Capsida Biotherapeutics said it still lacks answers in its investigation into a child’s death in its CAP-002 gene therapy trial last September. The company said its efforts have been stymied because the hospital conducting the study has declined to share autopsy tissue samples. CAP-002 is designed as a brain-delivery gene therapy using engineered viruses intended to cross the blood-brain barrier. The case adds to scrutiny around patient safety and post-event transparency in neurotropic gene therapy programs. The update matters for the field because it highlights how investigations can be slowed by data access and underscores the importance of early governance around tissue and investigation artifacts.
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