Gene Therapy CAP-002 Gains FDA Orphan Drug Designation for STXBP1 Developmental and Epileptic Encephalopathy

Capsida’s CAP-002 gene therapy, currently advancing through IND-enabling studies, aims to address the unmet needs of patients with STXBP1-related developmental and epileptic encephalopathy by stably supplementing STXBP1 protein.:

Source: Neurology Read More