NEW YORK – Taysha Gene Therapies on Wednesday said it has dosed the first pediatric patient in a Phase I/II trial of TSHA-102, it's gene therapy for Rett syndrome.
In the study, dubbed REVEAL, investigators are assessing the safety and preliminary efficacy of TSHA-102 in patients with the rare neurodevelopmental disorder. Rett syndrome is caused by certain mutations in the MECP2 gene and can lead to intellectual disabilities, loss of communication, and seizures, and is most commonly seen in women.
TSHA-102 is an adeno-associated virus 9 vector-based gene therapy that's administered intrathecally. It's designed to regulate cellular MECP2 protein expression.
Dallas-based Taysha in June kicked off the adolescent and adult version of the REVEAL trial in Canada, specifically testing the gene therapy in women with a loss-of-function mutation in MECP2. In this new pediatric trial, which launched in the US, the firm expanded the study to include female patients ages 5 to 8 years old and with stage 3 Rett syndrome.
"Dosing the first pediatric patient with Rett syndrome marks an important step forward in our efforts to broaden the clinical evaluation of TSHA-102 to younger patients with earlier stages of Rett syndrome," Taysha President and Head of R&D Sukumar Nagendran said.
The UK Medicines and Healthcare Products Regulatory Agency has also authorized the clinical trial in pediatric patients.
REVEAL is an open-label, randomized, dose-escalation, and dose-expansion study. In the pediatric study, investigators will initially determine the maximum tolerated or administered dose in patients ages 5 to 8 years old. In a second part of the study, they will evaluate that dose in two age cohorts: 5 to 8 years old and 3 to 5 years old.
Taysha expects to report initial safety and efficacy data from the low-dose cohort of the pediatric trial in mid-2024.