TSHA-117

TSHA-117 is an investigational adeno-associated virus (AAV)-based gene therapy designed to treat FOXG1 syndrome, a severe neurodevelopmental disorder caused by pathogenic mutations in the FOXG1 gene. The therapy aims to restore FOXG1 function by delivering a functional copy of the FOXG1 gene to affected cells in the central nervous system using an AAV vector, thus acting as a FOXG1 stimulant. TSHA-117 was initially developed by The University of Texas Southwestern Medical Center, with subsequent involvement by Taysha Gene Therapies. It is intended as a gene replacement therapy targeting nervous system diseases, particularly FOXG1 syndrome[1][7][9][12].

Brand names

TSHA-117TSHA117TSHA 117

Other names

TSHA-117TSHA117TSHA 117