Key Takeaways RCKT's resubmitted BLA for Kresladi to treat severe LAD-I has been accepted by the FDA.FDA's decision is expected by March 28, 2026, with potential approval marking RCKT's first product.The resubmission follows an earlier FDA CRL for Kresladi BLA issued to RCKT last June.Rocket Pharmaceuticals (RCKT) announced that the FDA has accepted the resubmitted biologics license application (BLA) seeking approval for its gene therapy Kresladi (marnetegragene autotemcel; marne-cel) to treat patients with ...

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 ...