TSHA-111-LAFORIN

TSHA-111-LAFORIN is an investigational gene therapy being developed for the treatment of Lafora disease, a fatal childhood-onset progressive myoclonus epilepsy caused by mutations in genes responsible for glycogen metabolism. The therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver a microRNA (miRNA) designed to mediate the knockdown of GYS1, the gene encoding glycogen synthase 1, with the aim of reducing toxic, insoluble glycogen accumulation (Lafora bodies) in the brain, thereby addressing the disease's root cause. Preclinical studies have shown that TSHA-111-LAFORIN effectively reduces GYS1 expression, lowers insoluble glycogen, and decreases Lafora body formation in relevant animal models[1][4][6].

Brand names

TSHA-111-LAFORINTSHA111-LAFORINTSHA 111-LAFORIN