Capsida Biotherapeutics announced the US Food and Drug Administration has granted orphan drug designation to its investigational gene therapy for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE). According to the company, STXBP1-DEE is a severe genetic disorder characterized by treatment-resistant seizures and developmental delays.
The company said its gene therapy is designed to deliver a functional copy of the STXBP1 gene using its proprietary capsid engineering platform. Capsida claims this platform enables targeted delivery to the central nervous system while limiting exposure to peripheral tissues.
According to the company, the orphan drug designation provides benefits including tax credits for clinical development, exemption from certain FDA fees, and seven years of market exclusivity if the therapy is approved. Capsida said the designation reflects the high unmet need for effective treatments for STXBP1-DEE.
The company claims its gene therapy program aims to restore normal synaptic function by addressing the underlying genetic cause of the disease. Capsida said preclinical data support further development and it plans to advance the candidate toward clinical evaluation.
Capsida claims this milestone highlights the potential of its gene therapy platform to address rare and severe neurological conditions by targeting genetic drivers of disease.
