GENE THERAPY FOR CEROID LIPOFUSCINOSES
Application
US20260078406A1
Kind: A1
Mar 19, 2026
Inventors
James M. Wilson, Nathan Katz, Christian Hinderer, Juliette Hordeaux
Abstract
Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR.
CPC Classifications
C12N 15/86
A61K 48/0075
C12N 9/485
A61K 9/0019
C12N 2710/16143
C12N 2750/14143
C12Y 304/14009
Filing Date
2025-08-26
Application No.
19309870