Taysha Gene Therapies, Inc. announced the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome. RMAT designation was granted following the FDA?s review of clinical data supporting the potential of TSHA-102 to address the unmet medical need for patients with Rett syndrome. RMAT designation was designed to expedite the development and review of regenerative medicine therapies.

A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious condition, and preliminary clinical evidence indicates the therapy has the potential to address unmet medical needs for such condition. Sponsor companies receiving RMAT designation can benefit from increased interactions with the FDA involving senior managers, with the goal of expediting drug development. RMAT designation follows the FDA?s review of available safety and efficacy data from the first three patients with Rett syndrome dosed with the low dose of TSHA-102 (5.7x1014 total vg) across the REVEAL Phase 1/2 adolescent and adult trial and the REVEAL Phase 1/2 pediatric trial.

TSHA-102 is being evaluated in the REVEAL Phase 1/2 adolescent and adult trial taking place in Canada and the U.S., and in the REVEAL Phase 1/2 pediatric trial taking place in the U.S. and cleared in the U.K.