Taysha Gene Therapies announces updates to TSHA-102 program

Taysha Gene Therapies announced updates to its TSHA-102 program in clinical evaluation for the treatment of Rett syndrome. The Independent Data Monitoring Committee, IDMC, approved the Company’s request to proceed to dose escalation in the REVEAL Phase 1/2 adolescent and adult trial, enabling earlier advancement to the high dose cohort of 1×1015 total vg. The IDMC also approved the dosing of the second patient in cohort one of 5.7×1014 total vg in the REVEAL Phase 1/2 pediatric trial. The decisions follow IDMC review of available clinical data from the patients dosed with TSHA-102. REVEAL Phase 1/2 adolescent and adult trial: a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adolescent and adult females aged 12 years and older with Rett syndrome due to MECP2 loss-of-function mutation. Dosing in cohort one is now considered complete. Update on available clinical data from completed cohort one of 5.7×1014 total vg expected in the first quarter of 2024. Dosing of the first patient in cohort two of 1×1015 total vg expected in the second quarter of 2024. Initial safety and efficacy data from cohort two expected in the second half of 2024. REVEAL Phase 1/2 pediatric trial: a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in pediatric females with Rett syndrome due to MECP2 loss-of-function mutation. The trial is taking place in the U.S. and cleared in the U.K. TSHA-102 is administered as a single lumbar intrathecal injection. Part A of the study will focus on determining MAD and MTD in patients aged 5-8 years old. Part B is the dose expansion phase and will evaluate TSHA-102 at the MAD or MTD in two age cohorts. Dosing of the second patient in cohort one of 5.7×1014 total vg expected in the first quarter of 2024. Initial safety and efficacy data for cohort one expected in mid-2024. Initial safety and efficacy data from cohort two of 1×1015 total vg expected in the second half of 2024.