On Tuesday, the U.S. Food and Drug Administration (FDA) accepted Rocket Pharmaceuticals, Inc.’s (NASDAQ:RCKT) resubmission of the Biologics License Application (BLA) for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).
LAD-I is a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant.
