Taysha Gene Therapies, Inc. (TSHA)'s TSHA-102 Shows Promising Results in Rett Syndrome Patients Aged 6+

Taysha Gene Therapies, Inc. (NASDAQ:TSHA) has announced pivotal progress in its TSHA-102 gene therapy program for Rett syndrome, following written alignment with the FDA on a single-arm, open-label pivotal trial targeting patients aged six and older.

This population, shown by natural history data to have virtually zero chance of regaining developmental milestones, saw a 100% responder rate in the REVEAL Part A trial: all 10 treated patients gained or regained at least one developmental milestone post-TSHA-102, with high-dose recipients showing faster and deeper improvements. No serious adverse events or dose-limiting toxicities were reported.

Taysha Gene Therapies, Inc. (NASDAQ:TSHA)'s pivotal Part B trial, expected to launch in Q3 2025, will enroll 15 females in the developmental plateau stage, using each patient as their own control and video-based, blinded milestone assessment as the primary endpoint. The FDA’s support, based on robust longitudinal data from over 1,100 Rett patients, marks a major regulatory milestone.

TSHA-102, a one-time AAV9 gene therapy delivering functional MECP2, is designed to address the genetic root cause of Rett syndrome, a devastating disorder with no current disease-modifying treatments. Taysha Gene Therapies, Inc. (NASDAQ:TSHA) will present further details at the IRSF Rett Syndrome Scientific Meeting in June.

While we acknowledge the potential of TSHA to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk.  If you are looking for an AI stock that is more promising than TSHA and that has 100x upside potential, check out our report about this cheapest AI stock.

READ NEXT: 10 Best Cancer Stocks to Invest in for Long-Term Gains and 10 Most Oversold Stocks to Buy According to Billionaires.

Disclosure: None.