FDA Policy/General

Capsida Pauses Gene Therapy Trial After Death

Capsida Biotherapeutics has halted enrollment in its SYNRGY clinical trial that is evaluating CAP-002, an investigational intravenous (IV)-administered gene therapy for treating STXBP1 developmental and epileptic encephalopathy, due to a patient death in the trial. The company says CAP-002 is the first IV-delivered, adeno-associated virus blood brain barrier-crossing genetic medicine entering a human clinical trial.

The company disclosed the development in a letter to the STXBP1 community. “We have voluntarily paused the CAP-002 SYNRGY study while we determine the root cause of the patient’s passing,” it said. The company confirmed it has notified FDA and will submit a full report in compliance with regulatory requirements.

The trial, part of Capsida’s gene therapy program for STXBP1-Related Disorders, is designed to address severe neurological symptoms associated with mutations in the STXBP1 gene. These disorders often present in infancy with developmental delay, seizures, and motor impairment.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome