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# FDA Gives Fast Track to Rett Gene Therapy
- URL: https://www.fdaweb.com/fda-gives-fast-track-to-rett-gene-therapy/
- Published: 2023-08-24T12:00:00.000Z
- Updated: 2026-09-14T18:52:33.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5155176

FDA has granted Taysha Gene Therapies a fast track designation for TSHA-102, an intrathecally delivered AAV9 gene transfer therapy for treating Rett syndrome. The therapy uses the company’s miRNA-Responsive Auto-Regulatory Element (miRARE) technology, which is designed to mediate levels of the MECP2 gene in the central nervous system on a cell-by-cell basis without the risk of overexpression, the company says.

“Initial data from the first adult patient in Canada with severe disease dosed with TSHA-102 is encouraging, and we expect to dose the second patient in our ongoing [REVEAL Phase 1/2 adult trial](https://www.globenewswire.com/Tracker?data=bStjXsZloTUhoXmSmg59Ji5h%5F0oVMizCEIs7x3WoJtYuoGSLzro%5FKjgvn3gvifrcymJAShUDNOC4kLkTefpvIenmkXD6xqrifsYilyYo3Q4Tp3xly0e0VzhNj2ny4i9N%5FOlA%5Ft5xiPpoxeIsGnhP4Y2sEKrfEF6RFiCQ%5Fihk%5FW9gruek-eyt7l0y7tQIYUuG&ref=fdaweb.com) in the current quarter,” the company says. “We look forward to expanding the clinical evaluation to earlier stages of disease progression following recent FDA clearance to initiate clinical development of TSHA-102 in pediatric patients in the United States.”

Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2gene, which is essential for neuronal and synaptic function in the brain, according to Taysha. Rett syndrome is characterized by intellectual disabilities, loss of communication, seizures, slowing and/or regression of development, motor and respiratory impairment, and shortened life expectancy.