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doi: 10.1038/nm.3009 44 CoskunTSloopKWLoghinCAlsina-FernandezJUrvaSBokvistKBet al

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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