Investigational Gene Therapy for Rett Syndrome Gets Fast Track Status

The Food and Drug Administration (FDA) has granted Fast Track designation to TSHA-102 for the treatment of Rett syndrome.

Rett syndrome is a rare genetic neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene. The disorder occurs primarily in girls and is characterized by near constant repetitive hand movements and loss of purposeful hand use in addition to loss of coordination and communication abilities.

TSHA-102 is an investigational adeno-associated virus (AAV) 9 gene transfer therapy that utilizes proprietary technology to mediate levels of MECP2 in the central nervous system on a cell-by cell basis without overexpression.

The phase 1/2 REVEAL trial (ClinicalTrials.gov Identifier: NCT05606614) is currently assessing the safety, tolerability and efficacy of TSHA-102 in 12 adult women with Rett syndrome. A trial in children with Rett syndrome is expected to begin in the first quarter of 2024.

Sukumar Nagendran, MD, President and Head of R&D of Taysha said, “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 in the current quarter.”

The FDA’s Fast Track designation accelerates the development and review of products for serious and life-threatening conditions where no treatment exists or where the investigational therapy is likely to provide an advantage over currently available treatments.

Earlier in 2023, the FDA approved trofinetide for the treatment of Rett syndrome in adult and pediatric patients 2 years of age and older.

This article originally appeared on MPR