TSHA-112

TSHA-112 is an investigational gene therapy employing an adeno-associated virus serotype 9 (AAV9) viral vector designed for intrathecal delivery. It contains a microRNA (miRNA) construct that enables knockdown of the glycogen synthase 1 (GYS1) gene in the central nervous system. The therapeutic goal is to treat adult polyglucosan body disease (APBD), a rare neurodegenerative glycogen storage disorder, by reducing pathological accumulation of polyglucosan bodies in neural tissues. TSHA-112 was co-developed by Taysha Gene Therapies and the University of Texas Southwestern Medical Center. Preclinical studies have shown reduction in neuroinflammatory markers and efficacy in relevant mouse models, and the program has advanced into IND-enabling studies but has not entered active clinical trials. Rights to TSHA-112 have since been transferred back to the originating institution[3][7][8][12][14].

Brand names

TSHA-112TSHA112TSHA 112

Other names

TSHA-112TSHA112TSHA 112