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Taysha Gene Therapies Announces Fast Track Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome
Fast Track Designation (FTD) is designed to accelerate the development and expedite the review of therapies with potential to address unmet medical needs for
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"Fast Track Designation (FTD) is designed to accelerate the development and expedite the review of therapies with potential to address unmet medical needs for a serious or life-threatening condition TSHA-102 has also received Orphan Drug and Rare Pediatric Disease designations from the United States (U.S.) Food and Drug Administration (FDA) and has been granted Orphan Drug designation from the European Commission DALLAS, Aug. 24, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today 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 (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. FTD is designed to help treatments reach patients faster by facilitating the development and expediting the review of therapies with potential to address unmet medical needs for a serious or life-threatening condition. Benefits of FTD to programs include early and frequent interactions with the FDA during the clinical development process and, if relevant criteria are met, the FDA may also review portions of a marketing application before the sponsor submits the complete application. “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.” Rumana Haque-Ahmed, Senior Vice President, Regulatory Affairs of Taysha, added, “Rett syndrome is a devast...