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STAT+: Participant in Rocket Pharmaceuticals gene therapy study dies
A patient in a Rocket Pharmaceuticals gene therapy trial for a rare heart condition has died, the company said.
Rocket Pharmaceuticals said Tuesday that a patient had died in a trial studying its experimental gene therapy for a rare heart condition called Danon disease.
Rocket did not disclose the patient’s age, although the Phase 2 study aimed to enroll boys and young men. The company said it was still investigating the cause of the death. It suspects a drug given to suppress patients’ immune systems before receiving the gene therapy may have played a role.
The Food and Drug Administration placed the trial on hold on May 23, after the patient developed a life-threatening complication called capillary leak syndrome. He died shortly after from a systemic infection.
