A phase 1/2 clinical study of TSHA-102, an innovative gene treatment for Rett syndrome, yielded promising results. One adult patient with advanced Rett syndrome participated in the research. The therapy was determined to be safe and well-tolerated after treatment, with no major adverse effects. At 4 weeks post-treatment, the patient improved in various parameters, including motor abilities, vocalization, and autonomic functioning. For the first time in over a decade, the patient was able to sit alone and utilize her fingers and hands. Based on these findings, a second patient’s dose was authorized, and more adult patients are likely to be enrolled in the second half of 2023. Taysha is also working on another gene treatment for large axonal neuropathy called TSHA-120.

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