Taysha Gene Therapies is a clinical-stage biotechnology company developing AAV-based gene therapies for severe monogenic diseases of the central nervous system, with its lead program targeting Rett syndrome, a rare neurodevelopmental disorder caused by genetic mutations with no approved disease-modifying treatments. The company's core therapy is designed as a one-time intrathecal treatment that delivers a functional gene directly to the cerebrospinal fluid and incorporates proprietary technology to maintain safe expression levels. Taysha operates pre-revenue and generates value through regulatory milestones and potential future sales-based royalties under its intellectual property licensing agreements, with plans to commercialize following regulatory approval.