Taysha Gene Therapies’ Epilepsy Drug Gains EU Orphan Drug Status

August 26, 2021

The European Commission granted Dallas, Texas-based Taysha Gene Therapies an Orphan Drug designation for TSHA-105, its investigational gene therapy for a rare form of infantile epilepsy.

The adeno associated virus-based therapy targets a mutation in the SLC13A5 gene that causes SLC13A5 deficiency, a type of epilepsy that begins within the first days of life and is characterized by developmental delay and seizure. An estimated 1,900 patients in Europe and the U.S. suffer from the disease.

Taysha notes that TSHA-105 addresses the underlying cause of the disease, whereas the current standard of care consists of anti-seizure medications which target only the symptoms.

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