Core Insights - Taysha Gene Therapies Inc. has regained full global rights to its lead program, TSHA-102, for the treatment of Rett syndrome, following the expiration of the 2022 Option Agreement with Astellas [1][2] - TSHA-102 is a one-time investigational gene transfer therapy designed to address the genetic root cause of Rett syndrome by delivering a functional form of the MECP2 gene to CNS cells [3][4] Company Overview - Taysha Gene Therapies Inc. is a clinical-stage biotechnology company focused on developing and commercializing adeno-associated virus-based gene therapies for monogenic diseases affecting the central nervous system [4] Program Details - The therapy TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element/miRARE technology, which allows for the regulation of MECP2 levels in the CNS on a cell-by-cell basis, minimizing the risk of overexpression [3]
Taysha Gene Therapies Regains Full Global Rights to Lead Rett Syndrome Program TSHA-102