TSHA-103

TSHA-103 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy designed for the treatment of SLC6A1 haploinsufficiency disorder, a rare monogenic neurodevelopmental disorder characterized by epilepsy, developmental delay, and behavioral abnormalities. This therapy delivers a codon-optimized version of the human SLC6A1 gene, aiming to restore normal function by gene replacement in central nervous system tissues. Preclinical studies have shown TSHA-103 improves nesting and EEG activity and reduces spike train activity in SLC6A1 knockout and heterozygous mouse models. The drug was originally developed by The University of Texas Southwestern Medical Center and further developed by Taysha Gene Therapies[1][2][7][11][12].

Brand names

TSHA-103TSHA103TSHA 103

Other names

TSHA-103TSHA103TSHA 103