Taysha Gene Therapies highlights TSHA-102 Rett gene therapy path toward BLA submission in corporate presentation
TSHA•ASPIRE pediatric dosing
ASPIRE dosing completed in 4 females aged 2 to <4 years; at least 3 months of safety data may support a broad age 2+ label.
TSHA-102 registration path and REVEAL data
Taysha Gene Therapies outlined an accelerated registration plan for TSHA-102, a one-time intrathecal gene therapy candidate for Rett syndrome.
REVEAL Part A showed a 100% primary-endpoint response rate in 12 patients, with no treatment-related serious adverse events or dose-limiting toxicities.
The REVEAL pivotal trial completed dosing in 17 females aged 6 to <22 years at 1x10^15 total vector genomes.
FDA alignment supports a potential BLA filing based on the REVEAL 6-month interim analysis, with Part A data included for comparability.
