TSHA-102

TSHA-102 is a self-complementary, intrathecally delivered adeno-associated virus serotype 9 (AAV9) investigational gene therapy designed for the treatment of Rett syndrome. It delivers a codon-optimized human MECP2 gene to cells in the central nervous system (CNS), aiming to address the genetic root cause of Rett syndrome by restoring functional MeCP2 protein. The therapy utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform, which regulates MECP2 expression on a cell-by-cell basis to prevent both underexpression and toxic overexpression of MeCP2. TSHA-102 is being developed as a one-time treatment and has received multiple regulatory designations including Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease from the FDA[1][3][5][8].

Other names

AAV9-miniMECP2-miRAREAAV-9-miniMECP2-miRAREAAV 9-miniMECP2-miRARE