Taysha Gene Therapies Announces Positive Results for TSHA-102 in Rett Syndrome Trials

1. Positive Results: Taysha Gene Therapies has announced positive data from its pivotal phase 1/2 REVEAL studies assessing TSHA-102, an adeno-associated virus (AAV)-based gene therapy for patients with Rett syndrome.
2. Durable Improvements: Treatment with TSHA-102 resulted in durable improvements across clinical domains in both adult and pediatric patients, including motor skills, communication/socialization, autonomic function, and seizures.
3. Safety Profile: The therapy demonstrated a safe profile with no serious adverse events or dose-limiting toxicities observed in the trials.
4. Patient Outcomes: Adult patients showed significant improvements in motor skills, social interest, and seizure control, while pediatric patients demonstrated early signs of developmental gains and improved hand function.
5. Trial Expansion: The company is expanding the trials, lowering the minimum age in the adolescent and adult study to 12 years and moving to a higher dose in both trials.
6. Broad Treatment Potential: The results suggest that TSHA-102 may be effective in a range of Rett patients with different genetic mutations and disease severities.