Gene therapy trial for rare childhood epilepsy halted after just one patient
Disease control
Terminated
This trial tested a single dose of CAP-002 gene therapy in children aged 18 months to 8 years with STXBP1 encephalopathy, a rare genetic brain disorder causing seizures and developmental delays. The study aimed to check safety and whether it could reduce seizures and improve skil…
Phase: PHASE1, PHASE2 • Sponsor: Capsida Biotherapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:06 UTC