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The Cranbury, New Jerseybased biotech announced FDA acceptance of its Biologics License Application for RP-L201 (marnetegragene autotemcel), an investigational lentiviral vector (LV)-based gene therapy for LAD-I, a rare, genetic immune disorder that predisposes patients to frequent and often fatal infections

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Patients should seek urgent medical attention for red flag symptoms including severe or persistent vomiting, inability to keep fluids down for more than 24 hours, progressive abdominal distension, inability to pass stool or gas, or severe abdominal pain

[1] [2] Understanding the potential causes requires consideration of both the medication's pharmacological effects and indirect physiological changes