Capsida Biotherapeutics still unable to determine cause of child death in brain-targeted gene therapy trial

Capsida Biotherapeutics reported that it still has no explanation for the death of a child who participated in its CAP-002 gene therapy trial last September.

The trial was intended to test a new class of brain-targeted gene therapies that use viral vectors to cross the blood-brain barrier, a strategy being explored for rare genetic disorders and diseases such as Alzheimer’s and Parkinson’s.

Capsida said its investigation has been hampered because the hospital where the study was conducted refused to provide tissue samples from the autopsy, limiting the scientists’ ability to identify the underlying cause.

The lack of answers highlights ongoing safety concerns in the emerging field of intracerebral gene delivery and may affect investor confidence and regulatory scrutiny of similar programs.