TSHA

Taysha Gene Therapies Inc is a clinical-stage biotechnology company, which is focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. The Company’s lead clinical program, TSHA-102, is in development for the treatment of Rett syndrome, a rare neurodevelopmental disorder. The Company is evaluating TSHA-102 in the REVEALPhase I/II adolescent and adult clinical trial, which is a first-in-human, open-label, randomized, dose escalation and dose-expansion, multicenter study evaluating the safety and preliminary efficacy of TSHA-102 in female patients aged 12-years and older with Rett syndrome. It has acquired a worldwide right to a clinical-stage, intrathecally dosed AAV9 gene therapy program, TSHA-120, for the treatment of giant axonal neuropathy (GAN). It received orphan drug designation from the European Commission for TSHA-120 for the treatment of GAN.