Taysha Gene Therapies Inc

Taysha Gene Therapies is a clinical-stage biotechnology company focused on developing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program, TSHA-102, is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in development for Rett syndrome, a rare neurodevelopmental disorder caused by mutations in the MECP2 gene affecting an estimated 15,000 to 20,000 patients in the U.S., EU, and U.K. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to regulate MECP2 expression on a cell-by-cell basis without risk of overexpression. The therapy is designed as a one-time intrathecal administration and has received multiple regulatory designations including Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, and Orphan Drug designations from the FDA. The company has a manufacturing partnership with Catalent for commercial supply and is advancing toward a potential BLA submission. Taysha trades on Nasdaq under the ticker TSHA. Updated Aug 11, 2026