TSHA-105

TSHA-105 is an investigational gene therapy developed for the treatment of SLC13A5 deficiency, a rare autosomal recessive epileptic encephalopathy. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a codon-optimized human SLC13A5 gene, aiming to restore function of the sodium-dependent citrate transporter (NaCT) that is deficient in affected patients. SLC13A5 deficiency leads to early-onset seizures and developmental delays due to impaired neuronal uptake of citrate and other metabolites critical for brain energy metabolism. Preclinical studies have shown that TSHA-105 can normalize plasma citrate levels, reduce seizure activity, and improve survival in knockout mouse models. There are currently no approved disease-modifying treatments for this condition.

Other names

SLC13A5 gene therapySLC-13A5 gene therapySLC 13A5 gene therapy