Rocket Pharma sinks after patient death, US FDA's clinical hold on gene therapy trial

Rocket Pharmaceuticals

27/05/2025 13:11

** Shares of Rocket Pharmaceuticals RCKT.O plummet 64.11% to $2.25 premarket

** Co says patient participating in mid-stage trial studying its experimental gene therapy, RP-A501, died after acute systemic infection

** U.S. FDA last Friday placed clinical hold on trial to allow further evaluation

** RCKT says it voluntarily paused further dosing in study after it learnt the patient experienced unexpected adverse event involving clinical complications related to capillary leak syndrome, where fluid leaks from small blood vessels into surrounding tissues

** Adds it is unable to provide guidance on anticipated timing for trial's completion

** Trial was studying gene therapy for Danon disease - rare genetic disorder that often affects heart and skeletal muscles and can lead to intellectual disabilities

** Up to last close, stock down ~50% YTD

 (Reporting by Sriparna Roy in Bengaluru)

 ((Sriparna.Roy@thomsonreuters.com;))