Patient dies in Rocket Pharmaceuticals’ Phase II Danon disease gene therapy trial (2025)
A patient has died in a pivotal Phase II trial of Rocket Pharmaceuticals’ gene therapy for Danon disease after suffering a serious adverse event (AE). Rocket said that the patient received a dose of RP-A501 in the Phase II study (NCT06092034) and experienced clinical complications related to capillary leak syndrome. The patient later died after suffering an acute systemic infection. Rocket Pharmaceutical CEO Dr Gaurav Shah said: “We are heartbroken by this loss and are fully committed to our mission to develop gene therapies that address the underlying cause of devastating diseases like Danon. We are immensely grateful for the patients and families who participate in this important research.” Following the 27 May announcement, Rocket Pharmaceuticals’ stock, listed on the Nasdaq exchange, opened 63.32% lower than its close on 23 May. William Blair healthcare analyst, Dr. Sami Corwin, said this drop is likely due to investors being hesitant about the programme’s safety and time to clinic...