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Title:
RECOMBINANT ADENO-ASSOCIATED VIRUSES FOR CNS TROPIC DELIVERY
Document Type and Number:
WIPO Patent Application WO/2023/060272
Kind Code:
A3
Abstract:
The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to include amino acid sequences and/or amino acid substitutions that confer and/or enhance desired properties, particularly increased transduction in CNS upon intraparenchymal administration relative to a rAAV having a reference capsid.

Inventors:
DANOS OLIVIER (US)
YOST SAMANTHA (US)
MERCER ANDREW (US)
LIU YE (US)
BRUDER JOSEPH (US)
KARUMUTHIL-MELETHIL SUBHA (US)
FIRNBERG ELAD (US)
QIAN RANDOLPH (US)
TEPE APRIL (US)
EGLEY JENNIFER (US)
SMITH JARED (US)
HOLLIDGE BRADLEY (US)
Application Number:
PCT/US2022/077821
Publication Date:
June 15, 2023
Filing Date:
October 07, 2022
Export Citation:
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Assignee:
REGENXBIO INC (US)
International Classes:
C07K14/005; A61P21/00; A61P25/00; C12N15/86
Domestic Patent References:
WO2018152333A12018-08-23
WO2020206189A12020-10-08
WO2018119330A22018-06-28
WO2020160508A12020-08-06
WO2012109570A12012-08-16
WO2013170078A12013-11-14
WO2022076711A22022-04-14
WO2022076750A22022-04-14
WO2022235702A12022-11-10
Other References:
GILES A ET AL: "Mapping an Adeno-associated Virus 9-specific Neutralizing Epitope tp Develop Next-Generation Gene Delivery Vectors", JOURNAL OF VIROLOGY, vol. 92, no. 20, 8 August 2018 (2018-08-08), US, pages 1 - 20, XP055699487, ISSN: 0022-538X, DOI: 10.1128/JVI.01011-18
Attorney, Agent or Firm:
BRIVANLOU, Margaret et al. (US)
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