Taysha Gene Therapies Soars 53% on FDA Breakthrough Designation
Gene therapy candidate TSHA-102 for Rett syndrome receives key designation, paving the way for an accelerated regulatory pathway.
Shares of Taysha Gene Therapies (NASDAQ: TSHA) skyrocketed over 53% in heavy trading after the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to its leading gene therapy candidate, TSHA-102, for the treatment of Rett syndrome. The announcement triggered a massive surge in investor interest, with trading volume swelling to more than 25 times the daily average.
The Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The FDA's decision was based on a review of compelling clinical data from the REVEAL Phase 1/2 trials, which demonstrated a for the trial's primary endpoint of patients gaining or regaining at least one developmental milestone.
In a significant concurrent development, Taysha announced it had reached an alignment with the FDA on the protocol for its upcoming pivotal trial. This alignment could accelerate the company's timeline for submitting a Biologics License Application (BLA). Patient enrollment for this pivotal study is slated to begin in the fourth quarter of 2025, according to a .
Rett syndrome is a rare and severe neurodevelopmental disorder caused by mutations in the MECP2 gene, primarily affecting females. There are currently no approved therapies that address the underlying genetic cause of the disease. TSHA-102 is an investigational AAV9 gene therapy delivered intrathecally, designed to introduce a functional copy of the MECP2 gene into the central nervous system.
The market's emphatic response underscores investor optimism that the FDA designation significantly de-risks the regulatory path for TSHA-102. The designation provides Taysha with more intensive FDA guidance and eligibility for priority review. This latest development, , positions Taysha as a formidable player in the race to bring a disease-modifying treatment to patients with Rett syndrome.