Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) has suspended dosing in its Danon disease gene therapy trial after a patient developed fatal complications from capillary leak syndrome, which caused a severe drop in blood pressure and led to an acute systemic infection. The FDA placed a formal clinical hold on the trial following the incident.
The company is investigating the cause, focusing on a newly introduced immune suppression agent designed to prevent complement system activation before administering the gene therapy. Rocket Pharmaceuticals, Inc. is collaborating with the FDA, an independent data board, and trial investigators to review safety measures and resume the study as soon as possible.
A microbiologist studying a petri dish culture of a gene therapy on a microscope.
The patient’s death has raised concerns among analysts about the unpredictable risks of gene therapies, with Jefferies warning that such events complicate investment decisions in the sector. However, BMO Capital Markets noted that the benefit-risk profile for RCKT’s RP-A501 remains justified, given the high mortality rate in Danon disease patients.
This incident adds to a series of recent safety setbacks in the gene therapy field, including patient deaths in trials by Sarepta Therapeutics, Neurogene, and Beam Therapeutics. Rocket Pharmaceuticals, Inc. reported $318.2 million in cash, providing financial runway into 2027, but analysts caution that cash burn will need close monitoring.
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