There’s big news associated with an announcement from Taysha Gene Therapies. Please see the full announcement at the link here and some selected excerpts that I’ve highlighted after the link.
Taysha Gene Therapies, Inc….today announced the initiation of clinical development of TSHA-102 for the treatment of Rett syndrome under a recently approved Clinical Trial Application (CTA) by Health Canada….
…“Treating Rett syndrome by gene replacement therapy requires an approach that can safely regulate transgene expression in a genotypic manner on a cell-by-cell basis without causing deleterious effects associated with overexpression. TSHA-102’s robust preclinical data package supports and validates the safe and controlled regulation of transgene expression using miRARE, a novel miRNA-responsive target sequence exclusively licensed to Taysha and developed by Drs. Sarah Sinnett and Steven Gray1 of UT Southwestern Medical Center….
…Pharmacologic activity of TSHA-102 following intrathecal (IT) administration was assessed in the MECP2 knockout mouse model of Rett syndrome
across three dose levels and three age groups (n=252). A one-time IT injection of TSHA-102 significantly increased survival at all dose levels, with the
mid to high doses improving survival across all age groups compared to vehicle-treated controls. Treatment with TSHA-102 significantly improved
body weight, motor function and respiratory assessments in MECP2 knockout mice.
…These preclinical safety and efficacy data will be presented at the International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting
taking place April 26-27, 2022 in Nashville, Tennessee.