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Taysha Gene Therapies’ TSHA-102 Receives FDA Breakthrough Therapy Designation for Rett Syndrome

06 Oct 2025
Admin
News Article

Taysha Gene Therapies’ TSHA-102 Receives FDA Breakthrough Therapy Designation for Rett Syndrome

Taysha Gene Therapies announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to TSHA-102, its investigational gene therapy for Rett syndrome.

Multiple Regulatory Designations

TSHA-102 has secured several regulatory recognitions, including:

FDA: Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations
European Commission: Orphan Drug designation
UK regulator: Innovative Licensing and Access Pathway (ILAP) designation

These designations highlight the therapy’s potential to address a serious unmet need in Rett syndrome, a rare genetic neurodevelopmental disorder caused by mutations in the MECP2 gene.

About Rett Syndrome

Rett syndrome primarily affects females and is characterized by:

Loss of communication skills and impaired hand function
Developmental regression and motor difficulties
Respiratory issues, seizures, and intellectual disability

There are no approved disease-modifying therapies, and it is estimated to affect 15,000–20,000 patients across the US, EU, and UK.

TSHA-102 and Clinical Progress

TSHA-102 is an intrathecally delivered AAV9 gene therapy designed as a one-time treatment to deliver a functional form of MECP2 to cells in the central nervous system.

Clinical data from Part A of the ongoing REVEAL Phase 1/2 trials showed a favorable safety profile and a 100% response rate at the May 2025 cutoff, with patients achieving at least one developmental milestone.
Dose-related improvements were observed in motor behavior and clinician-assessed global improvement.

Pivotal REVEAL Trial

The REVEAL pivotal trial will evaluate a single high dose of TSHA-102 in 15 females aged 6 to under 22 years with Rett syndrome in the developmental plateau stage.

Primary endpoint: Proportion of patients achieving one or more developmental milestones in communication, fine motor, or gross motor domains
Design: Each patient serves as their own control, with standardized, video-reviewed assessments by independent blinded raters
Interim analysis: Conducted at six months, potentially supporting a future Biologics License Application (BLA)

Previous findings indicated that 83% of patients receiving high-dose treatment achieved milestone gains within six months.