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Genetic Therapies for Brain Diseases

Taysha takes over development of giant axonal neuropathy gene therapy

April 12, 2021 Taysha Gene Therapies has acquired exclusive worldwide rights to a clinical-stage adeno-associated virus 9 (AAV9) gene therapy program (TSHA-120) for the treatment of giant axonal neuropathy. TSHA-120 is currently being evaluated in an ongoing open-label, nonrandomized, dose-escalation clinical trial conducted by the U.S. National Institute of Neurological Disorders and Stroke (NINDS) division of the National Institutes of Health (NIH). The primary end point is safety, and a primary measure of TSHA-120 s clinical efficacy is the Motor Function Measure 32 (MFM32) score. TSHA-120 has demonstrated the potential to improve MFM32 scores in patients. Additional data from the study are expected later this year.

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