Title:
RECOMBINANT ADENO-ASSOCIATED VIRUSES AND USES THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/044725
Kind Code:
A3
Abstract:
The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to include amino acid sequences that confer and/or enhance desired properties. In particular, the invention provides engineered capsid proteins comprising peptide insertions from heterologous proteins inserted within or near variable region IV (VR-IV) of the virus capsid, such that the insertion is surface exposed on the AAV particle. The invention also provides capsid proteins that direct rAAVs to target tissues, in particular, capsid proteins comprising peptides that are inserted into surface-exposed variable regions using a method for replacing a stop codon in the capsid gene with such peptide insert. The invention provides such methods for making the rAAV vectors into vector libraries and selecting the rAAV vectors having peptide inserts that target the rAAV to particular tissues of interest, and thus deliver therapeutics for treating disorders.
Inventors:
FIRNBERG ELAD (US)
MERCER ANDREW (US)
TEPE APRIL R (US)
YOST SAMANTHA (US)
LIU YE (US)
MERCER ANDREW (US)
TEPE APRIL R (US)
YOST SAMANTHA (US)
LIU YE (US)
Application Number:
PCT/US2023/072875
Publication Date:
April 25, 2024
Filing Date:
August 24, 2023
Export Citation:
Assignee:
REGENXBIO INC (US)
International Classes:
C12N15/864; C07K14/015
Domestic Patent References:
| WO2021226267A2 | 2021-11-11 | |||
| WO2020206189A1 | 2020-10-08 | |||
| WO2022076750A2 | 2022-04-14 | |||
| WO2022076711A2 | 2022-04-14 |
Foreign References:
| CA3193685A1 | 2022-04-14 |
Attorney, Agent or Firm:
BRIVANLOU, Margaret et al. (US)
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