TSHA
Taysha Gene Therapies is a clinical-stage biotechnology company developing AAV gene therapies for severe monogenic diseases of the central nervous system, with TSHA-102 as its lead program in Phase 1/2 trials for Rett syndrome, a rare neurodevelopmental disorder caused by MECP2 mutations. TSHA-102 is administered as a single lumbar intrathecal injection and has demonstrated in early clinical data that patients gained or regained developmental milestones in communication, fine motor, and gross motor function with no treatment-related serious adverse events reported. The company is advancing TSHA-102 toward potential regulatory approval through the REVEAL pivotal trial in patients aged 6 to under 22 years and the ASPIRE safety trial in younger children.