RECOMBINANT AAV VECTORS FOR TREATING PROTEINOPATHIES IN CENTRAL NERVOUS SYSTEM
Application
US20260091134A1
Kind: A1
Apr 02, 2026
Assignee
SHANGHAI VITALGEN BIOPHARMA CO., LTD.
Inventors
Yezheng TAO, Shin-Shay TIAN, Xiaoping ZHAO
Abstract
The present disclosure generally relates to a recombinant adeno-associated viral (rAAV) vector comprising one or two of (a) a nucleotide sequence encoding Progranulin (PGRN), and (b) a nucleotide sequence encoding Stathmin-2 (STMN2), for treating proteinopathies in the central nervous system. Also disclosed are codon-optimized coding sequences of PGRN and/or STMN2, and expression cassettes, vectors, and viral particles comprising the same.
CPC Classifications
A61K 48/005
A61P 25/28
C07K 14/005
C07K 14/475
C12N 15/86
A61K 38/00
C07K 2319/00
C12N 2750/14122
C12N 2750/14143
C12N 2750/14145
C12N 2800/22
Filing Date
2025-11-26
Application No.
19401460