European Commission Grants TASHA-102 Orphan Designation For Treatment Of Rett Syndrome

Release Date: 27-Sep-2021

European Commission has granted TSHA-102, an AAV9-based gene replacement therapy to treat Rett syndrome. TSHA-102 delivers the MECP2 gene, which includes the novel miRNA-Responsive Auto-Regulatory Element (miRARE), to regulate MECP2 expression in a genotypic-dependent manner on a cell-by-cell basis. The miRARE technology is designed to prevent toxicity associated with overexpression of MECP2.  Submission of an IND/CTA filing for TSHA-102 is expected in the second half of 2021, with initiation of a Phase 1/2 clinical trial by year-end 2021.

Rett syndrome mainly occurs in females is a severe genetic neurodevelopmental disorder caused by a mutation in the X-linked MECP2 gene essential for neuronal and synaptic function in the brain. Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 in the U.S and European Union.


Orphan designation by the EMA is designed to encourage the development of new treatments for life-threatening or chronically debilitating conditions affecting no more than five in 10,000 people in the European Union. Medicines that meet the criteria for orphan designation potentially qualify for several incentives, including 10-year market exclusivity, reduced fees for regulatory activities and protocol assistance.

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