Taysha Gene Therapies announces FTD granted by FDA for TSHA-102

Taysha Gene Therapies announced the U.S. FDA has granted Fast Track Designation, FTD, to TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. “We are pleased to receive FTD from the FDA, which underscores the significant unmet medical need in patients with Rett syndrome and the potential of TSHA-102 to serve as a meaningful treatment option,” said Sukumar Nagendran, M.D., President and Head of R&D of Taysha. “Initial data from the first adult patient in Canada with severe disease dosed with TSHA-102 is encouraging, and we expect to dose the second patient in our ongoing REVEAL Phase 1/2 adult trial in the current quarter. We look forward to expanding the clinical evaluation to earlier stages of disease progression following recent FDA clearance to initiate clinical development of TSHA-102 in pediatric patients in the United States.”