TSHA-113

TSHA-113 is an investigational gene therapy designed for the treatment of tauopathies, a group of neurodegenerative diseases characterized by pathological aggregation of tau protein such as frontotemporal dementia, progressive supranuclear palsy, and others[1][3][5][7][9][10][11]. The therapy uses an AAV9 viral capsid to deliver a tau-specific microRNA (miRNA) shuttle via cerebrospinal fluid (CSF) injection. This results in long-term, targeted gene silencing and a sustained reduction of tau protein in neuronal cells[3][5][9][11]. The core mechanisms include microtubule-associated protein tau inhibition, gene expression modulation, and RNA interference[1][3][5][11]. TSHA-113 was initially developed by The University of Texas Southwestern Medical Center and further advanced by Taysha Gene Therapies[1][11][12]. Development for tauopathies has been discontinued prior to clinical trials, with the highest phase being preclinical[1].

Brand names

TSHA-113TSHA113TSHA 113

Other names

TSHA-113TSHA113TSHA 113