Corporate deck outlines TSHA-102 Rett syndrome strategy
Taysha outlined a registration strategy for TSHA-102, a one-time intrathecal AAV9 gene therapy candidate for Rett syndrome.
REVEAL pivotal trial dosing completed in 17 females ages 6 to <22 years at 1x10^15 total vg; 6-month interim readout targeted for 1H 2027.
REVEAL Part A data showed a 100% response rate on the primary endpoint in 12 patients; no treatment-related serious adverse events or dose-limiting toxicities reported.
FDA alignment cited for a potential BLA submission based on the pivotal trial 6-month interim analysis, with Part A data included for comparability.
ASPIRE trial in 4 females ages 2 to <4 years remained ongoing; dosing completion expected in July 2026 to support a broad label for patients age 2+.
TSHA•
