Capsida Biotherapeutics Updates Investigation Into Death of Child in CAP-002 Gene Therapy Trial
The company said Tuesday it remains unable to determine what caused the death of a child last September in a trial of its experimental brain-targeted therapy CAP-002. Scientists have been blocked from full investigation because the hospital has declined to provide autopsy tissue samples. CAP-002 was the first in a new class of gene therapies designed to cross the blood-brain barrier.
prnewswire.comCapsida Biotherapeutics said Tuesday that it still had no answers in its investigation into the death of a child in a gene therapy trial last September. The company stated that its scientists’ efforts have been stymied because the hospital where the study was conducted has declined to share tissue samples from an autopsy. The therapy is known as CAP-002.
It was the first of a wave of new gene therapies designed to deliver genes deep into the brain. Scientists around the world engineered viruses that could slide through the blood-brain barrier. The blood-brain barrier walls off the brain from the rest of the body.
Companies have developed promising treatments using these viruses for devastating rare genetic diseases and common conditions like Alzheimer’s and Parkinson’s. The child’s death occurred in September 2025.
Key Facts
Story Timeline
2 events- 2026-05-12
Capsida Biotherapeutics states it still has no answers in the investigation and that the hospital has declined to share autopsy tissue samples
1 sourceStat - 2025-09-01
Child dies during Capsida Biotherapeutics gene therapy trial of CAP-002
1 sourceStat
Potential Impact
- 01
Delayed understanding of potential safety risks in brain-targeted gene therapies
- 02
Potential setback for companies developing similar virus-based treatments that cross the blood-brain barrier
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