Taysha Gene Therapies Inc. has provided an update on TSHA-102, its gene therapy candidate for Rett syndrome. The company highlighted that TSHA-102 is designed as a potential one-time treatment targeting the genetic root cause of Rett syndrome, a rare neurodevelopmental disorder primarily affecting females. The therapy uses an AAV9 capsid and is delivered intrathecally, aiming for broad central nervous system biodistribution while minimizing systemic exposure. The pivotal REVEAL trial is underway, enrolling patients aged 6 to under 22 years, with the primary endpoint focused on the proportion of patients who gain or regain at least one developmental milestone. Interim six-month data may serve as the basis for a biologics license application (BLA) with the FDA. Early data from Part A of the REVEAL trial indicated a 100% response rate for the primary endpoint, with no treatment-related serious adverse events or dose-limiting toxicities reported. You can access the full presentation through the link below.
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