Taysha Gene Therapies Presents New Preclinical In-Vitro Data on TSHA-102

Taysha Gene Therapies, Inc., 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), 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.