We are a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations. (AAV stands for adeno-associated virus.) Our most advanced product candidates include TSHA-101, which is being developed to treat genetic disorders that include Tay-Sachs disease, a rare progressive disease that destroys nerve cells in the brain and spinal cord, and TSHA-102, which is being developed for the treatment of Rett syndrome, one of the most common genetic causes of severe intellectual disability, characterized by rapid developmental regression.

We were founded in partnership with The University of Texas Southwestern Medical Center, or UT Southwestern, to develop and commercialize transformative gene therapy treatments. Together with UT Southwestern, we are advancing a deep and sustainable product portfolio of 18 gene therapy product candidates, with exclusive options to acquire four additional development programs at no cost.