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Taysha Gene Therapies Presents New Preclinical In-vitro Data on TSHA-102 in Rett Syndrome Supporting miRARE Regulation of MECP2 Expression at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress
In vitro data demonstrated the miRARE control element downregulates MECP2 transgene and protein expression in response to cellular levels of MeCP2 in cell
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"In vitro data demonstrated the miRARE control element downregulates MECP2 transgene and protein expression in response to cellular levels of MeCP2 in cell culture models Data recapitulate in vivo findings in neonatal mice demonstrating TSHA-102 regulated MeCP2 expression in deficient CNS cells and avoided toxic overexpression in cells already expressing MeCP2 Available clinical data from the two adult patients dosed with TSHA-102 in the first cohort (low dose) to be reported in mid-November; dosing of first pediatric Rett syndrome patient expected in the first quarter of 2024 DALLAS, Oct. 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 new preclinical in vitro data on TSHA-102 in Rett syndrome as part of a poster presentation at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress. TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a 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. These data demonstrate the function of the miRARE-RHD1pA regulatory element and its impact on MECP2 transgene and protein expression in human and mouse cell lines, providing further support for the regulatory control of miRARE. “Appropriate control of MECP2 transgene expression based on cellular levels of MeCP2 is fundamental to the development of a safe and effective gene therapy for Rett syndrome, given the mosaic pattern of MECP2 silencing in females with Rett syndrome,” said Sukumar Nagendran, M.D., President, and Head of R&D of Taysha. “These new in vitro data recapitulating our in vivo findings in neonatal mice further our mechanistic understanding of how the miRARE technology controls post-transcriptional MECP2 expression and reinforce the potential of TSHA-102 to address the root cause of Rett syndrome. We look forward to reporting available clinical data from the two adult patients dosed with TSHA-102 in the low-dose cohort of the REVEAL Phase 1/2 adult trial in mid-November and expect to dose the first pediatric patient wi...