First Pediatric Patient Dosed in Taysha US Clinical Trial

On January 10, 2024 Taysha Gene Therapies announced that their US-based REVEAL gene therapy clinical trial has dosed the first pediatric Rett syndrome patient with the TSHA-102 gene therapy. The pediatric trial in the US builds on the ongoing adolescent and adult trial taking place in Canada.

The first phase of the US study is enrolling six children ages 5 to 8 and is designed to evaluate whether TSHA-102 is safe and tolerable, and whether there are beneficial effects. Initial safety and efficacy data for cohort one is expected in mid-2024. The second phase of the study will expand the ages to include 3 to 8 years old, with a total of up to 27 patients enrolled. The study will test two different dose levels.

Taysha also announced that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has authorized proceeding with a pediatric clinical trial in the UK. The Taysha program will now be evaluating results across pediatric, adolescent, and adult patients in three countries—the US, UK, and Canada.

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