TSHA-111

TSHA-111 refers to a preclinical gene therapy program for Lafora disease. It appears in two versions, TSHA-111-LAFORIN and TSHA-111-MALIN, each using an adeno-associated virus serotype 9 (AAV9) vector to deliver miRNA sequences designed for knockdown of the GYS1 gene, which encodes glycogen synthase 1. Excess glycogen accumulation in neurons is the hallmark of Lafora disease. The therapy aims to produce therapeutic effects by reducing GYS1 expression, resulting in decreased accumulation of pathological glycogen. Both TSHA-111-LAFORIN and TSHA-111-MALIN have been evaluated in preclinical studies showing effective knockdown of GYS1 and insoluble glycogen. The therapy is delivered intrathecally for targeting the central nervous system. The main indication is Lafora disease, a rare and severe progressive myoclonic epilepsy. The gene therapy platform was originated at The University of Texas Southwestern Medical Center and developed by Taysha Gene Therapies.

Other names

TSHA-111-LAFORINTSHA111-LAFORINTSHA 111-LAFORIN