The FDA cleared Rocket Pharma’s gene therapy Kresladi for severe leukocyte adhesion deficiency type 1 (LAD-1), an ultra-rare disorder that can leave children vulnerable to life-threatening infections. STAT+ reported the therapy isn’t expected to become a major revenue driver given the estimated case size, but the clearance is a tangible step forward for Rocket following value losses after setbacks in 2021. Kresladi was initially rejected by the FDA in 2024 over manufacturing concerns, making this clearance a manufacturing quality turnaround as well as a clinical regulatory milestone. The therapy is approved for children without a matched sibling stem cell donor, where stem cell transplant remains the standard of care. The approval also underscores how FDA decisions in rare disease can pivot on production controls and consistency, even when the underlying therapeutic concept is already established. Rocket will now face the commercial challenge of delivering a complex, one-time therapy for a small eligible population. From an industry standpoint, LAD-1 clearance adds to the accumulating set of precedent cases for gene therapy commercialization pathways—particularly for manufacturing-sensitive reviews.