CAP-002

CAP-002 is a first-in-class, intravenously administered gene therapy developed by Capsida Biotherapeutics for the treatment of syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE), a rare and severe neurodevelopmental disorder caused by mutations in the STXBP1 gene. The therapy uses a proprietary engineered adeno-associated virus (AAV) capsid to deliver a functional copy of the STXBP1 gene across the blood-brain barrier, achieving widespread neuronal transduction while significantly detargeting off-target tissues such as liver and dorsal root ganglia. Preclinical studies demonstrated that CAP-002 can restore STXBP1 protein levels in up to 70% of neurons, reverse cognitive and motor deficits, reduce seizures, and correct disease phenotypes in animal models. The drug has shown robust safety with no significant adverse findings in non-human primate studies. As of mid-2025, CAP-002 is being evaluated in Phase 1/2a clinical trials for pediatric patients with STXBP1-DEE[1][2][3][4][5][6][7][8][9].

Other names

STXBP1 gene therapySTXBP-1 gene therapySTXBP 1 gene therapy