Capsida Biotherapeutics Pauses Rare Disease Gene Therapy Trial After Patient Death

Capsida Biotherapeutics has halted its Phase I/II clinical trial of gene therapy CAP-002 targeting STXBP1 encephalopathy in pediatric patients following the death of the first enrolled child. The company is investigating the cause while maintaining communication with the patient community. This development presents a setback for an emerging gene therapy approach aiming to treat difficult neurological disorders. It also underscores the inherent risks and vigilance required in early-stage gene therapy clinical research.