Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason on Signal at JasonMast.05.
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.