Taysha Gene Therapies to present TSHA-102 data at 2026 Rett meeting
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Taysha Gene Therapies (Nasdaq: TSHA) announced it will present multiple data sets from its TSHA-102 clinical program at the 2026 International Rett Syndrome Foundation Scientific Meeting in Prior Lake, MN, running June 29 through July 1, 2026.
The presentations include an oral session and three poster presentations covering clinical, natural history, and preclinical data related to TSHA-102, an investigational intrathecally delivered AAV9 gene therapy in development for Rett syndrome.
Clinical data from the REVEAL Part A Phase 1/2 trial will be presented by Elsa Rossignol, M.D., a principal investigator of the trial and professor at the Université de Montréal. According to the company, longer-term data from the trial showed functional improvements across multiple domains through at least 12 months after dosing, regardless of patient age or disease severity.
A separate presentation will cover a natural history data analysis indicating that patients with Rett syndrome reach a developmental plateau after age six, which the company states supports use of that population in its planned REVEAL pivotal trial.
Additional presentations will address the Rett Syndrome Developmental Milestone Assessment as a primary endpoint for interventional studies, and preclinical data comparing MeCP2 expression levels between self-complementary and single-stranded AAV9 constructs.
TSHA-102 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. Rett syndrome is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K., and currently has no approved disease-modifying therapy targeting its genetic cause, according to the company's press release statement.
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