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Taysha Gene Therapies Granted Fast Track Designation by U.S. FDA for TSHA-102 in Rett Syndrome Treatment

Friday, August 25, 2023

Taysha Gene Therapies, Inc. has been granted Fast Track Designation (FTD) by the U.S. FDA for TSHA-102, its AAV9 gene transfer therapy developed to treat Rett syndrome. Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the MECP2 gene, resulting in severe cognitive and motor impairments.

TSHA-102 employs a unique miRNA-Responsive Auto-Regulatory Element (miRARE) technology to precisely regulate MECP2 levels in the CNS on a cell-by-cell basis, minimizing the risk of overexpression. The Fast Track Designation aims to expedite the development and review process for therapies addressing critical medical needs. This designation facilitates increased engagement with the FDA during clinical development and potential pre-submission reviews of marketing applications.

Dr. Sukumar Nagendran, President and Head of R&D at Taysha, conveyed optimism about the potential of TSHA-102 as a Rett syndrome treatment, citing encouraging initial data from a Canadian patient. The ongoing REVEAL Phase 1/2 adult trial continues to evaluate its effectiveness. Furthermore, Taysha plans to extend clinical assessment to pediatric patients following FDA clearance for clinical trials in this demographic.

Rumana Haque-Ahmed, Senior Vice President of Regulatory Affairs at Taysha, emphasized the urgency of developing therapies targeting the genetic roots of Rett syndrome due to its profound impact on patients' lives. The FTD for TSHA-102 represents a significant advancement in addressing this unmet medical need.

At present, TSHA-102 is undergoing evaluation in the REVEAL Phase 1/2 adult trial in Canada. The company has gained FDA clearance to initiate trials of TSHA-102 in pediatric patients, with the first pediatric patient projected to receive treatment in Q1 2024.

Rett syndrome, resulting from mutations in the MECP2 gene, leads to intellectual disabilities, loss of communication, seizures, developmental regression, and motor impairments. The disorder lacks approved disease-modifying therapies that target its underlying genetic cause.

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