TSHA-111-MALIN

TSHA-111-MALIN is an **AAV9-based gene therapy** developed to treat **Lafora disease** (EPM2B). It is delivered via **intrathecal injection** and uses a microRNA (miRNA) vector targeting **GYS1** (glycogen synthase 1) expression in the brain. The reduction of GYS1 leads to decreased insoluble glycogen and Lafora body formation in relevant mouse models. TSHA-111-MALIN specifically utilizes a malin model to knock down GYS1, aiming to address the pathophysiology of Lafora disease, a rare and fatal neurodegenerative disorder. This candidate was advanced to preclinical development but its **global highest R&D status is discontinued**[1][2][3][4][7][9].

Brand names

TSHA-111-MALINTSHA111-MALINTSHA 111-MALIN

Other names

AAV9-based miRNA therapy containing malinAAV-9-based miRNA therapy containing malinAAV 9-based miRNA therapy containing malin