Overview

• The child died a few days after receiving CAP-002, the first dose given in the early-stage study.
• Capsida suspended the trial and said it will provide regulators a full report on the death.
• Investigators are conducting a root-cause analysis, and it remains unclear whether the treatment contributed.
• The FDA cleared the study in May, with plans to enroll about 12 children at Weill Cornell Medicine and the Children’s Hospital of Philadelphia.
• CAP-002 uses an engineered viral vector to reach the brain for STXBP1-related developmental and epileptic encephalopathy, and the STXBP1 Foundation said it will share verified updates with families.