (US)
HOU JINZHAO (US)
ADACHI KEI (US)
WANG WEI (US)
CHEN QINGMIN (US)
CARROLL JENNA (US)
REN AMY (US)
REN XIAO-QIN (US)
DEVERMAN BENJAMIN E (US)
GRADINARU VIVIANA (US)
HUANG QIN (US)
HOU JINZHAO (US)
ADACHI KEI (US)
WANG WEI (US)
CHEN QINGMIN (US)
CARROLL JENNA (US)
REN AMY (US)
REN XIAO-QIN (US)
DEVERMAN BENJAMIN E (US)
GRADINARU VIVIANA (US)
HUANG QIN (US)
Application Number:
PCT/US2019/044796
Publication Date:
March 05, 2020
Filing Date:
August 02, 2019
Export Citation:
Assignee:
VOYAGER THERAPEUTICS INC (US)
CALIFORNIA INST OF TECHN (US)
CALIFORNIA INST OF TECHN (US)
International Classes:
C07K14/005; C12N15/86
Domestic Patent References:
| WO2017100671A1 | 2017-06-15 | |||
| WO2015038958A1 | 2015-03-19 | |||
| WO2016054557A1 | 2016-04-07 |
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), New York, pages 204 - 209, XP055328659, ISSN: 1087-0156, DOI: 10.1038/nbt.3440
DATABASE Geneseq [online] 9 February 2017 (2017-02-09), "Methylobacterium sp. protein, SEQ ID 3938.", XP002796070, retrieved from EBI accession no. GSP:BDK64980 Database accession no. BDK64980
JAE-HYUNG JANG ET AL: "An Evolved Adeno-associated Viral Variant Enhances Gene Delivery and Gene Targeting in Neural Stem Cells", MOLECULAR THERAPY, vol. 19, no. 4, 11 January 2011 (2011-01-11), pages 667 - 675, XP055132115, ISSN: 1525-0016, DOI: 10.1038/mt.2010.287
JAMES T KOERBER ET AL: "Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery", MOLECULAR THERAPY, vol. 17, no. 12, 1 December 2009 (2009-12-01), pages 2088 - 2095, XP055468132, ISSN: 1525-0016, DOI: 10.1038/mt.2009.184
CASEY A MAGUIRE ET AL: "Directed evolution of adeno-associated virus for glioma cell transduction", JOURNAL OF NEURO-ONCOLOGY, KLUWER ACADEMIC PUBLISHERS, BO, vol. 96, no. 3, 19 July 2009 (2009-07-19), pages 337 - 347, XP019773413, ISSN: 1573-7373
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), New York, pages 204 - 209, XP055328659, ISSN: 1087-0156, DOI: 10.1038/nbt.3440
DATABASE Geneseq [online] 9 February 2017 (2017-02-09), "Methylobacterium sp. protein, SEQ ID 3938.", XP002796070, retrieved from EBI accession no. GSP:BDK64980 Database accession no. BDK64980
JAE-HYUNG JANG ET AL: "An Evolved Adeno-associated Viral Variant Enhances Gene Delivery and Gene Targeting in Neural Stem Cells", MOLECULAR THERAPY, vol. 19, no. 4, 11 January 2011 (2011-01-11), pages 667 - 675, XP055132115, ISSN: 1525-0016, DOI: 10.1038/mt.2010.287
JAMES T KOERBER ET AL: "Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery", MOLECULAR THERAPY, vol. 17, no. 12, 1 December 2009 (2009-12-01), pages 2088 - 2095, XP055468132, ISSN: 1525-0016, DOI: 10.1038/mt.2009.184
CASEY A MAGUIRE ET AL: "Directed evolution of adeno-associated virus for glioma cell transduction", JOURNAL OF NEURO-ONCOLOGY, KLUWER ACADEMIC PUBLISHERS, BO, vol. 96, no. 3, 19 July 2009 (2009-07-19), pages 337 - 347, XP019773413, ISSN: 1573-7373
Attorney, Agent or Firm:
WARD, Donna T. et al. (US)
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