Capsida Suspends Gene Therapy Trial After First Patient Death

Capsida Biotherapeutics has suspended its phase 1/2 gene therapy trial for CAP-002 after the death of the first patient treated in the study134.

The CAP-002 therapy was being trialed in children with STXBP1-related developmental and epileptic encephalopathy, a rare neurodevelopmental disorder marked by seizures and developmental delays1345.

The company paused the trial to investigate the root cause of the patient's death and has notified the FDA, pledging to provide a full report and conduct a formal safety review before deciding on next steps1345.

The CAP-002 gene therapy uses a specially engineered adeno-associated virus (AAV) to bypass the blood-brain barrier and target brain neurons, intending to compensate for deficient protein encoded by the STXBP1 gene134.

The affected trial had planned to enroll about 12 children at sites including Weill Cornell Medicine and the Children's Hospital of Philadelphia4.

Capsida has not yet disclosed whether the gene therapy was directly responsible for the patient's death; investigation is ongoing, and no disease-modifying treatments exist for STXBP1-driven conditions35.

This incident adds to recent concerns regarding safety in gene therapy trials following similar patient deaths in other studies targeting rare neurological and muscular disorders3.

Sources:

1. https://www.fiercebiotech.com/biotech/capsida-pauses-phase-1-gene-therapy-trial-after-child-dies

3. https://www.biopharmadive.com/news/capsida-gene-therapy-patient-death-stxbp1-disorders/759881/

4. https://trial.medpath.com/news/9ef8bfce0aad0bd2/capsida-suspends-gene-therapy-trial-after-first-patient-dies-following-treatment-for-rare-stxbp1-disorder

5. https://www.biospace.com/drug-development/capsida-reports-patient-death-in-gene-therapy-trial