Taysha's REVEAL Trial for Rett Syndrome Gene Therapy Begins Late 2025
Taysha Gene Therapies, a biotech firm, has finalised plans for its pivotal REVEAL trial of TSHA-102, a potential gene therapy for Rett syndrome. This rare neurological disorder affects around 15,000 to 20,000 patients in the U.S., EU, and UK. The trial, set to begin in late 2025, aims to treat a condition with no current disease-modifying therapies.
Rett syndrome is caused by mutations in the X-linked MECP2 gene. Taysha's lead program, TSHA-102, uses AAV-based gene therapy to address this root cause. The REVEAL trial will assess a single intrathecal dose of TSHA-102 in 15 females aged 6 to under 22. The primary endpoint is response rate, measured by developmental milestone gains or regains.
The FDA has granted TSHA-102 Breakthrough Therapy designation, expediting its development. The pivotal trial's protocol and statistical analysis plan have been finalized with the FDA. Patient enrollment begins in Q4 2025, with a potential 6-month interim analysis accelerating BLA submission.
Taysha Gene Therapies' REVEAL pivotal trial for TSHA-102, starting late 2025, offers hope for Rett syndrome patients. If successful, it could provide the first disease-modifying therapy addressing the disorder's genetic root cause.
