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» Capsida’s investigational epilepsy gene therapy gets orphan status
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Neurology/psychiatric
Capsida’s investigational epilepsy gene therapy gets orphan status
Oct. 31, 2024
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The FDA has granted orphan drug designation to Capsida Biotherapeutics Inc.’s CAP-002, an investigational gene therapy for the treatment of developmental and epileptic encephalopathy due to syntaxin-binding protein 1 (STXBP1) mutations.
BioWorld Science
Regulatory
Neurology/psychiatric
FDA
Orphan drug